SG10201912977RA - Aav vectors for retinal and cns gene therapy - Google Patents

Aav vectors for retinal and cns gene therapy

Info

Publication number
SG10201912977RA
SG10201912977RA SG10201912977RA SG10201912977RA SG10201912977RA SG 10201912977R A SG10201912977R A SG 10201912977RA SG 10201912977R A SG10201912977R A SG 10201912977RA SG 10201912977R A SG10201912977R A SG 10201912977RA SG 10201912977R A SG10201912977R A SG 10201912977RA
Authority
SG
Singapore
Prior art keywords
retinal
gene therapy
aav vectors
cns gene
cns
Prior art date
Application number
SG10201912977RA
Inventor
Abraham Scaria
Jennifer Sullivan
Lisa Stanek
Original Assignee
Genzyme Corp
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=53189202&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=SG10201912977R(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of SG10201912977RA publication Critical patent/SG10201912977RA/en

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
    • 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
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular 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
    • 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
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2750/14022—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
    • C12N2750/14023—Virus like particles [VLP]
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Psychology (AREA)
  • Cardiology (AREA)
  • Hospice & Palliative Care (AREA)
SG10201912977RA 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy SG10201912977RA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
US201562114575P 2015-02-10 2015-02-10

Publications (1)

Publication Number Publication Date
SG10201912977RA true SG10201912977RA (en) 2020-02-27

Family

ID=53189202

Family Applications (3)

Application Number Title Priority Date Filing Date
SG10201809739QA SG10201809739QA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy
SG11201607991WA SG11201607991WA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy
SG10201912977RA SG10201912977RA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Family Applications Before (2)

Application Number Title Priority Date Filing Date
SG10201809739QA SG10201809739QA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy
SG11201607991WA SG11201607991WA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Country Status (31)

Country Link
US (2) US10982228B2 (en)
EP (2) EP3913061A1 (en)
JP (3) JP6741591B2 (en)
KR (2) KR20240032156A (en)
CN (2) CN114231563B (en)
AU (3) AU2015252797C1 (en)
CA (1) CA2946593A1 (en)
CL (2) CL2016002713A1 (en)
CR (1) CR20160555A (en)
DK (1) DK3137497T3 (en)
DO (1) DOP2016000280A (en)
EA (1) EA034355B1 (en)
ES (1) ES2879636T3 (en)
GT (1) GT201600227A (en)
HR (1) HRP20211024T1 (en)
HU (1) HUE054768T2 (en)
IL (2) IL292951B2 (en)
LT (1) LT3137497T (en)
MX (2) MX2016014220A (en)
MY (1) MY188987A (en)
PE (1) PE20170260A1 (en)
PH (1) PH12016502046A1 (en)
PL (1) PL3137497T4 (en)
PT (1) PT3137497T (en)
RS (1) RS62078B1 (en)
SG (3) SG10201809739QA (en)
SI (1) SI3137497T1 (en)
TW (1) TWI686476B (en)
UY (1) UY36106A (en)
WO (1) WO2015168666A2 (en)
ZA (1) ZA201606763B (en)

