WO2019028306A2 - COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES - Google Patents

COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES Download PDF

Info

Publication number
WO2019028306A2
WO2019028306A2 PCT/US2018/045088 US2018045088W WO2019028306A2 WO 2019028306 A2 WO2019028306 A2 WO 2019028306A2 US 2018045088 W US2018045088 W US 2018045088W WO 2019028306 A2 WO2019028306 A2 WO 2019028306A2
Authority
WO
WIPO (PCT)
Prior art keywords
aav
seq
aavhu
aavrh
clv
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2018/045088
Other languages
English (en)
French (fr)
Other versions
WO2019028306A3 (en
Inventor
Dinah Wen-Yee Sah
Holger Patzke
Jinzhao Hou
Mathieu E. NONNENMACHER
Martin Goulet
Todd Carter
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Voyager Therapeutics Inc
Original Assignee
Voyager Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority to KR1020207003192A priority Critical patent/KR102850887B1/ko
Priority to EP20208355.6A priority patent/EP3808849A1/en
Priority to US16/636,094 priority patent/US11512327B2/en
Priority to MX2020001187A priority patent/MX2020001187A/es
Priority to JP2020505424A priority patent/JP7221275B2/ja
Priority to CN201880064712.6A priority patent/CN111448308A/zh
Priority to AU2018311069A priority patent/AU2018311069A1/en
Priority to EP18762419.2A priority patent/EP3662060A2/en
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Priority to CA3071978A priority patent/CA3071978A1/en
Publication of WO2019028306A2 publication Critical patent/WO2019028306A2/en
Publication of WO2019028306A3 publication Critical patent/WO2019028306A3/en
Anticipated expiration legal-status Critical
Priority to US17/970,036 priority patent/US12305189B2/en
Priority to JP2023013699A priority patent/JP2023052805A/ja
Ceased legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

Definitions

  • the present invention relates to compositions, methods and processes for the design, preparation, manufacture, use and/or formulation of adeno-associated virus capsids for improved biodistribution.
  • Adeno-associated viral (AAV) vectors are a promising candidate for therapeutic gene delivery and have proven safe and efficacious in clinical trial.
  • AAV AAV vectors that may be administered by intravenous delivery and yet are able to efficiently target regions critical for treating a multitude of diseases.
  • AAV central nervous system
  • CNS central nervous system
  • invasive surgeries for sufficient levels of gene transfer (See e.g., Bevan et al. Mol Ther. 2011 Nov; 19(11): 1971-1980).
  • Intravenous delivery has historically resulted in limited gene transfer to the CNS, in part due to the presence of the blood brain barrier (BBB).
  • BBB blood brain barrier
  • AAV AAV vectors that may be administered by intravenous delivery and yet are able to efficiently target regions of the cardiovascular system critical for treating a multitude of diseases.
  • the present invention addresses this need by providing novel AAV particles with engineered capsid proteins that allow for efficient transduction of CNS tissues following intravenous delivery. Further, the viral genomes of these AAV particles may be altered to suit the needs of any number of CNS diseases, providing platform capsids for crossing the blood brain barrier and targeting of CNS tissues.
  • the invention provides an adeno-associated viral (AAV) particle comprising a capsid and a viral genome.
  • AAV adeno-associated viral
  • the AAV particle may comprise a capsid or a peptide insert such as, but not limited to, VOY101, VOY201, AAVPHP.B (PHP.B), AAVPHP.A (PHP.A), AAVG2B-26, AAVG2B-13, AAVTHl .1-32, AAVTHl.1-35, AAVPHP.B2 (PHP.B2), AAVPHP.B3 (PHP.B3), AAVPHP.N/PHP.B-DGT, AAVPHP.B-EST, AAVPHP.B -GGT, AAVPHP.B- ATP, AAVPHP.B-ATT-T, AAVPHP.B-DGT-T, AAVPHP.B-GGT-T, AAVPHP.B-SGS, AAVPHP.B-AQP, AAVPHP.B-QQP, AAVPHP.B-SNP(3), AAVPHP.B-SNP, AAVPHP
  • AAVPHP.B-DST AAVPHP.B-DST
  • AAVPHP.B-STP AAVPHP.B-PQP
  • AAVPHP.B- SQP AAVPHP.B-QLP
  • AAVPHP.B-TMP AAVPHP.B-TTP
  • AAVPHP.S/G2A12 AAVG2A15/G2A3 (G2A3)
  • AAVG2B4 (G2B4) AAVG2B5 (G2B5)
  • PHP.S AAV1, AAV2, AAV2G9, AAV3, AAV3a, AAV3b, AAV3-3, AAV4, AAV4-4, AAV5, AAV 6, AAV6.1, AAV6.2, AAV6.1.2, AAV7, AAV7.2, AAV 8, AAV9, AAV9.11, AAV9.13, AAV9.16, AAV9.24, AAV9.45, AAV9.47, AAV9.61, AAV9.68, AAV9.84
  • AAVhu.44R3 AAVhu.45, AAVhu.46, AAVhu.47, AAVhu.48, AAVhu.48Rl,
  • AAV-PAEC AAV- LK01, AAV-LK02, AAV-LK03, AAV-LK04, AAV-LK05, AAV-LK06, AAV-LK07, AAV- LK08, AAV-LK09, AAV-LK10, AAV-LK11, AAV-LK12, AAV-LK13, AAV-LK14, AAV- LK15, AAV-LK16, AAV-LK17, AAV-LK18, AAV-LK19, AAV-PAEC2, AAVAV- LK01, AAV-LK02, AAV-LK03, AAV-LK04, AAV-LK05, AAV-LK06, AAV-LK07, AAV- LK08, AAV-LK09, AAV-LK10, AAV-LK11, AAV-LK12, AAV-LK13, AAV-LK14, AAV- LK15, AAV-LK16, AAV-LK17, AAV-LK18, AAV-LK19, AAV-PAEC2, AAV
  • AAVF14/HSC14 AAVF15/HSC15, AAVF16/HSC16, AAVF17/HSC17, AAVF2/HSC2, AAVF3/HSC3, AAVF4/HSC4, AAVF5/HSC5, AAVF6/HSC6, AAVF7/HSC7,
  • the capsid of the AAV particle is VOY101. In one aspect, the capsid of the AAV particle is VOY201. In one aspect, the AAV particle comprises a peptide insert and the peptide insert is AAVPHP.N. In one aspect, the AAV particle comprises a peptide insert and the peptide insert is AAVPHP.B. In one aspect, the AAV particle comprises a peptide insert and the peptide insert is
  • the AAV particle comprises a peptide insert and the peptide insert is AAVPHP.S.
  • the AAV particle comprises a viral genome which comprises a nucleic acid sequence position between two inverted terminal repeats (ITRs).
  • ITRs inverted terminal repeats
  • the viral genome transduces cardiomyocytes upon delivery of the AAV particle.
  • the delivery may be by any method known in the art, such as, but not limited to, intravenous administration or intracarotid artery delivery.
  • the capsid penetrates the blood brain barrier following delivery of the AAV particle.
  • the delivery may be by any method known in the art, such as, but not limited to, intravenous administration or intracarotid artery delivery.
  • the AAV particles of the present invention may transduce CNS structures following administration.
  • CNS structures include brain, spinal cord, brainstem nuclei, cerebellum, cerebrum, motor cortex, caudate nucleus, thalamus, hypothalamus, cervical spinal cord, thoracic spinal cord, lumbar spinal cord, striatum, substantia nigra, hippocampus, amygdala and/or cerebral cortex.
  • the AAV particle comprises a viral genome which comprises a nucleic acid sequence that, when expressed, inhibits or suppresses the expression of a gene of interest (e.g., SODl, HTT, APOE, and MAPT) in a cell.
  • the nucleic acid sequence comprises a sense strand sequence and an antisense strand sequence which may be independently 30 nucleotides in length or less and, the sense and/or antisense strands may comprise a 3' overhang of at least 1 or at least 2 nucleotides.
  • the sense sequence and antisense strand sequence may share a region of complementarity of at least four nucleotides in length (e.g., at least 17 nucleotides in length, between 19 and 21 nucleotides in length, or 19 nucleotides in length).
  • the antisense strand may be excised from the AAV particle at a rate of at least 80%, 85%, 90%, 95%, or more than 95%, or more than 98%, or more than 99%.
  • the antisense strand may be excised from the AAV particle at a rate greater than the excision of the sense strand (e.g., 2 times, 5 times, 10 times or more than 10 times greater).
  • compositions comprising AAV particles.
  • the AAV particles may comprise a viral genome comprising a nucleic acid sequence encoding a protein of interest (e.g., an antibody, AADC, APOE2, Frataxin, ATP2A2, and/or S100A1).
  • the AAV particles may comprise a viral genome comprising nucleic acid sequences that when expressed, inhibits or suppresses the expression of a gene of interest (e.g., SOD1, HTT, APOE, and/or MAPT) in a cell.
  • a gene of interest e.g., SOD1, HTT, APOE, and/or MAPT
  • a target gene in one aspect, provided are methods of inhibiting the expression of a target gene in a cell (e.g., mammalian cell, or mammalian cell of the CNS, or a cardiomyocyte).
  • a cell e.g., mammalian cell, or mammalian cell of the CNS, or a cardiomyocyte.
  • a target gene in a cell such as a mammalian cell (e.g., a mammalian cell of the CNS, or a cardiomyocyte)
  • a mammalian cell e.g., a mammalian cell of the CNS, or a cardiomyocyte
  • kits for treating and/or ameliorating a neurological disease in a subject by administering a therapeutically effective amount of a composition comprising the AAV particles described herein.
  • the administration may be by intravenous or intracarotid artery delivery.
  • the methods may be used to increase the expression of a protein of interest (e.g., an antibody, AADC, APOE2, and/or Frataxin).
  • the methods may be used to decrease the amount of expression of a gene of interest (e.g., SOD1, HTT, APOE, and/or MAPT).
  • kits for altering the level of a protein or gene of interest in a subject by administration of the AAV particles described herein are provided.
  • administration may be by intravenous or intracarotid artery delivery.
  • the methods may be used to increase the expression of a protein of interest (e.g., an antibody, AADC, APOE2, Frataxin, ATP2A2, and/or S 100A1).
  • the methods may be used to decrease the amount of expression of a gene of interest (e.g., SOD1, HTT, APOE, and/or MAPT).
  • a gene of interest e.g., SOD1, HTT, APOE, and/or MAPT.
  • compositions of the AAV particles described herein are methods for treating heart failure in a subject in need thereof by administering compositions of the AAV particles described herein.
  • the delivery may be by any method known in the art, such as, but not limited to, intravenous administration or intracarotid artery delivery.
  • FIG. 1 is a schematic of a viral genome of the invention.
  • Adeno-associated viruses AAVs
  • AAV particles AAV particles
  • Viruses of the Parvoviridae family are small non-enveloped icosahedral capsid viruses characterized by a single stranded DNA genome. Parvoviridae family viruses consist of two subfamilies: Parvovirinae, which infect vertebrates, and Densovirinae, which infect invertebrates. Due to its relatively simple structure, easily manipulated using standard molecular biology techniques, this virus family is useful as a biological tool.
  • the genome of the virus may be modified to contain a minimum of components for the assembly of a functional recombinant virus, or viral particle, which is loaded with or engineered to express or deliver a desired payload, which may be delivered to a target cell, tissue, organ, or organism.
  • parvoviruses and other members of the Parvoviridae family are generally described in Kenneth I. Bems, "Parvoviridae: The Viruses and Their Replication," Chapter 69 in FIELDS VIROLOGY (3d Ed. 1996), the contents of which are incorporated by reference in their entirety.
  • the Parvoviridae family comprises the Dependovirus genus which includes adeno- associated viruses (AAV) capable of replication in vertebrate hosts including, but not limited to, human, primate, bovine, canine, equine, and ovine species.
  • AAV adeno- associated viruses
  • the AAV viral genome is a linear, single-stranded DNA (ssDNA) molecule approximately 5,000 nucleotides (nt) in length.
  • the AAV viral genome can comprise a payload region and at least one inverted terminal repeat (ITR) or ITR region. ITRs traditionally flank the coding nucleotide sequences for the non-structural proteins (encoded by Rep genes) and the structural proteins (encoded by capsid genes or Cap genes). While not wishing to be bound by theory, an AAV viral genome typically comprises two ITR sequences.
  • the AAV vector genome comprises a characteristic T-shaped hairpin structure defined by the self-complementary terminal 145 nt of the 5' and 3' ends of the ssDNA which form an energetically stable double stranded region.
  • the double stranded hairpin structures comprise multiple functions including, but not limited to, acting as an origin for DNA replication by functioning as primers for the endogenous DNA polymerase complex of the host viral replication cell.
  • AAV particles may comprise the viral genome, in whole or in part, of any naturally occurring and/or recombinant AAV serotype nucleotide sequence or variant.
  • AAV variants may have sequences of significant homology at the nucleic acid (genome or capsid) and amino acid levels (capsids), to produce constructs which are generally physical and functional equivalents, replicate by similar mechanisms, and assemble by similar mechanisms. Chiorini et al, J. Vir. 71 : 6823-33(1997); Srivastava et al, J. Vir. 45:555-64 (1983); Chiorini et al, J. Vir. 73: 1309-1319 (1999);
  • AAV particles of the present invention are recombinant AAV viral vectors which are replication defective, lacking sequences encoding functional Rep and Cap proteins within their viral genome. These defective AAV particles may lack most or all parental coding sequences and essentially carry only one or two AAV ITR sequences and the nucleic acid of interest for delivery to a cell, a tissue, an organ or an organism.
  • the viral genome of the AAV particles of the present invention comprise at least one control element which provides for the replication, transcription and translation of a coding sequence encoded therein. Not all of the control elements need always be present as long as the coding sequence is capable of being replicated, transcribed and/or translated in an appropriate host cell.
  • expression control elements include sequences for transcription initiation and/or termination, promoter and/or enhancer sequences, efficient RNA processing signals such as splicing and polyadenylation signals, sequences that stabilize cytoplasmic mRNA, sequences that enhance translation efficacy (e.g., Kozak consensus sequence), sequences that enhance protein stability, and/or sequences that enhance protein processing and/or secretion.
  • AAV particles for use in therapeutics and/or diagnostics comprise a virus that has been distilled or reduced to the minimum components necessary for transduction of a nucleic acid payload or cargo of interest.
  • AAV particles are engineered as vehicles for specific delivery while lacking the deleterious replication and/or integration features found in wild-type viruses.
  • AAV particles of the present invention may be produced recombinantly and may be based on adeno-associated virus (AAV) parent or reference sequences.
  • AAV adeno-associated virus
  • a "vector” is any molecule or moiety which transports, transduces or otherwise acts as a carrier of a heterologous molecule such as the nucleic acids described herein.
  • the present invention also provides for self-complementary AAV (scAAVs) viral genomes.
  • scAAV vector genomes contain DNA strands which anneal together to form double stranded DNA. By skipping second strand synthesis, scAAVs allow for rapid expression in the cell.
  • the AAV particle of the present invention is an scAAV.
  • the AAV particle of the present invention is an ssAAV.
  • AAV particles may be modified to enhance the efficiency of delivery. Such modified AAV particles can be packaged efficiently and be used to successfully infect the target cells at high frequency and with minimal toxicity.
  • the capsids of the AAV particles are engineered according to the methods described in US Patent
  • the AAV particles comprising a payload region encoding the polypeptides of the invention may be introduced into mammalian cells.
  • AAV particles of the present invention may comprise or be derived from any natural or recombinant AAV serotype.
  • the AAV particles may utilize or be based on a serotype or include a peptide selected from any of the following VOY101, VOY201, AAVPHP.B (PHP.B), AAVPHP.A (PHP.A), AAVG2B-26, AAVG2B- 13, AAVTHl.1-32, AAVTHl .1-35, AAVPHP.B2 (PHP.B2), AAVPHP.B3 (PHP.B3), AAVPHP.N/PHP.B-DGT, AAVPHP.B-EST, AAVPHP.B-GGT, AAVPHP.B-ATP,
  • AAVPHP.B- ATT-T AAVPHP.B-DGT-T
  • AAVPHP.B-GGT-T AAVPHP.B-SGS
  • AAVPHP.B-AQP AAVPHP.B-QQP
  • AAVPHP.B-SNP(3) AAVPHP.B-SNP
  • AAVPHP.B- QGT AAVPHP.B-NQT
  • AAVPHP.B-EGS AAVPHP.B-SGN
  • AAVPHP.B-EGT AAVPHP.B-EGT
  • AAVPHP.B-DST AAVPHP.B-DST
  • AAVPHP.B-STP AAVPHP.B-PQP
  • AAVPHP.B- SQP AAVPHP.B-QLP
  • AAVPHP.B-TMP AAVPHP.B-TTP
  • AAVPHP.S/G2A12 AAVPHP.B-DST
  • AAVPHP.B-PQP AAVPHP.B- SQP
  • AAVPHP.B-QLP AAVPHP.B-TMP
  • AAVPHP.B-TTP AAVPHP.S/G2A12
  • AAVG2A15/G2A3 (G2A3), AAVG2B4 (G2B4), AAVG2B5 (G2B5), PHP.S, AAV1, AAV2, AAV2G9, AAV3, AAV3a, AAV3b, AAV3-3, AAV4, AAV4-4, AAV5, AAV 6, AAV6.1, AAV6.2, AAV6.1.2, AAV7, AAV7.2, AAV 8, AAV9, AAV9.11, AAV9.13, AAV9.16, AAV9.24, AAV9.45, AAV9.47, AAV9.61, AAV9.68, AAV9.84, AAV9.9, AAV10, AAV11, AAV 12, AAV16.3, AAV24.1, AAV27.3, AAV42.12, AAV42-lb, AAV42-2, AAV42-3a, AAV42-3b, AAV42-4, AAV42-5a, AAV42-5b, AAV42-6b, AAV42- 8, AAV42
  • AAVhu.2 AAVhu.3, AAVhu.4, AAVhu.5, AAVhu.6, AAVhu.7, AAVhu.9, AAVhu.lO, AAVhu.l l, AAVhu.13, AAVhu.15, AAVhu.16, AAVhu.17, AAVhu.18, AAVhu.20, AAVhu.21, AAVhu.22, AAVhu.23.2, AAVhu.24, AAVhu.25, AAVhu.27, AAVhu.28, AAVhu.29, AAVhu.29R, AAVhu.31, AAVhu.32, AAVhu.34, AAVhu.35, AAVhu.37, AAVhu.39, AAVhu.40, AAVhu.41, AAVhu.42, AAVhu.43, AAVhu.44, AAVhu.44R
  • AAVhu.44R3 AAVhu.45, AAVhu.46, AAVhu.47, AAVhu.48, AAVhu.48Rl,
  • AAV-PAEC AAV- LK01, AAV-LK02, AAV-LK03, AAV-LK04, AAV-LK05, AAV-LK06, AAV-LK07, AAV- LK08, AAV-LK09, AAV-LK10, AAV-LK11, AAV-LK12, AAV-LK13, AAV-LK14, AAV- LK15, AAV-LK16, AAV-LK17, AAV-LK18, AAV-LK19, AAV-PAEC2, AAV- LK01, AAV-LK02, AAV-LK03, AAV-LK04, AAV-LK05, AAV-LK06, AAV-LK07, AAV- LK08, AAV-LK09, AAV-LK10, AAV-LK11, AAV-LK12, AAV-LK13, AAV-LK14, AAV- LK15, AAV-LK16, AAV-LK17, AAV-LK18, AAV-LK19, AAV-PAEC2, AAV
  • AAVF14/HSC14 AAVF15/HSC15, AAVF16/HSC16, AAVF17/HSC17, AAVF2/HSC2, AAVF3/HSC3, AAVF4/HSC4, AAVF5/HSC5, AAVF6/HSC6, AAVF7/HSC7,
  • AAVF8/HSC8 and/or AAVF9/HSC9 and variants thereof.
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent Application Publication No. US20030138772, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV1 (SEQ ID NO: 6 and 64 of US20030138772), AAV2 (SEQ ID NO: 7 and 70 of US20030138772), AAV3 (SEQ ID NO: 8 and 71 of US20030138772), AAV 4 (SEQ ID NO: 63 of US20030138772), AAV5 (SEQ ID NO: 114 of US20030138772), AAV6 (SEQ ID NO: 65 of US20030138772), AAV7 (SEQ ID NO: 1-3 of US20030138772), AAV 8 (SEQ ID NO: 4 and 95 of
  • AAV29.5/bb.2 (US20030138772 SEQ ID NO: 13), AAV1.3 (US20030138772 SEQ ID NO: 14), AAV13.3 (US20030138772 SEQ ID NO: 15), AAV24.1 (US20030138772 SEQ ID NO: 16), AAV27.3 (US20030138772 SEQ ID NO: 17), AAV7.2 (US20030138772 SEQ ID NO: 18), AAVC1 (US20030138772 SEQ ID NO: 19), AAVC3 (US20030138772 SEQ ID NO: 20), AAVC5 (US20030138772 SEQ ID NO: 21), AAVF1 (US20030138772 SEQ ID NO: 22), AAVF3 (US20030138772 SEQ ID NO: 23), AAVF5 (US20030138772 SEQ ID NO: 24), AAVH6 (US20030138772 SEQ ID NO: 25), AAVH2 (US20030138772 SEQ ID NO: 26
  • AAV223.5 (US20030138772 SEQ ID NO: 51), AAV223.6 (US20030138772 SEQ ID NO: 52), AAV223.7 (US20030138772 SEQ ID NO: 53), AAV A3.4 (US20030138772 SEQ ID NO: 54), AAV A3.5 (US20030138772 SEQ ID NO: 55), AAV A3.7 (US20030138772 SEQ ID NO: 56), AAV A3.3 (US20030138772 SEQ ID NO: 57), AAV42.12 (US20030138772 SEQ ID NO: 58), AAV44.2 (US20030138772 SEQ ID NO: 59), AAV42-2 (US20030138772 SEQ ID NO: 9), or variants thereof.
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent Application Publication No. US20150159173, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV2 (SEQ ID NO: 7 and 23 of US20150159173), rh20 (SEQ ID NO: 1 of US20150159173), rh32/33 (SEQ ID NO: 2 of US20150159173), rh39 (SEQ ID NO: 3, 20 and 36 of US20150159173), rh46 (SEQ ID NO: 4 and 22 of US20150159173), rh73 (SEQ ID NO: 5 of US20150159173), rh74 (SEQ ID NO: 6 of US20150159173), AAV6.1 (SEQ ID NO: 29 of US20150159173), rh.8 (SEQ ID NO: 41 of US20150159173), rh.48.1 (SEQ ID NO:
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent No. US 7198951, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV9 (SEQ ID NO: 1-3 of US 7198951), AAV2 (SEQ ID NO: 4 of US 7198951), AAV1 (SEQ ID NO: 5 of US 7198951), AAV3 (SEQ ID NO: 6 of US 7198951), and AAV 8 (SEQ ID NO: 7 of US7198951).
  • AAV9 SEQ ID NO: 1-3 of US 7198951
  • AAV2 SEQ ID NO: 4 of US 7198951
  • AAV1 SEQ ID NO: 5 of US 7198951
  • AAV3 SEQ ID NO: 6 of US 7198951
  • AAV 8 SEQ ID NO: 7 of US7198951.
  • the AAV serotype may be, or have, a mutation in the AAV9 sequence as described by N Pulichla et al. (Molecular Therapy 19(6): 1070-1078 (2011), the contents of which are herein incorporated by reference in their entirety), such as but not limited to, AAV9.9, AAV9.11, AAV9.13, AAV9.16, AAV9.24, AAV9.45, AAV9.47, AAV9.61, AAV9.68, AAV9.84.
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent No. US 6156303, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV3B (SEQ ID NO: 1 and 10 of US 6156303), AAV6 (SEQ ID NO: 2, 7 and 11 of US 6156303), AAV2 (SEQ ID NO: 3 and 8 of US 6156303), AAV3A (SEQ ID NO: 4 and 9, of US 6156303), or derivatives thereof.
  • AAV3B SEQ ID NO: 1 and 10 of US 6156303
  • AAV6 SEQ ID NO: 2, 7 and 11 of US 6156303
  • AAV2 SEQ ID NO: 3 and 8 of US 6156303
  • AAV3A SEQ ID NO: 4 and 9, of US 6156303
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent Application Publication No. US20140359799, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV8 (SEQ ID NO: 1 of US20140359799), AAVDJ (SEQ ID NO: 2 and 3 of US20140359799), or variants thereof.
  • the serotype may be AAVDJ or a variant thereof, such as AAVDJ8 (or AAV-DJ8), as described by Grimm et al. (Journal of Virology 82(12): 5887- 5911 (2008), herein incorporated by reference in its entirety).