Families Citing this family (160)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
CN107083400A (en) 2009-05-02 2017-08-22 建新公司 The gene therapy of nerve degenerative diseases
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
JP2013533847A (en) 2010-04-23 2013-08-29 ユニバーシティ オブ マサチューセッツ AAV-based treatment of cholesterol-related disorders
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
CA3049237C (en) 2010-04-23 2024-06-11 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA2870511C (en) 2011-04-21 2023-08-08 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
SG194583A1 (en) 2011-04-22 2013-12-30 Univ California Adeno-associated virus virions with variant capsid and methods of use thereof
BR112015028605A8 (en) * 2013-05-15 2019-12-24 Univ Minnesota use of a composition and use of an immunosuppressant and a composition
JP6600624B2 (en) 2013-05-31 2019-10-30 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Adeno-associated virus mutant and method of use thereof
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
CA2942776C (en) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter
NZ724508A (en) 2014-03-18 2024-05-31 Univ Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
MY183472A (en) * 2014-05-14 2021-02-19 Esteve Labor Dr Adenoassociated virus vectors for the treatment of lysosomal storage disorders
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
RS62811B1 (en) * 2015-02-10 2022-02-28 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
MX2017010369A (en) 2015-02-10 2017-12-14 Genzyme Corp VARIANT RNAi.
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EA201791939A1 (en) 2015-03-02 2018-01-31 Адверум Байотекнолоджиз, Инк. COMPOSITIONS AND METHODS OF INTRAVITREAL DELIVERY OF POLYNUCLEOTIDES IN RETCHEMBALS
US10646588B2 (en) * 2015-03-11 2020-05-12 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services RP2 and RPGR vectors for treating X-linked retinitis pigmentosa
WO2016154344A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
TWI707951B (en) 2015-04-08 2020-10-21 美商健臻公司 Production of oversized adeno-associated vectors
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions and uses thereof
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CN108137655B (en) 2015-09-28 2022-04-22 北卡罗来纳-查佩尔山大学 Methods and compositions for antibody-evading viral vectors
CA3002982A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
AU2016372035B2 (en) * 2015-12-15 2023-03-30 Genzyme Corporation Adeno-associated viral vectors for treating mucolipidosis type ii
WO2017136536A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
EP3413928B1 (en) 2016-02-12 2022-04-20 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017147477A1 (en) * 2016-02-26 2017-08-31 University Of Florida Research Foundation, Inc. Aav heparin mutants that display significantly improved eye and brain transduction
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
CN107287238B (en) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 Gene vector and gene therapy medicine for treating Leber congenital amaurosis 2 type disease
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
US11951121B2 (en) * 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
EP3481958A4 (en) * 2016-07-08 2019-12-25 The Trustees of The University of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
EP3485005A1 (en) 2016-07-12 2019-05-22 The University Of Manchester Gene therapy
CN116286986A (en) 2016-07-29 2023-06-23 加利福尼亚大学董事会 Adeno-associated virus virions with variant capsids and methods of use thereof
CA3033125A1 (en) * 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
CN109996880A (en) * 2016-08-18 2019-07-09 加利福尼亚大学董事会 CRISPR-CAS genome project based on modularization AAV delivery system
WO2018035457A1 (en) * 2016-08-19 2018-02-22 Calimmune, Inc. Methods and compositions for treating canine conditions using recombinant self-complementary adeno-associated virus
KR20190055086A (en) * 2016-08-19 2019-05-22 칼리뮨, 인코포레이티드 Methods and compositions for treating conditions using recombinant self-complementary adeno-associated viruses
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
KR20190057110A (en) 2016-09-28 2019-05-27 코바, 인크. Therapeutic MOTS-C related peptides
WO2018064624A1 (en) 2016-09-29 2018-04-05 University Of Florida Research Foundation, Incorporated Aavrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
WO2018071831A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
AU2017371043B2 (en) 2016-12-07 2022-12-15 University Of Florida Research Foundation, Incorporated IL-1Ra cDNAs
WO2018145009A1 (en) * 2017-02-06 2018-08-09 University Of Tennessee Research Foundation Dna-zyme based methods & compositions for treating huntington's disease