  • the amino acid sequence of AAVDJ8 may comprise two or more mutations in order to remove the heparin binding domain (HBD).
  • HBD heparin binding domain
  • 7,588,772 may comprise two mutations: (1) R587Q where arginine (R; Arg) at amino acid 587 is changed to glutamine (Q; Gin) and (2) R590T where arginine (R; Arg) at amino acid 590 is changed to threonine (T; Thr).
  • K406R where lysine (K; Lys) at amino acid 406 is changed to arginine (R; Arg)
  • R587Q where arginine (R; Arg) at amino acid 587 is changed to glutamine (Q; Gin)
  • R590T where arginine (R; Arg) at amino acid 590 is changed to threonine (T; Thr).
  • the AAV serotype may be, or have, a sequence of AAV4 as described in International Publication No. WO1998011244, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to AAV4 (SEQ ID NO: 1- 20 of WO1998011244).
  • the AAV serotype may be, or have, a mutation in the AAV2 sequence to generate AAV2G9 as described in International Publication No.
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2005033321, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to AAV3-3 (SEQ ID NO: 217 of WO2005033321), AAV1 (SEQ ID NO: 219 and 202 of WO2005033321),
  • AAV106.1/hu.37 (SEQ ID No: 10 of WO2005033321), AAV114.3/hu.40 (SEQ ID No: 11 of WO2005033321), AAV127.2/hu.41 (SEQ ID NO:6 and 8 of WO2005033321),
  • AAV128.3/hu.44 (SEQ ID No: 81 of WO2005033321), AAV130.4/hu.48 (SEQ ID NO: 78 of WO2005033321), AAV145.1/hu.53 (SEQ ID No: 176 and 177 of WO2005033321), AAV145.6/hu.56 (SEQ ID NO: 168 and 192 of WO2005033321), AAV16.12/hu. l l (SEQ ID NO: 153 and 57 of WO2005033321), AAV16.8/hu. lO (SEQ ID NO: 156 and 56 of
  • WO2005033321 AAV161.10/hu.60 (SEQ ID No: 170 of WO2005033321),
  • AAV161.6/hu.61 (SEQ ID No: 174 of WO2005033321), AAVl-7/rh.48 (SEQ ID NO: 32 of WO2005033321), AAVl-8/rh.49 (SEQ ID NOs: 103 and 25 of WO2005033321), AAV2 (SEQ ID NO: 211 and 221 of WO2005033321), AAV2-15/rh.62 (SEQ ID No: 33 and 114 of WO2005033321), AAV2-3/rh.61 (SEQ ID NO: 21 of WO2005033321), AAV2-4/rh.50 (SEQ ID No: 23 and 108 of WO2005033321), AAV2-5/rh.51 (SEQ ID NO: 104 and 22 of
  • WO2005033321 AAV3.1/hu.6 (SEQ ID NO: 5 and 84 of WO2005033321), AAV3.1/hu.9 (SEQ ID NO: 155 and 58 of WO2005033321), AAV3-l l/rh.53 (SEQ ID NO: 186 and 176 of WO2005033321), AAV3-3 (SEQ ID NO: 200 of WO2005033321), AAV33.12/hu. l7 (SEQ ID NO:4 ofWO2005033321), AAV33.4/hu. l5 (SEQ ID No: 50 of WO2005033321), AAV33.8/hu.
  • AAV3.1/hu.6 SEQ ID NO: 5 and 84 of WO2005033321
  • AAV3.1/hu.9 SEQ ID NO: 155 and 58 of WO2005033321
  • AAV3-l l/rh.53 SEQ ID NO: 186 and 176 of WO2005033321
  • l6 (SEQ ID No: 51 of WO2005033321), AAV3-9/rh.52 (SEQ ID NO: 96 and 18 of WO2005033321), AAV4-19/rh.55 (SEQ ID NO: 117 of WO2005033321), AAV4-4 (SEQ ID NO: 201 and 218 of WO2005033321), AAV4-9/rh.54 (SEQ ID NO: 116 of WO2005033321), AAV5 (SEQ ID NO: 199 and 216 of WO2005033321), AAV52. l/hu.20 (SEQ ID NO: 63 of WO2005033321), AAV52/hu.
  • WO2005033321 SEQ ID NO: 59
  • AAVhu.13 SEQ ID NO: 129 of WO2005033321
  • AAVhu. l4/AAV9 SEQ ID NO: 123 and 3 of WO2005033321
  • AAVhu.15 SEQ ID NO: 147 of WO2005033321
  • AAVhu.16 SEQ ID NO: 148 of WO2005033321
  • AAVhu.17 SEQ ID NO: 83 of WO2005033321
  • AAVhu.18 SEQ ID NO: 149 of WO2005033321
  • AAVhu.19 SEQ ID NO: 133 of WO2005033321
  • AAVhu.2 SEQ ID NO: 143 of
  • WO2005033321 AAVhu.20 (SEQ ID NO: 134 of WO2005033321), AAVhu.21 (SEQ ID NO: 135 of WO2005033321), AAVhu.22 (SEQ ID NO: 138 of WO2005033321),
  • AAVhu.23.2 (SEQ ID NO: 137 of WO2005033321), AAVhu.24 (SEQ ID NO: 136 of WO2005033321), AAVhu.25 (SEQ ID NO: 146 of WO2005033321), AAVhu.27 (SEQ ID NO: 140 of WO2005033321), AAVhu.29 (SEQ ID NO: 132 of WO2005033321), AAVhu.3 (SEQ ID NO: 145 of WO2005033321), AAVhu.31 (SEQ ID NO: 121 of WO2005033321), AAVhu.32 (SEQ ID NO: 122 of WO2005033321), AAVhu.34 (SEQ ID NO: 125 of
  • WO2005033321 AAVhu.35 (SEQ ID NO: 164 of WO2005033321), AAVhu.37 (SEQ ID NO: 88 of WO2005033321), AAVhu.39 (SEQ ID NO: 102 of WO2005033321), AAVhu.4 (SEQ ID NO: 141 of WO2005033321), AAVhu.40 (SEQ ID NO: 87 of WO2005033321), AAVhu.41 (SEQ ID NO: 91 of WO2005033321), AAVhu.42 (SEQ ID NO: 85 of
  • WO2005033321 AAVhu.43 (SEQ ID NO: 160 of WO2005033321), AAVhu.44 (SEQ ID NO: 144 of WO2005033321), AAVhu.45 (SEQ ID NO: 127 of WO2005033321), AAVhu.46 (SEQ ID NO: 159 of WO2005033321), AAVhu.47 (SEQ ID NO: 128 of WO2005033321), AAVhu.48 (SEQ ID NO: 157 of WO2005033321), AAVhu.49 (SEQ ID NO: 189 of
  • WO2005033321 AAVhu.51 (SEQ ID NO: 190 of WO2005033321), AAVhu.52 (SEQ ID NO: 191 of WO2005033321), AAVhu.53 (SEQ ID NO: 186 of WO2005033321), AAVhu.54 (SEQ ID NO: 188 of WO2005033321), AAVhu.55 (SEQ ID NO: 187 of WO2005033321), AAVhu.56 (SEQ ID NO: 192 of WO2005033321), AAVhu.57 (SEQ ID NO: 193 of WO2005033321), AAVhu.58 (SEQ ID NO: 194 of WO2005033321), AAVhu.6 (SEQ ID NO: 84 of WO2005033321), AAVhu.60 (SEQ ID NO: 184 of WO2005033321), AAVhu.61 (SEQ ID NO: 185 of WO2005033321), AAVhu.63
  • AAVrh.47 (WO2005033321 SEQ ID NO: 38), AAVrh.48 (SEQ ID NO: 115 of
  • WO2005033321 WO2005033321
  • AAVrh.49 SEQ ID NO: 103 of WO2005033321
  • AAVrh.50 SEQ ID NO: 108 of WO2005033321
  • AAVrh.51 SEQ ID NO: 104 of WO2005033321
  • AAVrh.52 SEQ ID NO: 96 of WO2005033321
  • AAVrh.53 SEQ ID NO: 97 of WO2005033321
  • AAVrh.55 WO2005033321 SEQ ID NO: 37
  • AAVrh.56 SEQ ID NO: 152 of
  • WO2005033321 WO2005033321
  • AAVrh.57 SEQ ID NO: 105 of WO2005033321
  • AAVrh.58 SEQ ID NO: 106 of WO2005033321
  • AAVrh.59 WO2005033321 SEQ ID NO: 42
  • AAVrh.60 WO2005033321 SEQ ID NO: 31
  • AAVrh.61 SEQ ID NO: 107 of WO2005033321
  • AAVrh.62 SEQ ID NO: 114 of WO2005033321
  • AAVrh.64 SEQ ID NO: 99 of
  • AAVrh.70 (WO2005033321 SEQ ID NO: 20), AAVrh.72 (WO2005033321 SEQ ID NO: 9), or variants thereof including, but not limited to, AAVcy.2, AAVcy.3, AAVcy.4, AAVcy.5, AAVcy.6, AAVrh.12, AAVrh.17, AAVrh.18, AAVrh.19, AAVrh.21, AAVrh.22, AAVrh.23, AAVrh.24, AAVrh.25, AAVrh.25/42 15, AAVrh.31, AAVrh.32, AAVrh.33, AAVrh.34, AAVrh.35, AAVrh.36, AAVrh.37, AAVrhl4.
  • Non limiting examples of variants include SEQ ID NO: 13, 15, 17, 19, 24, 36, 40, 45, 47, 48, 51-54, 60-62, 64-77, 79, 80, 82, 89, 90, 93-95, 98, 100, 101, , 109-113, 118-120, 124, 126, 131, 139, 142, 151,154, 158, 161, 162, 165-183, 202, 204-212, 215, 219, 224-236, of WO2005033321, the contents of which are herein incorporated by reference in their entirety.
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2015168666, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAVrh8R (SEQ ID NO: 9 of WO2015168666), AAVrh8R A586R mutant (SEQ ID NO: 10 of WO2015168666), AAVrh8R R533A mutant (SEQ ID NO: 11 of WO2015168666), or variants thereof.
  • AAVrh8R SEQ ID NO: 9 of WO2015168666
  • AAVrh8R A586R mutant SEQ ID NO: 10 of WO2015168666
  • AAVrh8R R533A mutant SEQ ID NO: 11 of WO2015168666
  • the AAV serotype may be, or have, a sequence as described in United States Patent No. US9233131, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAVhEl .
  • AAVhEr3.1 (SEQ ID NO:59 of US9233131), AAV2.5T (SEQ ID NO:42 of US9233131), or variants thereof.
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent Application Publication No. US20150376607, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV-PAEC (SEQ ID NO: l of US20150376607), AAV-LK01 (SEQ ID NO:2 of US20150376607), AAV-LK02 (SEQ ID NO:3 of US20150376607), AAV-LK03 (SEQ ID NO:4 of US20150376607), AAV- LK04 (SEQ ID NO:5 of US20150376607), AAV-LK05 (SEQ ID NO:6 of US20150376607), AAV-LK06 (SEQ ID NO:7 of US20150376607), AAV-LK07 (SEQ ID NO:8 of
  • AAV-LK08 SEQ ID NO:9 of US20150376607
  • AAV-LK09 SEQ ID NO: 10 of US20150376607
  • AAV-LK10 SEQ ID NO: 11 of US20150376607
  • AAV-LK11 SEQ ID NO: 12 of US20150376607
  • AAV-LK12 SEQ ID NO: 13 of US20150376607
  • AAV-LK13 SEQ ID NO: 14 of US20150376607
  • AAV-LK14 SEQ ID NO: 15 of
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent No. US9163261, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV-2-pre-miRNA-101 (SEQ ID NO: 1
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent Application Publication No. US20150376240, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV-8h (SEQ ID NO: 6 of US20150376240), AAV-8b (SEQ ID NO: 5 of US20150376240), AAV-h (SEQ ID NO: 2 of US20150376240), AAV-b (SEQ ID NO: 1 of US20150376240), or variants thereof.
  • AAV-8h SEQ ID NO: 6 of US20150376240
  • AAV-8b SEQ ID NO: 5 of US20150376240
  • AAV-h SEQ ID NO: 2 of US20150376240
  • AAV-b SEQ ID NO: 1 of US20150376240
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent Application Publication No. US20160017295, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV SM 10-2 (SEQ ID NO: 22 of US20160017295), AAV Shuffle 100-1 (SEQ ID NO: 23 of US20160017295), AAV Shuffle 100-3 (SEQ ID NO: 24 of US20160017295), AAV Shuffle 100-7 (SEQ ID NO: 25 of US20160017295), AAV Shuffle 10-2 (SEQ ID NO: 34 of US20160017295), AAV Shuffle 10-6 (SEQ ID NO: 35 of US20160017295), AAV Shuffle 10-8 (SEQ ID NO: 36 of US20160017295), AAV Shuffle 100-2 (SEQ ID NO: 37 of US20160017295), AAV SM 10-1 (SEQ ID NO: 38 of US20160017295), AAV SM 10-8 (SEQEQ ID NO:
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent Application Publication No. US20150238550, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, BNP61 AAV (SEQ ID NO: 1 of US20150238550), BNP62 AAV (SEQ ID NO: 3 of US20150238550), BNP63 AAV (SEQ ID NO: 4 of US20150238550), or variants thereof.
  • the AAV serotype may be or may have a sequence as described in United States Patent Publication No. US20150315612, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAVrh.50 (SEQ ID NO: 108 of US20150315612), AAVrh.43 (SEQ ID NO: 163 of US20150315612), AAVrh.62 (SEQ ID NO: 114 of US20150315612), AAVrh.48 (SEQ ID NO: 115 of
  • US20150315612 AAV54.5/hu.23 (SEQ ID No: 60 of US20150315612), AAV54.2/hu.22 (SEQ ID No: 67 of US20150315612), AAV54.7/hu.24 (SEQ ID No: 66 of US20150315612), AAV54.1/hu.21 (SEQ ID No: 65 of US20150315612), AAV54.4R/hu.27 (SEQ ID No: 64 of US20150315612), AAV46.2/hu.28 (SEQ ID No: 68 of US20150315612), AAV46.6/hu.29 (SEQ ID No: 69 of US20150315612), AAV128.1/hu.43 (SEQ ID No: 80 of
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2015121501, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, true type AAV (ttAAV) (SEQ ID NO: 2 of WO2015121501), "UPenn AAV10” (SEQ ID NO: 8 of WO2015121501), “Japanese AAV10” (SEQ ID NO: 9 of WO2015121501), or variants thereof.
  • true type AAV ttAAV
  • UPenn AAV10 SEQ ID NO: 8 of WO2015121501
  • Japanese AAV10 Japanese AAV10
  • AAV capsid serotype selection or use may be from a variety of species.
  • the AAV may be an avian AAV (AAAV).
  • the AAAV serotype may be, or have, a sequence as described in U.S. Patent No. US 9238800, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAAV (SEQ ID NO: 1, 2, 4, 6, 8, 10, 12, and 14 of US 9,238,800), or variants thereof.
  • the AAV may be a bovine AAV (BAAV).
  • BAAV serotype may be, or have, a sequence as described in U.S. Patent No. US 9,193,769, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, BAAV (SEQ ID NO: 1 and 6 of US 9,193,769), or variants thereof.
  • BAAV serotype may be or have a sequence as described in United States Patent No. US7,427,396, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, BAAV (SEQ ID NO: 5 and 6 of US7,427,396), or variants thereof.
  • the AAV may be a caprine AAV.
  • the caprine AAV serotype may be, or have, a sequence as described in United States Patent No. US7,427,396, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, caprine AAV (SEQ ID NO: 3 of US7,427,396), or variants thereof.
  • the AAV may be engineered as a hybrid AAV from two or more parental serotypes.
  • the AAV may be AAV2G9 which comprises sequences from AAV2 and AAV9.
  • the AAV2G9 AAV serotype may be, or have, a sequence as described in U.S. Patent Application Publication No. US20160017005, the contents of which are herein incorporated by reference in its entirety.
  • the AAV may be a serotype generated by the AAV9 capsid library with mutations in amino acids 390-627 (VPl numbering) as described by Pulichla et al. (Molecular Therapy 19(6): 1070-1078 (2011); the contents of which are herein
  • the serotype and corresponding nucleotide and amino acid substitutions may be, but is not limited to, AAV9.1 (G1594C; D532H), AAV6.2 (T1418A and T1436X; V473D and I479K), AAV9.3 (T1238A; F413Y), AAV9.4 (T1250C and A1617T; F417S), AAV9.5 (A1235G, A1314T, A1642G, C1760T; Q412R, T548A, A587V), AAV9.6 (T1231A; F411I), AAV9.9 (G1203A, G1785T; W595C), AAV9.10 (A1500G, T1676C; M559T), AAV9.11 (A1425T, A1702C, A1769T; T568P, Q590L), AAV9.13 (A1369C, A1720T; N457H, T574
  • AAV9.58 C1475T, C1579A; T492I, H527N
  • AAV.59 T1336C; Y446H
  • AAV9.61 A1493T; N498I
  • AAV9.64 C1531A, A1617T; L511I
  • AAV9.65 C1335T, T1530C, C1568A; A523D
  • AAV9.68 C1510A; P504T
  • AAV9.80 G1441A,;G481R
  • AAV9.83 C1402A, A1500T; P468T, E500D
  • AAV9.87 T1464C, T1468C; S490P
  • AAV9.90 A1196T; Y399F
  • AAV9.91 T1316G, A1583T, C1782G, T1806C; L439R, K528I
  • AAV9.93 A1273G, A1421G, A1638C, C1712
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2016049230, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to AAVFl/HSCl (SEQ ID NO: 2 and 20 of WO2016049230), AAVF2/HSC2 (SEQ ID NO: 3 and 21 of
  • WO2016049230 AAVF3/HSC3 (SEQ ID NO: 5 and 22 of WO2016049230),
  • AAVF4/HSC4 (SEQ ID NO: 6 and 23 of WO2016049230), AAVF5/HSC5 (SEQ ID NO: 11 and 25 of WO2016049230), AAVF6/HSC6 (SEQ ID NO: 7 and 24 of WO2016049230), AAVF7/HSC7 (SEQ ID NO: 8 and 27 of WO2016049230), AAVF8/HSC8 (SEQ ID NO: 9 and 28 of WO2016049230), AAVF9/HSC9 (SEQ ID NO: 10 and 29 of WO2016049230), AAVF11/HSC11 (SEQ ID NO: 4 and 26 of WO2016049230), AAVF12/HSC12 (SEQ ID NO: 12 and 30 of WO2016049230), AAVF13/HSC13 (SEQ ID NO: 14 and 31 of
  • WO2016049230 AAVF14/HSC14 (SEQ ID NO: 15 and 32 of WO2016049230),
  • AAVF15/HSC15 SEQ ID NO: 16 and 33 of WO2016049230
  • AAVF16/HSC16 SEQ ID NO: 17 and 34 of WO2016049230
  • AAVF17/HSC17 SEQ ID NO: 13 and 35 of
  • the AAV serotype may be, or have, a sequence as described in U.S. Patent No. US 8734809, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to, AAV CBr-El (SEQ ID NO: 13 and 87 of US8734809), AAV CBr-E2 (SEQ ID NO: 14 and 88 of US8734809), AAV CBr-E3 (SEQ ID NO: 15 and 89 of US8734809), AAV CBr-E4 (SEQ ID NO: 16 and 90 of US8734809), AAV CBr-E5 (SEQ ID NO: 17 and 91 of US8734809), AAV CBr-e5 (SEQ ID NO: 18 and 92 of US8734809), AAV CBr-E6 (SEQ ID NO: 19 and 93 of US8734809), AAV CBr-E7 (SEQ ID NO: 20 and 94 of
  • the AAV serotype may be, or have, a sequence as described in International Publication No. WO2016065001, the contents of which are herein incorporated by reference in their entirety, such as, but not limited to AAV CHt-P2 (SEQ ID NO: 1 and 51 of WO2016065001), AAV CHt-P5 (SEQ ID NO: 2 and 52 of
  • WO2016065001 AAV CHt-P9 (SEQ ID NO: 3 and 53 of WO2016065001), AAV CBr-7.1 (SEQ ID NO: 4 and 54 of WO2016065001), AAV CBr-7.2 (SEQ ID NO: 5 and 55 of WO2016065001), AAV CBr-7.3 (SEQ ID NO: 6 and 56 of WO2016065001), AAV CBr-7.4 (SEQ ID NO: 7 and 57 of WO2016065001), AAV CBr-7.5 (SEQ ID NO: 8 and 58 of WO2016065001), AAV CBr-7.7 (SEQ ID NO: 9 and 59 of WO2016065001), AAV CBr-7.8 (SEQ ID NO: 10 and 60 of WO2016065001), AAV CBr-7.10 (SEQ ID NO: 11 and 61 of WO2016065001), AAV CKd-N3 (SEQ ID NO: 12 and 62 of WO201606500
  • the AAV may be a serotype selected from any of those found in Table 1.
  • the AAV may comprise a sequence, fragment or variant thereof, of the sequences in Table 1.
  • the AAV may be encoded by a sequence, fragment or variant as described in Table 1.
  • AAV9 (AAVhu.14) US7906111 SEQ ID NO: 3; WO2015038958 SEQ ID NO: 3;
  • AAV9 (AAVhu.14) US7906111 SEQ ID NO: 123; WO2015038958 SEQ ID NO: 123;
  • AAV29.3 (AAVbb.l) 146 US20030138772 SEQ ID NO 11
  • AAVcy.2 (AAV13.3) 149 US20030138772 SEQ ID NO 15
  • AAVcy.3 (AAV24.1) 151 US20030138772 SEQ ID NO 16
  • AAVcy.4 (AAV27.3) 153 US20030138772 SEQ ID NO 17
  • AAVcy.5 (AAV7.2) 156 US20030138772 SEQ ID NO 18
  • AAVcy.6 (AAV16.3) 158 US20030138772 SEQ ID NO 10
  • AAVhErl.23 (AAVhEr2.29) 176 US9233131 SEQIDNO 53
  • AAVhu.ll (AAV16.12) 192 US20150315612 SEQ ID NO 57
  • AAVhu.ll (AAV16.12) 193 US20150315612 SEQ ID NO 153
  • AAVhu.15 (AAV33.4) 203 US20150315612 SEQ ID NO: 50
  • AAVhu.16 (AAV33.8) 206 US20150315612 SEQ ID NO: 51
  • AAVhu.37 (AAV106.1) US20150315612 SEQ ID NO: 10, US20150159173 SEQ ID NO: 10
  • AAVhu.39 (AAVLG-9) 261 US20150315612 SEQ ID NO 24
  • AAVhu.40 (AAV114.3) 265 US20150315612 SEQ ID No: 11
  • AAVhu.41 (AAV127.2) 267 US20150315612 SEQ ID NO 6
  • AAVhu.42 (AAV127.5) 269 US20150315612 SEQ ID NO 8
  • AAVhu.43 (AAV128.1) 272 US20150315612 SEQ ID NO 80
  • AAVhu.44 (AAV128.3) 274 US20150315612 SEQ ID NO: 81
  • AAVhu.48 (AAV130.4) 287 US20150315612 SEQ ID NO 78
  • AAVhu.53 (AAV145.1) 301 US20150315612 SEQ ID NO 176
  • AAVhu.54 302 US20150315612 SEQ ID NO: 188
  • AAVhu.54 (AAV145.5) 303 US20150315612 SEQ ID No: 177
  • AAVhu.56 (AAV145.6) 306 US20150315612 SEQ ID NO: 168
  • AAVhu.56 (AAV145.6) 307 US20150315612 SEQ ID NO: 192
  • AAVhu.6 (AAV3.1) 313 US20150315612 SEQ ID NO: 5
  • AAVhu.6 (AAV3.1) 314 US20150315612 SEQ ID NO: 84
  • AAVhu.60 (AAV161.10) 316 US20150315612 SEQ ID NO: 170
  • AAVhu.61 (AAV161.6) 318 US20150315612 SEQ ID NO: 174
  • AAVhu.9 (AAV3.1) 333 US20150315612 SEQ ID NO: 58
  • AAVhu.9 (AAV3.1) 334 US20150315612 SEQ ID NO: 155
  • AAV-LK04 342 US20150376607 SEQ ID NO: 32
  • AAVrh.10 (AAV44.2) 400 US20030138772 SEQ ID NO 81
  • AAVrh.l2 (AAV42.1b) 402 US20030138772 SEQ ID NO 30
  • AAVrh.l4 (AAV42.3a) 408 US20030138772 SEQ ID NO 32
  • AAVrh.l7 (AAV42.5a) 410 US20030138772 SEQ ID NO 34
  • AAVrh.l8 (AAV42.5b) 412 US20030138772 SEQ ID NO 29
  • AAVrh.l9 (AAV42.6b) 414 US20030138772 SEQ ID NO 38
  • AAVrh.21 (AAV42.10) 419 US20030138772 SEQ ID NO 35
  • AAVrh.22 (AAV42.11) 421 US20030138772 SEQ ID NO 37
  • AAVrh.23 (AAV42.12) 423 US20030138772 SEQ ID NO 58
  • AAVrh.24 (AAV42.13) 425 US20030138772 SEQ ID NO 31
  • AAVrh.25 (AAV42.15) 427 US20030138772 SEQ ID NO 28
  • AAVrh.31 (AAV223.1) 429 US20030138772 SEQ ID NO: 48
  • AAVrh.32 (AAVCl) 431 US20030138772 SEQ ID NO: 19
  • AAVrh.53 (AAV3-11) 474 US20150315612 SEQ ID NO 17
  • AAVrh.53 (AAV3-11) 475 US20150315612 SEQ ID NO 186
  • AAVrh.55 (AAV4-19) 479 US20150315612 SEQ ID NO 117
  • AAVrh.61 (AAV2-3) 492 US20150315612 SEQ ID NO: 21
  • AAVrh.62 (AAV2-15) 493 US20150315612 SEQ ID No: 33
  • AAVrh.62 (AAV2-15) 494 US20150315612 SEQ ID NO: 114
  • BAAV bovine AAV 520 US9193769 SEQ ID NO 8
  • BAAV bovine AAV 521 US9193769 SEQ ID NO 10
  • BAAV bovine AAV 522 US9193769 SEQ ID NO 4
  • BAAV bovine AAV 523 US9193769 SEQ ID NO 2
  • BAAV bovine AAV 524 US9193769 SEQ ID NO 6
  • BAAV bovine AAV 525 US9193769 SEQ ID NO 1
  • BAAV bovine AAV 526 US9193769 SEQ ID NO 5
  • BAAV bovine AAV 529 US7427396 SEQ ID NO 5
  • BAAV bovine AAV 530 US7427396 SEQ ID NO 6
  • BAAV bovine AAV 531 US9193769 SEQ ID NO 7
  • BAAV bovine AAV 532 US9193769 SEQ ID NO 9
  • AAAV (Avian AAV) 540 US9238800 SEQ ID NO 12
  • AAAV (Avian AAV) 541 US9238800 SEQ ID NO 2
  • AAAV (Avian AAV) 542 US9238800 SEQ ID NO 6
  • AAAV (Avian AAV) 543 US9238800 SEQ ID NO 4
  • AAAV (Avian AAV) 544 US9238800 SEQ ID NO 8
  • AAAV (Avian AAV) 545 US9238800 SEQ ID NO 14
  • AAAV (Avian AAV) 546 US9238800 SEQ ID NO 10
  • AAAV (Avian AAV) 547 US9238800 SEQ ID NO 15
  • AAAV (Avian AAV) 548 US9238800 SEQ ID NO 5
  • AAAV (Avian AAV) 549 US9238800 SEQ ID NO 9
  • AAAV (Avian AAV) 550 US9238800 SEQ ID NO 3
  • AAAV (Avian AAV) 551 US9238800 SEQ ID NO 7
  • AAAV (Avian AAV) 552 US9238800 SEQ ID NO 11
  • AAAV (Avian AAV) 553 US9238800 SEQ ID NO 13
  • AAAV (Avian AAV) 554 US9238800 SEQ ID NO 1
  • rh74 (RHM15-6) 907 US2015023924A1 SEQ ID NO 16 AAV2 (comprising lung
  • AAV2 (comprising lung

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Dermatology (AREA)
  • Mycology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
PCT/US2018/045088 2017-08-03 2018-08-03 COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES Ceased WO2019028306A2 (en)

Priority Applications (11)

Application Number Priority Date Filing Date Title
AU2018311069A AU2018311069A1 (en) 2017-08-03 2018-08-03 Compositions and methods for delivery of AAV
US16/636,094 US11512327B2 (en) 2017-08-03 2018-08-03 Compositions and methods for delivery of AAV
MX2020001187A MX2020001187A (es) 2017-08-03 2018-08-03 Composiciones y métodos para la administración de virus adenoasociados.
JP2020505424A JP7221275B2 (ja) 2017-08-03 2018-08-03 Ａａｖを送達するための組成物および方法
CN201880064712.6A CN111448308A (zh) 2017-08-03 2018-08-03 递送aav的组合物和方法
EP18762419.2A EP3662060A2 (en) 2017-08-03 2018-08-03 Compositions and methods for delivery of aav
CA3071978A CA3071978A1 (en) 2017-08-03 2018-08-03 Compositions and methods for delivery of aav
KR1020207003192A KR102850887B1 (ko) 2017-08-03 2018-08-03 Aav의 전달을 위한 조성물 및 방법
EP20208355.6A EP3808849A1 (en) 2017-08-03 2018-08-03 Compositions and methods for delivery of aav
US17/970,036 US12305189B2 (en) 2017-08-03 2022-10-20 Compositions and methods for delivery of AAV
JP2023013699A JP2023052805A (ja) 2017-08-03 2023-02-01 Ａａｖを送達するための組成物および方法

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201762540776P 2017-08-03 2017-08-03
US62/540,776 2017-08-03
US201762565264P 2017-09-29 2017-09-29
US62/565,264 2017-09-29
US201762572706P 2017-10-16 2017-10-16
US62/572,706 2017-10-16

Related Child Applications (2)

Application Number Title Priority Date Filing Date
US16/636,094 A-371-Of-International US11512327B2 (en) 2017-08-03 2018-08-03 Compositions and methods for delivery of AAV
US17/970,036 Continuation US12305189B2 (en) 2017-08-03 2022-10-20 Compositions and methods for delivery of AAV

Publications (2)

Publication Number Publication Date
WO2019028306A2 true WO2019028306A2 (en) 2019-02-07
WO2019028306A3 WO2019028306A3 (en) 2019-03-07

Family

ID=63442774

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2018/045088 Ceased WO2019028306A2 (en) 2017-08-03 2018-08-03 COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES

Country Status (10)

Country Link
US (2) US11512327B2 (enExample)
EP (2) EP3662060A2 (enExample)
JP (2) JP7221275B2 (enExample)
KR (1) KR102850887B1 (enExample)
CN (1) CN111448308A (enExample)
AU (1) AU2018311069A1 (enExample)
CA (1) CA3071978A1 (enExample)
MX (1) MX2020001187A (enExample)
TW (2) TWI832036B (enExample)
WO (1) WO2019028306A2 (enExample)

Cited By (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10414803B2 (en) 2015-05-11 2019-09-17 Ucl Business Plc Capsid
WO2019222329A1 (en) * 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019246480A1 (en) 2018-06-21 2019-12-26 The Board Of Regents Of The University Of Texas System Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy
WO2020077165A1 (en) * 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020198737A1 (en) * 2019-03-28 2020-10-01 The General Hospital Corporation Engineered adeno-associated (aav) vectors for transgene expression
WO2020223276A1 (en) * 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tauopathy
WO2020223296A1 (en) * 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020242892A1 (en) * 2019-05-24 2020-12-03 The Brigham And Women's Hospital, Inc. Gene therapy for alzheimer's disease
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
WO2021025995A1 (en) * 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2021042073A1 (en) 2019-08-29 2021-03-04 Dorian Therapeutics, Inc. Methods and compositions for modulating cellular aging
WO2021108809A1 (en) * 2019-11-25 2021-06-03 Cornell University Apoe gene therapy
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
WO2021168216A1 (en) 2020-02-21 2021-08-26 The Board Of Regents Of The University Of Texas System Crispr/cas9 correction of mutations in dystrophin exons 43, 45 and 52
WO2021205028A1 (en) 2020-04-09 2021-10-14 University Of Sheffield Gene therapy treatment
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2021214443A1 (en) 2020-04-20 2021-10-28 Synpromics Limited Regulatory nucleic acid sequences
CN113853209A (zh) * 2019-04-29 2021-12-28 宾夕法尼亚州大学信托人 新型aav衣壳和含有其的组合物
WO2022023773A1 (en) * 2020-07-30 2022-02-03 Cambridge Enterprise Limited Composition and method
WO2022049385A1 (en) 2020-09-04 2022-03-10 Asklepios Biopharmaceutical, Inc. Regulatory nucleic acid sequences
CN114667349A (zh) * 2019-09-09 2022-06-24 马萨诸塞眼科耳科诊所 用于调节腺相关病毒(aav)和aav受体(aavr)之间的相互作用以改变aav生物分布的方法和组合物
WO2022136655A1 (en) * 2020-12-23 2022-06-30 Boehringer Ingelheim International Gmbh Viral capsid proteins with specificity to heart tissue cells
WO2022165538A1 (en) * 2021-02-01 2022-08-04 The Regents Of The University Of California Viral delivery of therapeutics to the central nervous system
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022129609A3 (en) * 2020-12-18 2022-09-15 Ac Immune Sa Antibody delivery
WO2022236008A1 (en) * 2021-05-07 2022-11-10 Board Of Regents, The University Of Texas System Methods and compositions for transport, storage, and delivery of adeno-associated viral vector and other molecules
US11518787B2 (en) 2018-07-11 2022-12-06 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of agents across the blood-brain barrier
US11525139B2 (en) 2016-08-23 2022-12-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11542506B2 (en) 2014-11-14 2023-01-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2023283649A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11634742B2 (en) 2020-07-27 2023-04-25 Anjarium Biosciences Ag Compositions of DNA molecules, methods of making therefor, and methods of use thereof
WO2023111580A1 (en) 2021-12-16 2023-06-22 University Of Dundee Targeted degradation of alpha-synuclein
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
WO2023152504A1 (en) 2022-02-10 2023-08-17 University Of Dundee An affinity-directed phosphatase system for targeted protein dephosphorylation
US11759531B2 (en) 2020-02-13 2023-09-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
EP3952923A4 (en) * 2019-04-10 2023-10-18 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US11807867B2 (en) 2020-02-21 2023-11-07 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
EP4022073A4 (en) * 2019-08-29 2023-12-20 The Board Of Trustees Of The Leland Stanford Junior University ADENO-ASSOCIATED VIRAL VECTORS TO CROSS THE HUMAN BLOOD-BRAIN BARRIER
US11859200B2 (en) 2020-05-13 2024-01-02 Voyager Therapeutics, Inc. AAV capsids with increased tropism to brain tissue
WO2024011112A1 (en) * 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP3999120A4 (en) * 2019-07-04 2024-02-21 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
EP4085144A4 (en) * 2019-12-19 2024-03-27 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF FRIEDREICH’S ATAXIA
WO2024079292A1 (en) 2022-10-14 2024-04-18 University Of Sheffield Gene therapy treatment
US11981705B2 (en) 2020-01-10 2024-05-14 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
US11993790B2 (en) 2017-10-03 2024-05-28 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US12049626B2 (en) 2017-10-03 2024-07-30 Prevail Therapeutics, Inc. Gene therapy for neurodegenerative disorders
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
US12116385B2 (en) 2021-10-08 2024-10-15 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
EP4179098A4 (en) * 2020-07-08 2024-10-23 Baylor College Of Medicine GENE THERAPY FOR STXBP1 ENCEPHALOPATHY
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
US12146150B2 (en) 2017-09-29 2024-11-19 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2025049993A1 (en) 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
AU2019346655B2 (en) * 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US12296025B2 (en) 2021-11-02 2025-05-13 Voyager Therapeutics, Inc. AAV capsid variants and uses thereof
WO2025114699A1 (en) 2023-11-30 2025-06-05 Cytomos Limited Cell selection
WO2025128817A1 (en) * 2023-12-15 2025-06-19 Genzyme Corporation Artificial micrornas targeting tau
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
US12440550B2 (en) 2015-03-25 2025-10-14 Board Of Regents, The University Of Texas System Immunogenic compositions and uses thereof
US12467046B2 (en) 2018-10-02 2025-11-11 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
US12472145B2 (en) 2010-07-26 2025-11-18 Board Of Regents, The University Of Texas System Methods for inducing an immune response via buccal and/or sublingual administration of a vaccine
US12491267B2 (en) 2019-11-29 2025-12-09 The Brigham And Women's Hospital, Inc. Gene therapy for neurodegenerative disorders
US12497608B2 (en) 2019-05-22 2025-12-16 The Brigham And Women's Hospital, Inc. Gene therapy for Alzheimer's disease

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AR117842A1 (es) * 2019-09-17 2021-09-01 Voyager Therapeutics Inc Constructos de expresión de frataxina que tienen promotores modificados por ingeniería genética y métodos de uso de estos
CN114728049A (zh) * 2019-10-07 2022-07-08 再生生物股份有限公司 腺相关病毒载体药物组合物和方法
CN112076774A (zh) * 2020-09-11 2020-12-15 华东理工大学 一种碳化钛量子点负载碳缺陷反蛋白石氮化碳的催化剂及其制备方法
US20220127347A1 (en) * 2020-10-26 2022-04-28 The Regents Of The University Of California Inhibition of Tau Propagation
US20220175812A1 (en) 2020-12-03 2022-06-09 Battelle Memorial Institute Polymer nanoparticle and dna nanostructure compositions and methods for non-viral delivery
JP2024516108A (ja) 2021-04-07 2024-04-12 バテル・メモリアル・インスティテュート 非ウイルス性担体を同定および使用するための迅速な設計、構築、試験、および学習技術
CN113913464B (zh) * 2021-09-28 2022-11-22 武汉枢密脑科学技术有限公司 一种表达载体、重组腺相关病毒及其在制备2019新型冠状病毒疫苗中的应用
WO2024020229A2 (en) * 2022-07-22 2024-01-25 Neuronity Therapeutics, Inc. Aav-mediated delivery of rgs10 to microglia
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2024229125A2 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
WO2025072751A1 (en) 2023-09-29 2025-04-03 Battelle Memorial Institute Polymer nanoparticle compositions for in vivo expression of polypeptides
CN117624311B (zh) * 2023-11-29 2024-05-28 广州译码基因科技有限公司 可提高aav病毒神经靶向性的衣壳蛋白突变体及其应用
US12441996B2 (en) 2023-12-08 2025-10-14 Battelle Memorial Institute Use of DNA origami nanostructures for molecular information based data storage systems