US11117930B2 (en) 2017-02-23 2021-09-14 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
JOP20190200A1 (en) 2017-02-28 2019-08-27 Univ Pennsylvania Compositions useful in treatment of spinal muscular atrophy
WO2018191450A2 (en) 2017-04-14 2018-10-18 National Taiwan University Hospital Gene therapy for aadc deficiency
WO2018200419A1 (en) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Viral vectors comprising engineered aav capsids and compositions containing the same
CN111108198A (en) * 2017-05-05 2020-05-05 沃雅戈治疗公司 Compositions and methods for treating huntington's disease
WO2018208972A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
WO2018209154A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (app)-dependence of viruses
US20230137562A1 (en) 2017-06-07 2023-05-04 Adrx, Inc. Tau aggregation inhibitors
BR112019019015A2 (en) * 2017-06-30 2020-04-14 Univ California viruses of adeno-associated viruses with variant capsids and their methods of use
JP7221275B2 (en) * 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Compositions and methods for delivering AAV
MX2020001997A (en) 2017-08-28 2020-07-20 Univ California Adeno-associated virus capsid variants and methods of use thereof.
US20200377887A1 (en) * 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CA3075643A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts Sod1 dual expression vectors and uses thereof
JP7248658B2 (en) 2017-09-22 2023-03-29 ジェンザイム・コーポレーション Variant RNAi
JP7420710B2 (en) 2017-10-20 2024-01-23 アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） A method of expressing a polynucleotide of interest in cone photoreceptor cells of a subject comprising subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector.
US11464803B2 (en) 2017-11-14 2022-10-11 Arcellx, Inc. D-domain containing polypeptides and uses thereof
KR20200087176A (en) 2017-11-14 2020-07-20 아르셀엑스, 인크. Multifunctional immune cell therapy
CR20200210A (en) * 2017-11-15 2020-09-23 Friedrich Miescher Institute For Biomedical Res Primate retinal pigment epithelium cell-specific promoter
EP3710590B1 (en) * 2017-11-15 2024-07-17 The Regents of The University of Michigan Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
WO2019102381A1 (en) 2017-11-21 2019-05-31 Casebia Therapeutics Llp Materials and methods for treatment of autosomal dominant retinitis pigmentosa
CN108085316B (en) * 2017-12-25 2021-02-09 中国人民解放军第四军医大学 Method for promoting in-vitro amplification of mouse retina precursor cells by inhibiting micro ribonucleic acid
EP4186921A1 (en) * 2018-03-23 2023-05-31 The Trustees of Columbia University in the City of New York Gene editing for autosomal dominant diseases
MX2020010465A (en) 2018-04-03 2021-01-08 Virus vectors for targeting ophthalmic tissues.
JP7406677B2 (en) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド Viral vectors that evade antibodies
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
US20210230229A1 (en) * 2018-05-15 2021-07-29 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
WO2019222328A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
US20210207147A1 (en) * 2018-06-01 2021-07-08 University Of Florida Research Foundation, Incorporated Compositions and methods for treatment of dominant retinitis pigmentosa
TW202022116A (en) 2018-08-03 2020-06-16 美商健臻公司 Variant rnai against alpha-synuclein
CN109022487A (en) * 2018-08-15 2018-12-18 上海市第人民医院 A kind of genophore for expressing p65 and its gene therapy medicament for treating retinal ganglion cells degeneration
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
SG11202103151RA (en) 2018-09-28 2021-04-29 Voyager Therapeutics Inc Frataxin expression constructs having engineered promoters and methods of use thereof
CA3115782A1 (en) * 2018-10-10 2020-04-16 Wisconsin Alumni Research Foundation Kir 7.1 gene therapy vectors and methods of using the same
SG11202103614RA (en) * 2018-10-12 2021-05-28 Genzyme Corp Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (en) 2018-10-15 2020-05-29 Novartis Ag TREM2 STABILIZING ANTIBODIES
US20230061069A1 (en) * 2018-11-13 2023-03-02 Jayandharan Giridhara Rao A process for producing a plurality of sumoylation target-site modified aav vector and product thereof
WO2020140007A1 (en) * 2018-12-28 2020-07-02 University Of Rochester Gene therapy for best1 dominant mutations
JP7531495B2 (en) * 2019-01-04 2024-08-09 ウルトラジェニックス ファーマシューティカル インコーポレイテッド Gene therapy constructs for treating Wilson's disease
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
US20220133909A1 (en) * 2019-01-23 2022-05-05 University Of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
CA3127336A1 (en) 2019-01-28 2020-08-06 Cohbar, Inc. Therapeutic peptides
WO2020180951A1 (en) * 2019-03-04 2020-09-10 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
US20220125948A1 (en) 2019-03-04 2022-04-28 Duke University Compositions and methods for the diagnosis and treatment of retinopathies
US20220175961A1 (en) * 2019-03-08 2022-06-09 Universite Paris-Saclay Improved therapeutic method for rare ocular diseases by gene replacement
MX2021011468A (en) 2019-03-21 2021-12-15 Recombinant adeno-associated virus vectors.
KR20220006527A (en) * 2019-04-10 2022-01-17 프리베일 테라퓨틱스, 인크. Gene therapy for lysosomal disorders
EP3966227A1 (en) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
US20220249700A1 (en) * 2019-05-13 2022-08-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Polymer-based implant for retinal therapy and methods of making and using the same
KR20220009427A (en) * 2019-05-17 2022-01-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Improved Delivery of Gene Therapy Vectors to Retinal Cells Using Glycoside Hydrolases
EP3980447A4 (en) * 2019-06-10 2023-07-26 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
US11141425B2 (en) * 2019-06-27 2021-10-12 University Of Florida Research Foundation, Incorporated Enhancing AAV-mediated transduction of ocular tissues with hyaluronic acid
US11446084B2 (en) 2019-07-12 2022-09-20 Neuralink Corp. Laser drilling of pia mater
EP3999119B1 (en) * 2019-07-15 2024-09-11 Meiragtx UK II Limited Modified aav capsid proteins for treatment of arthritic disease
CN110295151A (en) * 2019-07-17 2019-10-01 博鑫仪器(天津)有限公司 A kind of gland dependovirus virus recombinant plasmid, recombinant virus and construction method
US20220281930A1 (en) * 2019-08-23 2022-09-08 Duke University Compositions and methods for the treatment of pathological pain and itch
EP4025258A4 (en) * 2019-09-03 2023-09-06 University of Cincinnati Methods and compositions for the treatment of als
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20220105158A (en) * 2019-10-23 2022-07-26 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Compositions for DRG-specific reduction of transgene expression
CN113025618B (en) * 2019-12-24 2024-02-06 朗信启昇(苏州)生物制药有限公司 Gene therapy scheme and application of X-linked hereditary retinal splitting disease
JP2023513004A (en) 2020-01-29 2023-03-30 ジェンザイム・コーポレーション Modified adeno-associated virus capsid proteins for ophthalmic gene therapy and methods of their use
AU2021217222A1 (en) * 2020-02-07 2022-09-29 The Trustees Of Columbia University In The City Of New York Reprogramming the metabolome to delay onset or treat neurodegeneration
CA3164321A1 (en) * 2020-02-18 2021-08-26 The University Of North Carolina At Chapel Hill Aav capsid-promoter interactions and cell selective gene expression
EP4110913A4 (en) * 2020-02-28 2024-07-24 Univ Massachusetts Oligonucleotides for prnp modulation
BR112022017970A2 (en) * 2020-03-11 2022-10-25 Massachusetts Eye & Ear Infirmary GENE THERAPY FOR NMNAT1 ASSOCIATED RETINA DEGENERATION
AU2021248577A1 (en) * 2020-03-31 2022-09-29 Sichuan Univeristy AAV capsids variants and uses thereof
IL297200A (en) 2020-04-15 2022-12-01 Voyager Therapeutics Inc Tau binding compounds
TW202208397A (en) 2020-05-13 2022-03-01 美商航海家醫療公司 Redirection of tropism of aav capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
JP2023529503A (en) * 2020-06-13 2023-07-10 オクロジェネクス インコーポレイテッド AAV-mediated gene transfer for retinopathy
CN113952472A (en) * 2020-07-21 2022-01-21 英斯培瑞有限公司 Compositions and methods for treating ocular diseases
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
MX2023000815A (en) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency.
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
US20230257431A1 (en) 2020-08-05 2023-08-17 Spacecraft Seven, Llc Csrp3 (cysteine and glycine rich protein 3) gene therapy
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (pkp2) gene therapy using aav vector
MX2023002016A (en) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Adeno-associated virus vectors for treatment of rett syndrome.
US20230330267A1 (en) * 2020-09-04 2023-10-19 Ohio State Innovation Foundation Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
WO2022087238A1 (en) * 2020-10-21 2022-04-28 Wisconsin Alumni Research Foundation Anti-apoptotic vector and method of using the same
US20220160825A1 (en) * 2020-11-25 2022-05-26 The Penn State Research Foundation Brain repair after traumatic brain injury through neurod1-mediated astrocyte-to-neuron conversion
WO2022178410A1 (en) * 2021-02-22 2022-08-25 The Regents Of The University Of Colorado, A Body Corporate Viral vector-based gene therapy for ocular conditions
CN115044614B (en) * 2021-03-09 2023-10-20 上海目镜生物医药科技有限公司 Modified vector of AAV-8 serotype for gene targeting and expression, construction method and application thereof