Citations (110)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5064764A (en) 1988-12-20 1991-11-12 Commissariat A L'energie Atomique Mineral hollow fiber bioreactor for the cultivation of animal cells
US5387484A (en) 1992-07-07 1995-02-07 International Business Machines Corporation Two-sided mask for patterning of materials with electromagnetic radiation
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
WO1996023810A1 (en) 1994-11-10 1996-08-08 The Regents Of The University Of California Modified green fluorescenct proteins
WO1996030540A2 (en) 1995-03-20 1996-10-03 The Regents Of The University Of California Substrates for beta-lactamase and uses thereof
WO1996039530A2 (en) 1995-06-05 1996-12-12 The Trustees Of The University Of Pennsylvania Recombinant adenovirus and adeno-associated virus, cell lines, and methods of production and use thereof
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5691176A (en) 1990-10-30 1997-11-25 Applied Immune Sciences, Inc. Recombinant adeno-associated virus vector packaging cells and methods for use
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
WO1999015685A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
WO1999047691A1 (en) 1998-03-20 1999-09-23 Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
WO2000024916A1 (en) 1998-10-27 2000-05-04 Crucell Holland B.V. Improved aav vector production
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
WO2000047757A1 (de) 1999-02-10 2000-08-17 Medigene Ag Verfahren zur herstellung eines rekombinanten adeno-assoziierten virus, geeignete mittel hierzu sowie verwendung zur herstellung eines arzneimittels
WO2000055342A1 (en) 1999-03-18 2000-09-21 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO2000075353A1 (en) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
US6180613B1 (en) 1994-04-13 2001-01-30 The Rockefeller University AAV-mediated delivery of DNA to cells of the nervous system
US6194191B1 (en) 1996-11-20 2001-02-27 Introgen Therapeutics, Inc. Method for the production and purification of adenoviral vectors
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6211163B1 (en) 1996-01-18 2001-04-03 Avigen, Inc. Methods for delivering DNA to the bloodstream using recombinant adeno-associated virus vectors
WO2001023597A2 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of e1-deleted adenoviruses in absence of replication competent adenovirus
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
WO2001089583A2 (en) 2000-05-23 2001-11-29 Neurologix, Inc. Glutamic acid decarboxylase (gad)_delivery system for treating neurodegenerative diseases
US20020081721A1 (en) 1996-12-18 2002-06-27 James M. Allen Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
US6436708B1 (en) 1997-04-17 2002-08-20 Paola Leone Delivery system for gene therapy to the brain
US6485966B2 (en) 1999-03-18 2002-11-26 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US20030138772A1 (en) 2001-11-13 2003-07-24 Guangping Gao Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
US20050014264A1 (en) 2002-12-11 2005-01-20 University Of Massachusetts Method of introducing siRNA into adipocytes
WO2005033321A2 (en) 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
EP1752536A1 (en) 2004-05-11 2007-02-14 RNAi Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
US7198951B2 (en) 2001-12-17 2007-04-03 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
WO2008144585A1 (en) 2007-05-17 2008-11-27 Medgenesis Therapeutix Inc. Convection-enhanced delivery catheter with removable stiffening member and method for using same
US7491508B2 (en) 2003-06-20 2009-02-17 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US7588757B2 (en) 2001-03-14 2009-09-15 Genzyme Corporation Methods of treating Parkinson's disease using recombinant adeno-associated virus virions
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20100130594A1 (en) 2007-07-23 2010-05-27 Martine Barkats Cns gene delivery using peripheral administration of aav vectors
US20100240739A1 (en) 2007-10-05 2010-09-23 Martine Barkats Widespread gene delivery to motor neurons using peripheral injection of aav vectors
WO2011008495A2 (en) 2009-06-29 2011-01-20 The Board Of Regents Of The University Of Texas System Arginase formulations and methods
US20110212529A1 (en) 2001-05-24 2011-09-01 David Souza Muscle-specific expression vectors
WO2011130107A2 (en) 2010-04-16 2011-10-20 Surgivision, Inc. Mri surgical systems including mri-compatible surgical cannulae for transferring a substance to and/or from a patient
US8099150B2 (en) 2007-09-24 2012-01-17 MRI Interventions, Inc. MRI-compatible head fixation frame with cooperating head coil apparatus
US8119611B2 (en) 2002-11-26 2012-02-21 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of SIRNA
US8137948B2 (en) 2003-05-21 2012-03-20 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
US8175677B2 (en) 2007-06-07 2012-05-08 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
WO2012116265A2 (en) 2011-02-24 2012-08-30 MRI Interventions, Inc. Mri-guided catheters
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US20120264807A1 (en) 2006-07-11 2012-10-18 Pci Biotech As Method for introducing sirna into cells by photochemical internalisation
US8340743B2 (en) 2007-11-21 2012-12-25 MRI Interventions, Inc. Methods, systems and computer program products for positioning a guidance apparatus relative to a patient
US8369930B2 (en) 2009-06-16 2013-02-05 MRI Interventions, Inc. MRI-guided devices and MRI-guided interventional systems that can track and generate dynamic visualizations of the devices in near real time
WO2013019830A2 (en) 2011-08-01 2013-02-07 Alcyone Lifesciences, Inc. Microfluidic drug delivery devices
US8374677B2 (en) 2007-06-07 2013-02-12 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
US20130195801A1 (en) 2010-04-23 2013-08-01 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US8548569B2 (en) 2007-09-24 2013-10-01 MRI Interventions, Inc. Head fixation assemblies for medical procedures
US20130325012A1 (en) 2012-05-31 2013-12-05 MRI Interventions, Inc. Mri compatible surgical drills and related methods
EP2678433A1 (en) 2011-02-22 2014-01-01 California Institute of Technology Delivery of proteins using adeno-associated virus (aav) vectors
WO2014014585A1 (en) 2012-07-19 2014-01-23 MRI Interventions, Inc. Mri-compatible head fixation apparatus
US20140024927A1 (en) 2012-07-19 2014-01-23 MRI Interventions, Inc. Mri-guided medical interventional systems and methods
US20140066750A1 (en) 2012-09-05 2014-03-06 MRI Interventions, Inc. Trajectory Guide Frame for MRI-Guided Surgeries
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2014144229A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
US20140275980A1 (en) 2013-03-15 2014-09-18 MRI Interventions, Inc. Tip assembly for mri-compatible medical devices and mri-compatible medical devices incorporating same
US20140276614A1 (en) 2013-03-14 2014-09-18 MRI Interventions, Inc. Substance delivery devices, systems and methods
US20140276582A1 (en) 2013-03-13 2014-09-18 MRI Interventions, Inc. Substance delivery devices, systems and methods
WO2014178863A1 (en) 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
US20140359799A1 (en) 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
US20150031982A1 (en) 2012-07-19 2015-01-29 MRI Interventions, Inc. Surgical navigation devices and methods
US8946152B2 (en) 2003-02-24 2015-02-03 Amgen Inc. Method of treating parkinson's disease in humans by convection-enhanced infusion of glial cell-line derived neurotrophic factor to the putamen
WO2015035190A1 (en) 2013-09-05 2015-03-12 Cornell University Gene therapy for alzheimer's and other neurodegenerative diseases and conditions
WO2015038958A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
US8999948B2 (en) 2010-06-10 2015-04-07 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2015057807A1 (en) 2013-10-16 2015-04-23 MRI Interventions, Inc. Surgical navigation systems, related devices and methods
US20150126590A1 (en) 2013-11-01 2015-05-07 Cornell University Gene therapy for alzheimer's and other neurodegenerative diseases and conditions
US9042958B2 (en) 2005-11-29 2015-05-26 MRI Interventions, Inc. MRI-guided localization and/or lead placement systems, related methods, devices and computer program products
US20150159173A1 (en) 2005-04-07 2015-06-11 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US20150230871A1 (en) 2014-02-14 2015-08-20 MRI Interventions, Inc. Surgical tool-positioning devices and related methods
WO2015121501A1 (en) 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector
US20150238550A1 (en) 2012-09-28 2015-08-27 The University Of North Carolina At Chapel Hill AAV Vectors Targeted to Oligodendrocytes
US9163261B2 (en) 2010-02-22 2015-10-20 Koteswara Rao KOLLIPARA Adeno-associated virus 2/8—micro RNA-101 therapy for liver cancer
WO2015168666A2 (en) 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
US9193769B2 (en) 2003-12-04 2015-11-24 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Bovine adeno-associated viral (BAAV) vector and uses thereof
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
US20150376607A1 (en) 2011-08-24 2015-12-31 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins for nucleic acid transfer
US20150376240A1 (en) 2013-02-08 2015-12-31 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9238800B2 (en) 2003-05-19 2016-01-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
US20160017295A1 (en) 2013-05-31 2016-01-21 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US20160032254A1 (en) 2007-07-26 2016-02-04 Uniqure Ip B.V. Aav vectors produced by insect cells comprising rep52 and rep78 coding sequences with differential codon biases
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
WO2016134375A1 (en) 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
US9475845B2 (en) 2009-02-11 2016-10-25 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
WO2017058892A2 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
US9624274B2 (en) 2007-05-31 2017-04-18 Medigene Ag Mutated structural protein of a parvovirus
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
US20170145405A1 (en) 2015-11-25 2017-05-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas-mediated genome editing to treat egfr-mutant lung cancer
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)

Family Cites Families (246)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5693531A (en) 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
US5652224A (en) 1995-02-24 1997-07-29 The Trustees Of The University Of Pennsylvania Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
US6197293B1 (en) 1997-03-03 2001-03-06 Calydon, Inc. Adenovirus vectors specific for cells expressing androgen receptor and methods of use thereof
US6676935B2 (en) 1995-06-27 2004-01-13 Cell Genesys, Inc. Tissue specific adenoviral vectors
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5952221A (en) 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US7026468B2 (en) 1996-07-19 2006-04-11 Valentis, Inc. Process and equipment for plasmid purification
AU722375B2 (en) 1996-09-06 2000-08-03 Trustees Of The University Of Pennsylvania, The Methods using cre-lox for production of recombinant adeno-associated viruses
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
WO1998010087A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
ATE465267T1 (de) 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
US7732129B1 (en) 1998-12-01 2010-06-08 Crucell Holland B.V. Method for the production and purification of adenoviral vectors
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6642051B1 (en) 1997-10-21 2003-11-04 Targeted Genetics Corporation Amplifiable adeno-associated virus(AAV) packaging cassettes for the production of recombinant AAV vectors
IT1297074B1 (it) 1997-11-21 1999-08-03 Angeletti P Ist Richerche Bio Forme ormone-dipendenti delle proteine rep del virus adeno-associato (aav-2), sequenze di dna codificanti per esse, vettori che le
JP2001526900A (ja) 1997-12-23 2001-12-25 イントロヘーネ ベスローテン フェンノートシャップ 標的細胞の染色体ｄｎａへの外来遺伝子情報の組み込みに有用な、アデノ随伴ウイルスおよびアデノウイルスのキメラ組換えウイルス
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
WO1999043360A1 (en) 1998-02-26 1999-09-02 The Trustees Of The University Of Pennsylvania Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex
US6521426B1 (en) 1998-04-08 2003-02-18 Istituto Di Ricerche Di Biologia Molecolare P. Angeletti S.P.A. Preparation of recombinant adenovirus carrying a rep gene of adeno-associated virus
FR2778413B1 (fr) 1998-05-07 2000-08-04 Immunotech Sa Nouveaux reactifs et methode de lyse des erythrocytes
EP1078096A1 (en) 1998-05-11 2001-02-28 Ariad Gene Therapeutics, Inc. Multiviral compositions and uses thereof
EP1849872A1 (en) 1998-05-20 2007-10-31 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
JP2002516345A (ja) 1998-05-27 2002-06-04 セル ジェネシス インコーポレイテッド 第ｖｉｉｉ因子活性のアデノ随伴ウイルスベクター媒介性発現
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
ES2313784T3 (es) 1998-05-28 2009-03-01 The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services Vector aav5 y usos del mismo.
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
GB2338236B (en) 1998-06-13 2003-04-09 Aea Technology Plc Microbiological cell processing
WO2000022152A1 (en) 1998-10-13 2000-04-20 Avigen, Inc. Compositions and methods for producing recombinant adeno-associated virus
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6689600B1 (en) 1998-11-16 2004-02-10 Introgen Therapeutics, Inc. Formulation of adenovirus for gene therapy
US6225113B1 (en) 1998-12-04 2001-05-01 Genvec, Inc. Use of trans-activation and cis-activation to modulate the persistence of expression of a transgene in an at least E4-deficient adenovirus
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6509150B1 (en) 1999-03-05 2003-01-21 Universite De Nantes Compositions and methods for recombinant Adeno-Associated Virus production
JP2002542805A (ja) 1999-04-30 2002-12-17 ユニバーシティ オブ フロリダ アデノ随伴ウイルス送達リボザイム組成物および使用方法
WO2000075365A2 (en) 1999-06-08 2000-12-14 University Of Iowa Research Foundation Compounds and methods to enhance raav transduction
CA2382483A1 (en) 1999-08-20 2001-03-01 Johns Hopkins University School Of Medicine Methods and compositions for the construction and use of fusion libraries
EP1218035A2 (en) 1999-09-29 2002-07-03 The Trustees Of The University Of Pennsylvania Rapid peg-modification of viral vectors
WO2001025465A1 (en) 1999-10-07 2001-04-12 University Of Iowa Research Foundation Adeno-associated viruses and uses thereof
US7241447B1 (en) 1999-10-07 2007-07-10 University Of Iowa Research Foundation Adeno-associated virus vectors and uses thereof
WO2001032711A2 (en) 1999-10-21 2001-05-10 Board Of Trustees Of The University Of Arkansas Adeno-associated virus aav rep78 major regulatory protein, mutants thereof and uses thereof
WO2001036623A2 (en) 1999-11-05 2001-05-25 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
US6582692B1 (en) 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
EP1240345A2 (en) 1999-12-10 2002-09-18 Ariad Gene Therapeutics, Inc. Methods for expression of genes in primates
US7638120B2 (en) 2000-03-14 2009-12-29 Thomas Jefferson University High transgene expression of a pseudotyped adeno-associated virus type
EP1228234A2 (en) 2000-03-14 2002-08-07 Neurologix, Inc. Production of chimeric capsid vectors
US6468524B1 (en) 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
US7048920B2 (en) 2000-03-24 2006-05-23 Cell Genesys, Inc. Recombinant oncolytic adenovirus for human melanoma
WO2001083797A2 (en) 2000-04-28 2001-11-08 Avigen, Inc. Polynucleotides for use in recombinant adeno-associated virus virion production
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US20030013189A1 (en) 2000-04-28 2003-01-16 Wilson James M. Compositions and methods useful for non-invasive delivery of therapeutic molecules to the bloodstream
AU2001268373A1 (en) 2000-06-13 2001-12-24 The Children's Hospital Of Philadelphia Methods for administering recombinant adeno-associated virus virions to humans previously exposed to adeno-associated virus
WO2002006483A1 (fr) 2000-07-18 2002-01-24 Takeda Chemical Industries, Ltd. Nouveau peptide actif sur le plan physiologique et utilisation associee
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US6593123B1 (en) 2000-08-07 2003-07-15 Avigen, Inc. Large-scale recombinant adeno-associated virus (rAAV) production and purification
WO2002014487A2 (en) 2000-08-17 2002-02-21 Keiya Ozawa Adeno-associated virus-mediated delivery of angiogenic factors
DE10044384A1 (de) 2000-09-08 2002-04-18 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
FR2813891B1 (fr) 2000-09-14 2005-01-14 Immunotech Sa Reactif multifonctionnel pour erythrocytes mettant en jeu des carbamates et applications
WO2002070719A2 (en) 2001-01-19 2002-09-12 Trustees Of The University Of Pennsylvania Regulatable gene expression system
JP2004532822A (ja) 2001-03-14 2004-10-28 アビジェン， インコーポレイテッド ビリオンの管逆行感染による組換えアデノ随伴ウイルス媒介遺伝子移入
ES2375557T3 (es) 2001-06-22 2012-03-02 The Trustees Of The University Of Pennsylvania Adenovirus recombinantes que comprenden prote�?nas de adenovirus de simios y usos de los mismos.
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
EP1279740A1 (en) 2001-07-26 2003-01-29 Vrije Universiteit Brussel Recombinant vector derived from adeno-associated virus for gene therapy
CA2455499C (en) 2001-08-08 2011-06-21 The Trustees Of The University Of Pennsylvania Method for purification of viral vectors having proteins which bind sialic acid
AU2002360355B2 (en) 2001-11-09 2005-07-07 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1944043A1 (en) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
KR100987360B1 (ko) 2001-11-21 2010-10-12 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 원숭이 아데노바이러스 핵산과 아미노산 서열, 이를함유하는 벡터 및 사용방법
WO2003049763A1 (en) 2001-12-12 2003-06-19 Fh Faulding & Co Limited Composition for the preservation of viruses
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
EP1463530A4 (en) 2001-12-19 2006-09-06 Lijun Wang ADENO-ASSOCIATED VIRUS-RELATED DELIVERY OF GDNF TO SKELETAL MUSCLES
US8110351B2 (en) 2002-01-16 2012-02-07 Invitrogen Dynal As Method for isolating nucleic acids and protein from a single sample
GB0208390D0 (en) 2002-04-11 2002-05-22 Univ London Adeno-associated virus producer system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
ES2280694T3 (es) 2002-04-29 2007-09-16 The Trustees Of The University Of Pennsylvania Metodo para la extraccion directa y la amplificacion de virus integrados del adn celular de tejidos.
ATE427990T1 (de) 2002-04-30 2009-04-15 Oncolytics Biotech Inc Verbesserte reinigungsmethode fur viren
WO2003097797A2 (en) 2002-05-14 2003-11-27 Merck & Co., Inc. Methods of adenovirus purification
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
WO2003104413A2 (en) 2002-06-05 2003-12-18 University Of Florida Production of pseudotyped recombinant aav virions
EP1418185A1 (en) 2002-11-11 2004-05-12 Aventis Pharma Deutschland GmbH Use of EDG2 receptor in an animal model of heart failure
US7169612B2 (en) 2002-11-11 2007-01-30 Sanofi-Aventis Deutschland Gmbh Use of EDG2 receptor in an animal model of heart failure
US7510872B2 (en) 2003-02-26 2009-03-31 Nationwide Children's Hospital Recombinant adeno-associated virus production
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
JP4888876B2 (ja) 2003-06-13 2012-02-29 田平 武 アルツハイマー病の治療のための組換えアデノ随伴ウィルスベクター
PL3235827T3 (pl) 2003-06-19 2021-07-05 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
ES2391975T3 (es) 2003-07-25 2012-12-03 Genvec, Inc. Vacunas a base de vector adenovírico
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
WO2006078279A2 (en) 2004-04-28 2006-07-27 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
EP4170024A1 (en) 2004-06-01 2023-04-26 Genzyme Corporation Compositions and methods to prevent aav vector aggregation
US7901921B2 (en) 2004-10-22 2011-03-08 Oncolytics Biotech Inc. Viral purification methods
CN1286981C (zh) 2004-11-30 2006-11-29 华中科技大学同济医学院附属同济医院 表达人类cyp2j2反义基因的重组腺相关病毒及其制备方法
US8614101B2 (en) 2008-05-20 2013-12-24 Rapid Pathogen Screening, Inc. In situ lysis of cells in lateral flow immunoassays
US7625570B1 (en) 2005-03-10 2009-12-01 The Regents Of The University Of California Methods for purifying adeno-associated virus
WO2006102072A2 (en) 2005-03-23 2006-09-28 The Trustees Of The University Of Pennsylvania Use of a pa131 polypeptide in treatment of atherosclerosis
CN107007842A (zh) 2005-05-02 2017-08-04 建新公司 神经代谢疾病的基因治疗
WO2007024841A2 (en) 2005-08-23 2007-03-01 The Regents Of The University Of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
EP1857552A1 (en) 2006-05-20 2007-11-21 Cargill Incorporated Thermostable xylose isomerase enzyme
WO2007046703A2 (en) 2005-10-20 2007-04-26 Amsterdam Molecular Therapeutics B.V. Improved aav vectors produced in insect cells
US20080003565A1 (en) 2006-05-02 2008-01-03 Government Of The Us, As Represented By The Secretary, Department Of Health And Human Services Viral nucleic acid microarray and method of use
US8512981B2 (en) 2006-06-21 2013-08-20 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV
WO2008018795A1 (en) 2006-08-11 2008-02-14 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
RU2491344C2 (ru) 2007-06-29 2013-08-27 Ф.Хоффманн-Ля Рош Аг Промотор
ES2615180T3 (es) 2007-07-14 2017-06-05 University Of Iowa Research Foundation Métodos y composiciones para el tratamiento de enfermedades cerebrales
US8420372B2 (en) 2007-09-04 2013-04-16 Alexander Bello Porcine adeno-associated viruses
US9115373B2 (en) 2007-09-19 2015-08-25 Uniqure Ip B.V. Use of AAV replication machinery for improved protein production
CA2706257C (en) 2007-11-28 2017-06-06 The Trustees Of The University Of Pennsylvania Simian subfamily b adenovirus sadv-28, -27, -29, -32, -33 and -35 and uses thereof
EP2220217A2 (en) 2007-11-28 2010-08-25 The Trustees of the University of Pennsylvania Simian subfamily c adenoviruses sadv-40, -31, and-34 and uses thereof
CN101883858B (zh) 2007-11-28 2015-07-22 宾夕法尼亚大学托管会 猿猴亚家族E腺病毒SAdV-39、-25.2、-26、-30、-37和-38及其应用
WO2009104964A1 (en) 2008-02-19 2009-08-27 Amsterdam Molecular Therapeutics B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
CN102016011B (zh) 2008-03-04 2013-12-11 宾夕法尼亚大学托管会 猿猴腺病毒sadv-36、-42.1、-42.2和-44及其应用
WO2009134681A2 (en) 2008-04-30 2009-11-05 The Trustees Of The University Of Pennsylvania Aav7 viral vectors for targeted delivery of rpe cells
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
MX2011003229A (es) 2008-09-29 2011-09-06 Proyecto Biomedicina Cima Sl Terapia genica con porfobilinogeno desaminasa.
DK2350269T3 (en) 2008-10-31 2015-12-07 Univ Pennsylvania ABE ADENOVIRUS WITH SADV-46 HEXONCAPSIDE PROTEINS AND APPLICATIONS THEREOF
MX2011009193A (es) 2009-03-04 2011-12-16 Deutsches Krebsforsch Proteina activadora del ensamblaje (aap) y su uso para la fabricacion de particulas de parvovirus que consisten esencialmente de vp3.
MX2011010097A (es) 2009-03-27 2011-10-19 Proyecto Biomedicina Cima Sl Metodos y composiciones para el tratamiento de cirrosis y fibrosis hepatica.
ES2927225T3 (es) 2009-04-17 2022-11-03 Univ Oxford Innovation Ltd Composición para entrega de material genético
CA2762203A1 (en) 2009-05-29 2010-12-02 Soumitra Roy Simian adenovirus 41 and uses thereof
CN102803478B (zh) 2009-06-16 2016-04-27 建新公司 用于纯化重组aav载体的改进方法
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
US20120309816A1 (en) 2009-11-09 2012-12-06 Genepod Therapeutics Ab Novel viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
CA2788682C (en) 2010-02-05 2019-03-05 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
WO2011097641A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
WO2011097614A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Mehods and compositions useful in diseases or conditions related to repeat expansion
EP2550021A2 (en) 2010-03-22 2013-01-30 Association Institut de Myologie Methods of increasing efficiency of vector penetration of target tissue
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
KR20180105730A (ko) 2010-07-19 2018-09-28 아이오니스 파마수티컬즈, 인코포레이티드 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
CN103080314B (zh) 2010-09-30 2016-04-13 Lsip基金运营联合公司 显性突变基因表达抑制剂
EP2612909B1 (en) 2010-10-05 2015-02-25 Takara Bio, Inc. Method for producing virus vector
EP3075860A1 (en) 2010-11-23 2016-10-05 The Trustees of the University of Pennsylvania Subfamily e simian adenovirus a1295 and uses thereof
GB201103062D0 (en) 2011-02-22 2011-04-06 Isis Innovation Method
EP2500434A1 (en) 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
EP2700399B1 (en) 2011-04-18 2017-05-31 National Center of Neurology and Psychiatry Drug delivery particles and method for producing same
ES2623780T3 (es) 2011-07-27 2017-07-12 Genethon Sistemas de expresión de baculovirus mejorados
US9976138B2 (en) 2011-08-29 2018-05-22 Ionis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion
WO2013036889A1 (en) 2011-09-09 2013-03-14 University Of Washington Retrograde transport peptide and use of same for delivery to central nervous system
JP6165752B2 (ja) 2011-10-28 2017-07-19 ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル アデノ随伴ウイルスの産生のための細胞株
WO2013078199A2 (en) 2011-11-23 2013-05-30 Children's Medical Center Corporation Methods for enhanced in vivo delivery of synthetic, modified rnas
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
US20140162319A2 (en) 2012-05-02 2014-06-12 Sangeetha Hareendran Nucleotide sequences, methods, kit and a recombinant cell thereof
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
EP2847337A4 (en) 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
JP6293738B2 (ja) 2012-05-18 2018-03-14 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 亜科ｅサルアデノウイルスａ１３０２、ａ１３２０、ａ１３３１及びａ１３３７ならびにそれらの使用
WO2014007120A1 (ja) 2012-07-06 2014-01-09 タカラバイオ株式会社 アデノ随伴ウイルスベクターの産生細胞
EP3769789A1 (en) 2012-08-01 2021-01-27 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
EP2900821B1 (en) 2012-09-25 2020-04-01 Genzyme Corporation Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy
EP2970946A4 (en) 2013-03-13 2016-09-07 Philadelphia Children Hospital ADENOASSOZED VIRUS VECTORS AND METHOD FOR USE THEREOF
EP2983707B1 (en) 2013-04-08 2019-06-12 University of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
WO2014167074A1 (en) 2013-04-12 2014-10-16 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for modulating rna alternative splicing in a subject in need thereof
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
US10494645B2 (en) 2013-04-18 2019-12-03 Fondazione Telethon Effective delivery of large genes by dual AAV vectors
MX388221B (es) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2.
WO2015012924A2 (en) 2013-04-29 2015-01-29 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof
RU2692251C2 (ru) 2013-05-15 2019-06-24 Риджентс Оф Зэ Юниверсити Оф Миннесота Опосредованный аденоассоциированным вирусом перенос генов в центральную нервную систему
WO2014186746A1 (en) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION
WO2014201252A2 (en) 2013-06-13 2014-12-18 Shire Human Genetic Therapies, Inc. Messenger rna based viral production
ITTO20130669A1 (it) 2013-08-05 2015-02-06 Consiglio Nazionale Ricerche Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
US11078464B2 (en) 2013-08-30 2021-08-03 Amgen Inc. High titer recombinant AAV vector production in adherent and suspension cells
US20160354489A1 (en) 2013-09-26 2016-12-08 Universitat Autònome de Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
CA2927366A1 (en) 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
WO2015108610A1 (en) 2013-11-08 2015-07-23 The Board Of Trustees Of The University Of Arkansas Adeno-associated virus "x" oncogene
GB201401707D0 (en) 2014-01-31 2014-03-19 Sec Dep For Health The Adeno-associated viral vectors
WO2015124546A1 (en) 2014-02-19 2015-08-27 Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii- Cnic Aav vectors for the treatment of ischemic and non-ischemic heart disease
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
PL3117005T3 (pl) 2014-03-10 2024-11-04 Uniqure Ip B.V. Dodatkowo ulepszone wektory aav produkowane w komórkach owadzich
WO2015164778A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennysylvania Ldlr variants and their use in compositions for reducing cholesterol levels
MX381946B (es) 2014-05-23 2025-03-13 Genzyme Corp Restos oligonucleotidicos multiples sobre soporte peptidico.
WO2015196179A1 (en) 2014-06-20 2015-12-23 University Of Florida Research Foundation, Inc. Methods of packaging multiple adeno-associated virus vectors
WO2016006658A1 (ja) 2014-07-10 2016-01-14 タカラバイオ株式会社 非エンベロープウイルス粒子の製造方法
WO2016019364A1 (en) 2014-08-01 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
RU2727015C2 (ru) 2014-11-21 2020-07-17 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Векторы aav, нацеленные на центральную нервную систему
US10900053B2 (en) 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
WO2016115382A1 (en) 2015-01-14 2016-07-21 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
MX2017009336A (es) * 2015-01-16 2017-11-15 Voyager Therapeutics Inc Polinucleótidos dirigidos al sistema nervioso central.
EP3250279B1 (en) 2015-01-30 2020-08-19 The Regents of the University of California Spinal subpial gene delivery system
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
AU2016219398A1 (en) 2015-02-10 2017-09-28 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex
US20180245073A1 (en) 2015-02-23 2018-08-30 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
EP3268475B1 (en) 2015-03-11 2020-10-21 Yissum Research and Development Company of the Hebrew University of Jerusalem Ltd. Decoy oligonucleotides for the treatment of diseases
CN107614008A (zh) 2015-03-20 2018-01-19 蓝鸟生物公司 载体制剂
EP3273999A1 (en) * 2015-03-23 2018-01-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment and the prevention of neurological phenotype associated with friedreich ataxia
EP3277819B1 (en) 2015-03-24 2021-03-03 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
US10058624B2 (en) 2015-04-16 2018-08-28 Emory University Recombinant promoters and vectors for protein expression in liver and use thereof
US11020443B2 (en) 2015-04-23 2021-06-01 University Of Massachusetts Modulation of AAV vector transgene expression
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions and uses thereof
EP3286321A4 (en) * 2015-04-24 2018-10-17 University of Florida Research Foundation, Inc. Aav vector for treatment of friedreich's ataxia
EP3291765A4 (en) 2015-05-07 2019-01-23 Massachusetts Eye & Ear Infirmary METHOD FOR THE DELIVERY OF AN ACTIVE SUBSTANCE TO THE EYE
CA2985786A1 (en) 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
WO2016191418A1 (en) 2015-05-26 2016-12-01 Salk Institute For Biological Studies Motor neuron-specific expression vectors
WO2016196507A1 (en) 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
US11266748B2 (en) 2015-07-02 2022-03-08 University Of Florida Research Foundation, Incorporated Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
ES2895652T3 (es) 2015-07-07 2022-02-22 Inst Nat Sante Rech Med Métodos y composiciones farmacéuticas para expresar un polinucleótido de interés en el sistema nervioso periférico de un sujeto
US20180214576A1 (en) 2015-07-28 2018-08-02 University Of Massachusetts Transgenic expression of dnasei in vivo delivered by an adeno-associated virus vector
LT3872085T (lt) 2015-07-30 2023-05-10 Massachusetts Eye & Ear Infirmary Viruso prototipo sekos ir jų panaudojimas
US11116843B2 (en) 2015-09-25 2021-09-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
CA3002982A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070678A1 (en) 2015-10-23 2017-04-27 University Of Iowa Research Foundation Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
WO2017075338A2 (en) 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
US10633662B2 (en) 2015-11-10 2020-04-28 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating AAV infection
KR20180091863A (ko) 2015-12-01 2018-08-16 스파크 테라퓨틱스, 인코포레이티드 임상적 사용에 적합한 무혈청 현탁 세포 배양 시스템에서 재조합 아데노-관련 바이러스(aav) 벡터를 생성하기 위한 확장가능한 방법
EP3383896A1 (en) 2015-12-03 2018-10-10 Genethon Compositions and methods for improving viral vector efficiency
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
ES2934848T3 (es) 2015-12-11 2023-02-27 Univ Pennsylvania Método de purificación escalable para AAV8
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
GB201605126D0 (en) 2016-03-24 2016-05-11 Univ Nottingham Inhibitors and their uses
US10940161B2 (en) 2016-04-04 2021-03-09 University Of Florida Research Foundation, Incorporated Manipulation of EIF3 to modulate repeat associated non-ATG (RAN) translation
WO2017182581A1 (en) 2016-04-20 2017-10-26 Universität Basel A method for evaluating a candidate compound (t) for treating myotonic dystrophy type i (dm1)
ES2659845B1 (es) 2016-09-19 2019-01-04 Univ Valencia Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
WO2018115477A1 (en) 2016-12-23 2018-06-28 Universite De Strasbourg Dynamin 2 inhibitor for the treatment of myotonic dystrophy
WO2018129384A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US11680249B2 (en) 2017-08-28 2023-06-20 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
EP3687582A4 (en) * 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
TW202015742A (zh) * 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒（ａａｖ）之組成物和方法
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav

Patent Citations (136)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5064764A (en) 1988-12-20 1991-11-12 Commissariat A L'energie Atomique Mineral hollow fiber bioreactor for the cultivation of animal cells
US5691176A (en) 1990-10-30 1997-11-25 Applied Immune Sciences, Inc. Recombinant adeno-associated virus vector packaging cells and methods for use
US5387484A (en) 1992-07-07 1995-02-07 International Business Machines Corporation Two-sided mask for patterning of materials with electromagnetic radiation
US6180613B1 (en) 1994-04-13 2001-01-30 The Rockefeller University AAV-mediated delivery of DNA to cells of the nervous system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
WO1996023810A1 (en) 1994-11-10 1996-08-08 The Regents Of The University Of California Modified green fluorescenct proteins
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
WO1996030540A2 (en) 1995-03-20 1996-10-03 The Regents Of The University Of California Substrates for beta-lactamase and uses thereof
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US6261551B1 (en) 1995-06-05 2001-07-17 The Trustees Of The University Of Pennsylvania Recombinant adenovirus and adeno-associated virus, cell lines, and methods of production and use thereof
US6270996B1 (en) 1995-06-05 2001-08-07 The Trustees Of The University Of Pennsylvania Recombinant adenovirus and adeno-associated virus, cell lines and methods of production and use thereof
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
WO1996039530A2 (en) 1995-06-05 1996-12-12 The Trustees Of The University Of Pennsylvania Recombinant adenovirus and adeno-associated virus, cell lines, and methods of production and use thereof
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6555525B2 (en) 1995-08-31 2003-04-29 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US6211163B1 (en) 1996-01-18 2001-04-03 Avigen, Inc. Methods for delivering DNA to the bloodstream using recombinant adeno-associated virus vectors
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US6194191B1 (en) 1996-11-20 2001-02-27 Introgen Therapeutics, Inc. Method for the production and purification of adenoviral vectors
US20020081721A1 (en) 1996-12-18 2002-06-27 James M. Allen Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
US6436708B1 (en) 1997-04-17 2002-08-20 Paola Leone Delivery system for gene therapy to the brain
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999015685A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
US6475769B1 (en) 1997-09-19 2002-11-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
US6943019B2 (en) 1997-09-19 2005-09-13 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant AAV
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
US6482634B1 (en) 1997-09-19 2002-11-19 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant AAV
US7238526B2 (en) 1997-09-19 2007-07-03 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
WO1999047691A1 (en) 1998-03-20 1999-09-23 Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
WO2000024916A1 (en) 1998-10-27 2000-05-04 Crucell Holland B.V. Improved aav vector production
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
WO2000047757A1 (de) 1999-02-10 2000-08-17 Medigene Ag Verfahren zur herstellung eines rekombinanten adeno-assoziierten virus, geeignete mittel hierzu sowie verwendung zur herstellung eines arzneimittels
WO2000055342A1 (en) 1999-03-18 2000-09-21 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US7022519B2 (en) 1999-03-18 2006-04-04 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6485966B2 (en) 1999-03-18 2002-11-26 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
WO2000075353A1 (en) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
WO2001023597A2 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of e1-deleted adenoviruses in absence of replication competent adenovirus
WO2001089583A2 (en) 2000-05-23 2001-11-29 Neurologix, Inc. Glutamic acid decarboxylase (gad)_delivery system for treating neurodegenerative diseases
US7588757B2 (en) 2001-03-14 2009-09-15 Genzyme Corporation Methods of treating Parkinson's disease using recombinant adeno-associated virus virions
US20110212529A1 (en) 2001-05-24 2011-09-01 David Souza Muscle-specific expression vectors
US20030138772A1 (en) 2001-11-13 2003-07-24 Guangping Gao Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
US7198951B2 (en) 2001-12-17 2007-04-03 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
US8119611B2 (en) 2002-11-26 2012-02-21 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of SIRNA
US20050014264A1 (en) 2002-12-11 2005-01-20 University Of Massachusetts Method of introducing siRNA into adipocytes
US8946152B2 (en) 2003-02-24 2015-02-03 Amgen Inc. Method of treating parkinson's disease in humans by convection-enhanced infusion of glial cell-line derived neurotrophic factor to the putamen
US9238800B2 (en) 2003-05-19 2016-01-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
US8137948B2 (en) 2003-05-21 2012-03-20 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
US7491508B2 (en) 2003-06-20 2009-02-17 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005033321A2 (en) 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US20150315612A1 (en) 2003-09-30 2015-11-05 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US9193769B2 (en) 2003-12-04 2015-11-24 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Bovine adeno-associated viral (BAAV) vector and uses thereof
EP1752536A1 (en) 2004-05-11 2007-02-14 RNAi Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
US20150159173A1 (en) 2005-04-07 2015-06-11 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US9042958B2 (en) 2005-11-29 2015-05-26 MRI Interventions, Inc. MRI-guided localization and/or lead placement systems, related methods, devices and computer program products
US20150223905A1 (en) 2005-11-29 2015-08-13 MRI Interventions, Inc. Surgical image-guided navigation devices and related systems
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20120264807A1 (en) 2006-07-11 2012-10-18 Pci Biotech As Method for introducing sirna into cells by photochemical internalisation
US20100217228A1 (en) 2007-05-17 2010-08-26 Medgenesis Therapeutix, Inc. Convection-Enhanced Delivery Catheter with Removable Stiffening Member and Method for Using Same
WO2008144585A1 (en) 2007-05-17 2008-11-27 Medgenesis Therapeutix Inc. Convection-enhanced delivery catheter with removable stiffening member and method for using same
US9624274B2 (en) 2007-05-31 2017-04-18 Medigene Ag Mutated structural protein of a parvovirus
US9055884B2 (en) 2007-06-07 2015-06-16 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
US8374677B2 (en) 2007-06-07 2013-02-12 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
US8175677B2 (en) 2007-06-07 2012-05-08 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
US20100130594A1 (en) 2007-07-23 2010-05-27 Martine Barkats Cns gene delivery using peripheral administration of aav vectors
US20160032254A1 (en) 2007-07-26 2016-02-04 Uniqure Ip B.V. Aav vectors produced by insect cells comprising rep52 and rep78 coding sequences with differential codon biases
US8099150B2 (en) 2007-09-24 2012-01-17 MRI Interventions, Inc. MRI-compatible head fixation frame with cooperating head coil apparatus
US8548569B2 (en) 2007-09-24 2013-10-01 MRI Interventions, Inc. Head fixation assemblies for medical procedures
US20100240739A1 (en) 2007-10-05 2010-09-23 Martine Barkats Widespread gene delivery to motor neurons using peripheral injection of aav vectors
US8340743B2 (en) 2007-11-21 2012-12-25 MRI Interventions, Inc. Methods, systems and computer program products for positioning a guidance apparatus relative to a patient
US9475845B2 (en) 2009-02-11 2016-10-25 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US8886288B2 (en) 2009-06-16 2014-11-11 MRI Interventions, Inc. MRI-guided devices and MRI-guided interventional systems that can track and generate dynamic visualizations of the devices in near real time
US8369930B2 (en) 2009-06-16 2013-02-05 MRI Interventions, Inc. MRI-guided devices and MRI-guided interventional systems that can track and generate dynamic visualizations of the devices in near real time
US8768433B2 (en) 2009-06-16 2014-07-01 MRI Interventions, Inc. MRI-guided devices and MRI-guided interventional systems that can track and generate dynamic visualizations of the devices in near real time
US8825133B2 (en) 2009-06-16 2014-09-02 MRI Interventions, Inc. MRI-guided catheters
US8396532B2 (en) 2009-06-16 2013-03-12 MRI Interventions, Inc. MRI-guided devices and MRI-guided interventional systems that can track and generate dynamic visualizations of the devices in near real time
WO2011008495A2 (en) 2009-06-29 2011-01-20 The Board Of Regents Of The University Of Texas System Arginase formulations and methods
US9163261B2 (en) 2010-02-22 2015-10-20 Koteswara Rao KOLLIPARA Adeno-associated virus 2/8—micro RNA-101 therapy for liver cancer
WO2011130107A2 (en) 2010-04-16 2011-10-20 Surgivision, Inc. Mri surgical systems including mri-compatible surgical cannulae for transferring a substance to and/or from a patient
US20130195801A1 (en) 2010-04-23 2013-08-01 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US8999948B2 (en) 2010-06-10 2015-04-07 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
EP2678433A1 (en) 2011-02-22 2014-01-01 California Institute of Technology Delivery of proteins using adeno-associated virus (aav) vectors
US20140024909A1 (en) 2011-02-24 2014-01-23 MRI Interventions, Inc. Mri-guided catheters
WO2012116265A2 (en) 2011-02-24 2012-08-30 MRI Interventions, Inc. Mri-guided catheters
US20130035660A1 (en) 2011-08-01 2013-02-07 Alcyone Lifesciences, Inc. Multidirectional microfluidic drug delivery devices with conformable balloons
US20130035574A1 (en) 2011-08-01 2013-02-07 Alcyone Lifesciences, Inc. Microfluidic drug delivery devices with venturi effect
WO2013019830A2 (en) 2011-08-01 2013-02-07 Alcyone Lifesciences, Inc. Microfluidic drug delivery devices
US20150376607A1 (en) 2011-08-24 2015-12-31 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins for nucleic acid transfer
US20140359799A1 (en) 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
US20130325012A1 (en) 2012-05-31 2013-12-05 MRI Interventions, Inc. Mri compatible surgical drills and related methods
WO2013181008A1 (en) 2012-05-31 2013-12-05 MRI Interventions, Inc. Mri compatible surgical drills and related methods
US20150031982A1 (en) 2012-07-19 2015-01-29 MRI Interventions, Inc. Surgical navigation devices and methods
US20140024927A1 (en) 2012-07-19 2014-01-23 MRI Interventions, Inc. Mri-guided medical interventional systems and methods
WO2014014585A1 (en) 2012-07-19 2014-01-23 MRI Interventions, Inc. Mri-compatible head fixation apparatus
US9031636B2 (en) 2012-07-19 2015-05-12 MRI Interventions, Inc. MRI-compatible head fixation apparatus
US20140066750A1 (en) 2012-09-05 2014-03-06 MRI Interventions, Inc. Trajectory Guide Frame for MRI-Guided Surgeries
WO2014039481A1 (en) 2012-09-05 2014-03-13 MRI Interventions, Inc. Trajectory guide frame for mri-guided surgeries
US20150238550A1 (en) 2012-09-28 2015-08-27 The University Of North Carolina At Chapel Hill AAV Vectors Targeted to Oligodendrocytes
US20150376240A1 (en) 2013-02-08 2015-12-31 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
US20140276582A1 (en) 2013-03-13 2014-09-18 MRI Interventions, Inc. Substance delivery devices, systems and methods
US20140276614A1 (en) 2013-03-14 2014-09-18 MRI Interventions, Inc. Substance delivery devices, systems and methods
WO2014144229A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
US20140275980A1 (en) 2013-03-15 2014-09-18 MRI Interventions, Inc. Tip assembly for mri-compatible medical devices and mri-compatible medical devices incorporating same
US20160017005A1 (en) 2013-03-15 2016-01-21 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
WO2014178863A1 (en) 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
US20160017295A1 (en) 2013-05-31 2016-01-21 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
WO2015035190A1 (en) 2013-09-05 2015-03-12 Cornell University Gene therapy for alzheimer's and other neurodegenerative diseases and conditions
WO2015038958A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015057807A1 (en) 2013-10-16 2015-04-23 MRI Interventions, Inc. Surgical navigation systems, related devices and methods
US20150126590A1 (en) 2013-11-01 2015-05-07 Cornell University Gene therapy for alzheimer's and other neurodegenerative diseases and conditions
US20150230871A1 (en) 2014-02-14 2015-08-20 MRI Interventions, Inc. Surgical tool-positioning devices and related methods
WO2015121501A1 (en) 2014-02-17 2015-08-20 King's College London Adeno-associated virus vector
WO2015168666A2 (en) 2014-05-02 2015-11-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2016049230A1 (en) 2014-09-24 2016-03-31 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
WO2016134375A1 (en) 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
WO2017058892A2 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
US20170145405A1 (en) 2015-11-25 2017-05-25 The Board Of Trustees Of The Leland Stanford Junior University Crispr/cas-mediated genome editing to treat egfr-mutant lung cancer
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)

Non-Patent Citations (82)

* Cited by examiner, † Cited by third party
Title
"Biocomputing: Informatics and Genome Projects", 1993, ACADEMIC PRESS
"Computational Molecular Biology", 1988, OXFORD UNIVERSITY PRESS
"Computer Analysis of Sequence Data, Part I", 1994, HUMANA PRESS
"Methods In Molecular Biology", 1995, HUMANA PRESS
"NCBI", Database accession no. NM _002111.7
"NCBI", Database accession no. NM_000454.4
"NCBI", Database accession no. NP 002102.4
"Pharmaceutical Salts: Properties, Selection, and Use", 2008, WILEY-VCH
"Remington's Pharmaceutical Sciences", 1985, MACK PUBLISHING COMPANY, pages: 1418
"Sequence Analysis Primer", 1991, M STOCKTON PRESS
A. R. GENNARO: "Remington: The Science and Practice of Pharmacy", 2006, LIPPINCOTT, WILLIAMS & WILKINS
ALLEN ET AL., J NEUROSCI., vol. 22, no. 21, 2002, pages 9340 - 51
ALTSCHUL, S. F. ET AL., J. MOLEC. BIOI., vol. 215, 1990, pages 403
ARAI ET AL., CIRCULATION RESEARCH, vol. 72, no. 2, February 2013 (2013-02-01)
BELLUCCI ET AL., AM J PATHOL., vol. 165, no. 5, 2004, pages 1643 - 52
BERGE ET AL., JOURNAL OF PHARMACEUTICAL SCIENCE, vol. 66, 1977, pages 1 - 19
BEVAN ET AL., MOL THER., vol. 19, no. 11, November 2011 (2011-11-01), pages 1971 - 1980
BOUR ET AL., BEHAV BRAIN RES., vol. 193, no. 2, 2008, pages 174 - 82
CAMPUZANO ET AL., SCIENCE, vol. 271.5254, 8 March 1996 (1996-03-08), pages 1423
CARILLO, H.; LIPMAN, D., SIAM J APPLIED MATH., vol. 48, 1988, pages 1073
CHIORINI ET AL., J. VIR., vol. 71, 1997, pages 6823 - 33
CHIORINI ET AL., J. VIR., vol. 73, 1999, pages 1309 - 1319
CLARK ET AL., HUM. GENE THER., vol. 10, 1999, pages 1031 - 1039
DENK; WADE-MARTINS, NEUROBIOL AGING, vol. 30, no. 1, 2009, pages 1 - 13
DEVEREUX, J. ET AL., NUCLEIC ACIDS RESEARCH, vol. t2, no. t, 1984, pages 387
DEVERMAN ET AL., NATURE BIOTECHNOLOGY, vol. 34, no. 2, 2016, pages 204 - 209
DREWS ET AL.: "Identification of evolutionary conserved. functional noncoding elements in the promoter region of the sodium channel gene SCN8A", MAMM GENOME, vol. 18, 2007, pages 723 - 731, XP019561869, DOI: doi:10.1007/s00335-007-9059-8
ELBASHIR SM ET AL., NATURE, vol. 411, 2001, pages 494 - 498
ELDER ET AL., MT SINAI J MED., vol. 77, no. 1, 2010, pages 69 - 81
G. S. BANKER: "Modern Pharmaceutics, Drugs and the Pharmaceutical Sciences", vol. 72, 1996, MARCEL DEKKER, NEW YORK, INC.
GILL ET AL., GENE THERAPY, vol. 8, 2001, pages 1539 - 1546
GRIMM ET AL., JOURNAL OF VIROLOGY, vol. 82, no. 12, 2008, pages 5887 - 5911
GROOTENDORST, BEHAV BRAIN RES., vol. 159, no. 1, 2005, pages 1 - 14
HALL; ROBERSON, BRAIN RES BULL., vol. 88, no. 1, 2012, pages 3 - 12
HEIM ET AL., CURRENT BIOLOGY, vol. 2, 1996, pages 178 - 182
HEIM ET AL., PROC. NATL. ACAD. SCI. USA, 1995
HEIM ET AL., SCIENCE, vol. 373, 1995, pages 663 - 664
HEINJE, G.: "Sequence Analysis in Molecular Biology", 1987, ACADEMIC PRESS
HUSAIN ET AL., GENE THERAPY, vol. 16, no. 7, 2009, pages 927 - 932
HUTTON, M. ET AL., NATURE, vol. 393, no. 6686, 1998, pages 702 - 5
J. MOL BIOL, vol. 266, no. 4, 1997, pages 814 - 830
JACKSON ET AL., FRONTIERS IN MOLECULAR NEUROSCIENCE, vol. 9, 2016, pages 154
JENNER, J NEUROL NEUROSURG PSYCHIATRY, 1989
KAJIGAYA ET AL., PROC. NAT'L. ACAD. SCI. USA, vol. 88, 1991, pages 4646 - 50
KENNETH I. BEMS: "FIELDS VIROLOGY", 1996, article "Parvoviridae: The Viruses and Their Replication"
KIMBAUER ET AL., VIR., vol. 219, 1996, pages 37 - 44
KNOUFF ET AL., J CLIN INVEST., vol. 103, no. 11, 1999, pages 1579 - 86
KOEPPEN, AMULF, J NEUROL SCI., vol. 303, no. 1-2, 15 April 2011 (2011-04-15), pages 1 - 12
KRISHNAMURTHY; SIGURDSSON, N BIOTECHNOL., vol. 28, no. 5, 2011, pages 511 - 7
MARQUES; OUTEIRO, CELL DEATH DIS, vol. 3, 2012, pages e350
MATHUPALA, EXPERT OPIN THER PAT., vol. 19, 2009, pages 137 - 140
MEYERS; MILLER, CABIOS, vol. 4, 1989, pages 11 - 17
MUSARO, FEBS J., vol. 280, no. 17, 2013, pages 4315 - 22
N PULICHERLA ET AL., MOLECULAR THERAPY, vol. 19, no. 6, 2011, pages 1070 - 1078
NAGESHWARAN; FESTENSTEIN, FRONTIERS IN NEUROLOGY, vol. 6, 2015
ONOS ET AL., BRAIN RES BULL., vol. 122, 2016, pages 1 - 11
O'REILLY ET AL.: "Baculovirus Expression Vectors, A Laboratory Manual", 1994, OXFORD UNIV. PRESS
PARKINSON ET AL., JOURNAL OF NEUROCHEMISTRY, vol. 126, no. 1, 2013, pages 103 - 117
PARKINSON ET AL., JOURNAL OFNEUROCHEMISTRY, vol. 126, no. 1, 2013, pages 103 - 117
PARR ET AL., NAT. MED., vol. 3, 1997, pages 1145 - 9
PASSINI; WOLFE, J. VIROL., 2001, pages 12382 - 12392
POWELL ET AL.: "Viral Expression Cassette Elements to Enhance Transgene Target Specificity and Expression in Gene Therapy", DISCOV. MED, vol. 19, no. 102, 2015, pages 49 - 57, XP055272358
PULICHERL ET AL., MOLECULAR THERAPY, vol. 19, no. 6, 2011, pages 1070 - 1078
RAYMOND ET AL.: "Expression of Alternatively Spliced Sodium Channel a-subunit genes", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 279, no. 44, 2004, pages 46234 - 46241
RUFFING ET AL., J. VIR., vol. 66, 1992, pages 6922 - 30
RUTLEDGE ET AL., J. VIR., vol. 72, 1998, pages 309 - 319
SAMULSKI ET AL., J. VIR., vol. 63, 1989, pages 3822 - 8
See also references of EP3662060A2
SHEEHAN B, THER ADV NEUROL DISORD., vol. 5, no. 6, 2012, pages 349 - 358
SRIVASTAVA ET AL., J. VIR., vol. 45, 1983, pages 555 - 64
STILWELL; SAMULSKI ET AL., BIOTECHNIQUES, vol. 34, 2003, pages 148 - 150
SULLIVAN ET AL., J BIOL CHEM., vol. 272, no. 29, 1997, pages 17972 - 80
SULLIVAN ET AL., J CLIN INVEST., vol. 102, no. 1, 1998, pages 130 - 5
T. E. CREIGHTON: "Proteins: Structure and Molecular Properties", 1983, W.H. FREEMAN & CO., pages: 79 - 86
VELDWIJK ET AL., MOL. THER., vol. 6, 2002, pages 272 - 278
VINCENT ET AL., NEUROMOLECULAR MEDICINE, vol. 6, 2004, pages 79 - 85
WANG ET AL., J NEUROSCI., vol. 22, 2002, pages 6920 - 6928
WU ET AL., J. VIR., vol. 74, 2000, pages 8635 - 47
XU ET AL., GENE THERAPY, vol. 8, 2001, pages 1323 - 1332
YOSHIYAMA ET AL., NEURON, vol. 53, no. 3, 2007, pages 337 - 51
YU ET AL., MOLECULAR PAIN, vol. 7, 2011, pages 63
ZHAO ET AL., VIR., vol. 272, 2000, pages 382 - 93