WO2022231950A1 (en) * 2021-04-26 2022-11-03 Grace Science, Llc Compositions and methods for treating ngyl1 deficiency
CN115997012B (en) * 2021-09-06 2024-03-08 辉大(上海)生物科技有限公司 Treatment of RPE65 related eye diseases and disorders
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
TW202325850A (en) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc and gdnf polynucleotides and their uses in treating parkinson's disease
CN118660913A (en) 2022-02-21 2024-09-17 上海瑞宏迪医药有限公司 VEGF binding molecules and their medical uses
WO2023183623A1 (en) * 2022-03-25 2023-09-28 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
WO2023211857A1 (en) 2022-04-25 2023-11-02 Lineage Cell Therapeutics, Inc. Methods and compositions for treating vision loss
WO2023212521A2 (en) * 2022-04-25 2023-11-02 Myrtelle Inc. Treatments of disorders of myelin
WO2023214346A1 (en) 2022-05-06 2023-11-09 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023240220A1 (en) * 2022-06-09 2023-12-14 The University Of North Carolina At Chapel Hill Aav-sgsh vectors for treatment of mucopolysaccharidosis iiia
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
EP4303226A1 (en) 2022-07-08 2024-01-10 Ospedale San Raffaele S.r.l. Transgene cassettes and epigenetic silencers for the treatment of disorders
WO2024008950A1 (en) 2022-07-08 2024-01-11 Ospedale San Raffaele S.R.L. Transgene cassettes
WO2024015794A2 (en) * 2022-07-12 2024-01-18 The Research Institute At Nationwide Children's Hospital Adeno-associated virus gene therapy products and methods
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4692147A (en) 1980-04-02 1987-09-08 Medtronic, Inc. Drug administration device
US5735815A (en) 1993-07-26 1998-04-07 Sentinel Medical, Inc. Method of using fluid jet surgical cutting tool
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6225291B1 (en) 1997-04-21 2001-05-01 University Of Florida Rod opsin mRNA-specific ribozyme compositions and methods for the treatment of retinal diseases
US6042579A (en) 1997-04-30 2000-03-28 Medtronic, Inc. Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP2002516295A (en) 1998-05-27 2002-06-04 アビジェン， インコーポレイテッド Convective increased delivery of AAV vectors
DK1916258T3 (en) 1999-08-09 2014-07-28 Genzyme Corp Increasing expression of a single-stranded heterologous nucleotide sequence from recombinant viral vectors in such a design of the sequence to form intra-strand base pairs
DE60117550T2 (en) 2000-06-01 2006-12-07 University Of North Carolina At Chapel Hill DOUBLE-SIDED PARVOVIRUS VECTORS
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
GB2389791B (en) 2002-04-30 2006-12-13 Steven Gill Implantable drug delivery pump
SI1496944T1 (en) * 2002-05-01 2009-02-28 Univ Florida Improved raav expression systems for genetic modification of specific capsid proteins
WO2004099423A1 (en) * 2003-05-01 2004-11-18 University Of Florida Research Foundation, Inc. Vp2-modified raav vectors and uses thereof
EP2281877A3 (en) 2003-05-21 2011-06-01 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
WO2004112727A2 (en) * 2003-06-19 2004-12-29 Avigen, Inc. Aav virions with decreased immunoreactivity and uses therefor
SI1807009T1 (en) 2004-10-05 2015-04-30 Genzyme Corporation Stepped cannula
DK2359867T3 (en) * 2005-04-07 2015-01-05 Univ Pennsylvania A method for increasing an AAV vector function
CA2619882C (en) 2005-08-23 2015-05-26 The Regents Of The University Of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
ES2618787T5 (en) 2006-04-25 2022-10-21 Univ California Administration of growth factors for the treatment of CNS disorders
US20070259031A1 (en) 2006-04-26 2007-11-08 The Regents Of The University Of California Compositions and methods for convection enhanced delivery of high molecular weight neurotherapeutics
US20090317417A1 (en) 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
AU2008254768C1 (en) 2007-05-17 2015-03-12 Medgenesis Therapeutix Inc. Convention-enhanced delivery catheter with removable stiffening member
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
CA2751266A1 (en) 2009-01-29 2010-08-05 Krystof Bankiewicz Methods for distributing high levels of therapeutic agent throughout the cortex to treat neurological disorders
EP2396343B1 (en) * 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
PL3067417T3 (en) 2009-06-16 2019-02-28 Genzyme Corporation Improved methods for purification of recombinant aav vectors
CA3049237C (en) 2010-04-23 2024-06-11 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
SG194583A1 (en) 2011-04-22 2013-12-30 Univ California Adeno-associated virus virions with variant capsid and methods of use thereof
TWI698240B (en) * 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 Treatment of amd using aav sflt-1

Also Published As

Publication number Publication date
MY188987A (en) 2022-01-17
JP2017518271A (en) 2017-07-06
SG11201607991WA (en) 2016-10-28
IL248102B (en) 2022-07-01
MX2016014220A (en) 2017-02-06
US20210189430A1 (en) 2021-06-24
BR112016025263A2 (en) 2018-02-20
CR20160555A (en) 2017-01-27
SI3137497T1 (en) 2021-08-31
EP3137497A2 (en) 2017-03-08
AU2021200242B2 (en) 2023-09-21
IL292951B2 (en) 2024-10-01
CN114231563B (en) 2024-08-16
JP2023011812A (en) 2023-01-24
ZA201606763B (en) 2023-05-31
GT201600227A (en) 2018-01-22
US20170096683A1 (en) 2017-04-06
CA2946593A1 (en) 2015-11-05
AU2015252797B2 (en) 2020-10-15
JP2020164540A (en) 2020-10-08
EA034355B1 (en) 2020-01-30
AU2021200242A1 (en) 2021-03-18
US10982228B2 (en) 2021-04-20
CL2016002713A1 (en) 2017-08-11
HRP20211024T1 (en) 2021-10-01
IL248102A0 (en) 2016-11-30
CN106661591B (en) 2021-10-29
WO2015168666A2 (en) 2015-11-05
PH12016502046A1 (en) 2017-02-06
CL2018000170A1 (en) 2018-06-29
EP3913061A1 (en) 2021-11-24
JP6741591B2 (en) 2020-08-19
AU2021200242B9 (en) 2023-10-05
PL3137497T3 (en) 2021-10-25
CN114231563A (en) 2022-03-25
MX2022012490A (en) 2022-10-27
ES2879636T3 (en) 2021-11-22
SG10201809739QA (en) 2018-12-28
CN106661591A (en) 2017-05-10
WO2015168666A3 (en) 2015-12-23
JP7174011B2 (en) 2022-11-17
TWI686476B (en) 2020-03-01
UY36106A (en) 2015-11-30
IL292951A (en) 2022-07-01
DOP2016000280A (en) 2016-11-30
LT3137497T (en) 2021-07-26
PL3137497T4 (en) 2023-04-11
KR20240032156A (en) 2024-03-08
EA201692206A1 (en) 2017-03-31
TW201625792A (en) 2016-07-16
RS62078B1 (en) 2021-07-30
AU2015252797C1 (en) 2021-04-22
PT3137497T (en) 2021-07-12
PE20170260A1 (en) 2017-04-12
IL292951B1 (en) 2024-06-01
EP3137497B1 (en) 2021-04-07
KR20160147974A (en) 2016-12-23
DK3137497T3 (en) 2021-07-12
HUE054768T2 (en) 2021-09-28
AU2023285817A1 (en) 2024-01-18
AU2015252797A1 (en) 2016-11-17

Similar Documents

Publication Publication Date Title
ZA201606763B (en) Aav vectors for retinal and cns gene therapy
IL251468A0 (en) Aav-based gene therapy
HK1257801A1 (en) Intrathecal administration of adeno-associated-viral vectors for gene therapy
HK1248723B (en) Gene therapy
SG11201703193XA (en) Treatment for depression and depressive disorders
HK1258518A1 (en) Gene therapy for ocular disorders
GB201407322D0 (en) Gene therapy
LT3612237T (en) Gene therapy
PT3133923T (en) Aav-based gene therapy for multiple sclerosis
GB201707212D0 (en) Gene therapy for ciliopathies
GB201701968D0 (en) Gene therapy
PT3285871T (en) Vectors and methods for regenerative therapy
IL275613A (en) Gene therapy for eosinohilic disorders
IL263085A (en) Gene therapy
GB201513151D0 (en) Functional gene replacement therapy
GB201616821D0 (en) Gene therapy
GB201612105D0 (en) Gene therapy
GB201612104D0 (en) Gene therapy
GB201704634D0 (en) Gene therapy