Cited By (105)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12472145B2 (en) 2010-07-26 2025-11-18 Board Of Regents, The University Of Texas System Methods for inducing an immune response via buccal and/or sublingual administration of a vaccine
US12123002B2 (en) 2014-11-14 2024-10-22 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11542506B2 (en) 2014-11-14 2023-01-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US12472244B2 (en) 2015-03-25 2025-11-18 Board Of Regents, The University Of Texas System Immunogenic compositions and uses thereof
US12440550B2 (en) 2015-03-25 2025-10-14 Board Of Regents, The University Of Texas System Immunogenic compositions and uses thereof
US12138318B2 (en) 2015-05-11 2024-11-12 Ucl Business Ltd Capsid
US10414803B2 (en) 2015-05-11 2019-09-17 Ucl Business Plc Capsid
US12071627B2 (en) 2016-08-23 2024-08-27 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11781145B2 (en) 2016-08-23 2023-10-10 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11993777B2 (en) 2016-08-23 2024-05-28 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12264317B2 (en) 2016-08-23 2025-04-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11525139B2 (en) 2016-08-23 2022-12-13 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US11865188B2 (en) 2017-04-14 2024-01-09 National Taiwan University Gene therapy for AADC deficiency
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
US12409235B2 (en) 2017-04-14 2025-09-09 National Taiwan University Gene therapy for AADC deficiency
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US12146150B2 (en) 2017-09-29 2024-11-19 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
US11993790B2 (en) 2017-10-03 2024-05-28 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
US12049626B2 (en) 2017-10-03 2024-07-30 Prevail Therapeutics, Inc. Gene therapy for neurodegenerative disorders
US12116589B2 (en) 2017-10-16 2024-10-15 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
US12054724B2 (en) 2018-04-10 2024-08-06 President And Fellows Of Harvard College AAV vectors encoding clarin-1 or GJB2 and uses thereof
WO2019222329A1 (en) * 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019246480A1 (en) 2018-06-21 2019-12-26 The Board Of Regents Of The University Of Texas System Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy
US12398181B2 (en) 2018-07-11 2025-08-26 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of agents across the blood-brain barrier
US11518787B2 (en) 2018-07-11 2022-12-06 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of agents across the blood-brain barrier
US12049648B2 (en) 2018-09-26 2024-07-30 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
AU2019346655B2 (en) * 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
US12467046B2 (en) 2018-10-02 2025-11-11 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
WO2020077165A1 (en) * 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020198737A1 (en) * 2019-03-28 2020-10-01 The General Hospital Corporation Engineered adeno-associated (aav) vectors for transgene expression
EP3952923A4 (en) * 2019-04-10 2023-10-18 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
CN113853209B (zh) * 2019-04-29 2024-12-13 宾夕法尼亚州大学信托人 新型aav衣壳和含有其的组合物
EP3976077A4 (en) * 2019-04-29 2023-08-30 The Trustees of The University of Pennsylvania NEW AAV CAPSIDS AND COMPOSITIONS CONTAINING THEM
CN113853209A (zh) * 2019-04-29 2021-12-28 宾夕法尼亚州大学信托人 新型aav衣壳和含有其的组合物
WO2020223276A1 (en) * 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tauopathy
JP2022530544A (ja) * 2019-04-29 2022-06-29 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規ａａｖカプシドおよびそれを含む組成物
EP3962507A4 (en) * 2019-04-29 2023-10-11 The Trustees of The University of Pennsylvania NOVEL AAV CAPSIDS AND COMPOSITIONS CONTAINING SAME
WO2020223296A1 (en) * 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
JP7644026B2 (ja) 2019-04-29 2025-03-11 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規ａａｖカプシドおよびそれを含む組成物
JP2022530656A (ja) * 2019-04-29 2022-06-30 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 新規ａａｖカプシドおよびそれを含む組成物
US12497608B2 (en) 2019-05-22 2025-12-16 The Brigham And Women's Hospital, Inc. Gene therapy for Alzheimer's disease
JP2022533451A (ja) * 2019-05-24 2022-07-22 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド アルツハイマー病のための遺伝子治療
JP7686573B6 (ja) 2019-05-24 2025-07-04 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド アルツハイマー病のための遺伝子治療
WO2020242892A1 (en) * 2019-05-24 2020-12-03 The Brigham And Women's Hospital, Inc. Gene therapy for alzheimer's disease
US20220235374A1 (en) * 2019-05-24 2022-07-28 The Brigham And Women`S Hospital, Inc. Gene therapy for alzheimer`s disease
JP7686573B2 (ja) 2019-05-24 2025-06-02 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド アルツハイマー病のための遺伝子治療
EP3999120A4 (en) * 2019-07-04 2024-02-21 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
WO2021025995A1 (en) * 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP4022073A4 (en) * 2019-08-29 2023-12-20 The Board Of Trustees Of The Leland Stanford Junior University ADENO-ASSOCIATED VIRAL VECTORS TO CROSS THE HUMAN BLOOD-BRAIN BARRIER
WO2021042073A1 (en) 2019-08-29 2021-03-04 Dorian Therapeutics, Inc. Methods and compositions for modulating cellular aging
CN114667349A (zh) * 2019-09-09 2022-06-24 马萨诸塞眼科耳科诊所 用于调节腺相关病毒(aav)和aav受体(aavr)之间的相互作用以改变aav生物分布的方法和组合物
AU2020391518B2 (en) * 2019-11-25 2025-02-13 Cornell University APOE gene therapy
WO2021108809A1 (en) * 2019-11-25 2021-06-03 Cornell University Apoe gene therapy
CN114829390A (zh) * 2019-11-25 2022-07-29 康奈尔大学 Apoe基因疗法
JP2023502515A (ja) * 2019-11-25 2023-01-24 コーネル・ユニバーシティー Ａｐｏｅ遺伝子療法
US12491267B2 (en) 2019-11-29 2025-12-09 The Brigham And Women's Hospital, Inc. Gene therapy for neurodegenerative disorders
EP4085144A4 (en) * 2019-12-19 2024-03-27 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF FRIEDREICH’S ATAXIA
US11981705B2 (en) 2020-01-10 2024-05-14 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
US12478691B2 (en) 2020-02-13 2025-11-25 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
US11759531B2 (en) 2020-02-13 2023-09-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
US11807867B2 (en) 2020-02-21 2023-11-07 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
US12077773B2 (en) 2020-02-21 2024-09-03 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2021168216A1 (en) 2020-02-21 2021-08-26 The Board Of Regents Of The University Of Texas System Crispr/cas9 correction of mutations in dystrophin exons 43, 45 and 52
US12305191B2 (en) 2020-02-21 2025-05-20 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2021205028A1 (en) 2020-04-09 2021-10-14 University Of Sheffield Gene therapy treatment
WO2021214443A1 (en) 2020-04-20 2021-10-28 Synpromics Limited Regulatory nucleic acid sequences
US11859200B2 (en) 2020-05-13 2024-01-02 Voyager Therapeutics, Inc. AAV capsids with increased tropism to brain tissue
EP4179098A4 (en) * 2020-07-08 2024-10-23 Baylor College Of Medicine GENE THERAPY FOR STXBP1 ENCEPHALOPATHY
EP4591708A2 (en) 2020-07-08 2025-07-30 Baylor College of Medicine Gene therapy for stxbp1 encephalopathy
US11634742B2 (en) 2020-07-27 2023-04-25 Anjarium Biosciences Ag Compositions of DNA molecules, methods of making therefor, and methods of use thereof
JP2023535620A (ja) * 2020-07-30 2023-08-18 ケンブリッジ エンタープライズ リミテッド 組成物および方法
WO2022023773A1 (en) * 2020-07-30 2022-02-03 Cambridge Enterprise Limited Composition and method
WO2022049385A1 (en) 2020-09-04 2022-03-10 Asklepios Biopharmaceutical, Inc. Regulatory nucleic acid sequences
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
WO2022129609A3 (en) * 2020-12-18 2022-09-15 Ac Immune Sa Antibody delivery
WO2022136655A1 (en) * 2020-12-23 2022-06-30 Boehringer Ingelheim International Gmbh Viral capsid proteins with specificity to heart tissue cells
WO2022165538A1 (en) * 2021-02-01 2022-08-04 The Regents Of The University Of California Viral delivery of therapeutics to the central nervous system
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022236008A1 (en) * 2021-05-07 2022-11-10 Board Of Regents, The University Of Texas System Methods and compositions for transport, storage, and delivery of adeno-associated viral vector and other molecules
WO2023283649A1 (en) 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
US12116385B2 (en) 2021-10-08 2024-10-15 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US12331081B2 (en) 2021-10-08 2025-06-17 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US12331082B2 (en) 2021-10-08 2025-06-17 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
US12296025B2 (en) 2021-11-02 2025-05-13 Voyager Therapeutics, Inc. AAV capsid variants and uses thereof
US12419969B2 (en) 2021-11-02 2025-09-23 Voyager Therapeutics, Inc. AAV capsid variants and uses thereof
WO2023111580A1 (en) 2021-12-16 2023-06-22 University Of Dundee Targeted degradation of alpha-synuclein
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
WO2023152504A1 (en) 2022-02-10 2023-08-17 University Of Dundee An affinity-directed phosphatase system for targeted protein dephosphorylation
WO2024011112A1 (en) * 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024079292A1 (en) 2022-10-14 2024-04-18 University Of Sheffield Gene therapy treatment
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2025049993A1 (en) 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025114699A1 (en) 2023-11-30 2025-06-05 Cytomos Limited Cell selection
WO2025128817A1 (en) * 2023-12-15 2025-06-19 Genzyme Corporation Artificial micrornas targeting tau
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Also Published As

Publication number Publication date
CA3071978A1 (en) 2019-02-07
KR102850887B1 (ko) 2025-08-28
TWI832036B (zh) 2024-02-11
MX2020001187A (es) 2020-10-05
US11512327B2 (en) 2022-11-29
EP3662060A2 (en) 2020-06-10
TWI722310B (zh) 2021-03-21
WO2019028306A3 (en) 2019-03-07
JP2023052805A (ja) 2023-04-12
JP2020533959A (ja) 2020-11-26
US20230304032A1 (en) 2023-09-28
TW201919714A (zh) 2019-06-01
AU2018311069A1 (en) 2020-02-20
EP3808849A1 (en) 2021-04-21
JP7221275B2 (ja) 2023-02-13
US20210163985A1 (en) 2021-06-03
AU2018311069A2 (en) 2020-07-09
US12305189B2 (en) 2025-05-20
KR20200044793A (ko) 2020-04-29
TW202133888A (zh) 2021-09-16
CN111448308A (zh) 2020-07-24

Similar Documents

Publication Publication Date Title
US12305189B2 (en) Compositions and methods for delivery of AAV
US20210371470A1 (en) Compositions and methods for delivery of aav
US20210230632A1 (en) Compositions and methods for delivery of aav
US11759506B2 (en) AADC polynucleotides for the treatment of Parkinson's disease
US20210277418A1 (en) Aav variants with enhanced tropism
US20210355454A1 (en) Systems and methods for producing gene therapy formulations
US20220064671A1 (en) Methods and systems for producing aav particles
US12319929B2 (en) Compositions and methods for the treatment of Parkinson's disease
US20220281922A1 (en) Aav variants with enhanced tropism
WO2020223280A1 (en) Aav variants with enhanced tropism
WO2021226167A1 (en) Aav variants from second round libraries with tropism for central nervous system tissues
EP3861113A1 (en) Methods for measuring the titer and potency of viral vector particles
HK40050637A (en) Compositions and methods for delivery of aav

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 18762419

Country of ref document: EP

Kind code of ref document: A2

ENP Entry into the national phase

Ref document number: 2020505424

Country of ref document: JP

Kind code of ref document: A

ENP Entry into the national phase

Ref document number: 3071978

Country of ref document: CA

NENP Non-entry into the national phase

Ref country code: DE

ENP Entry into the national phase

Ref document number: 2018311069

Country of ref document: AU

Date of ref document: 20180803

Kind code of ref document: A

ENP Entry into the national phase

Ref document number: 2018762419

Country of ref document: EP

Effective date: 20200303