SG10201912495SA - Compositions useful in treatment of spinal muscular atrophy - Google Patents

Compositions useful in treatment of spinal muscular atrophy

Info

Publication number
SG10201912495SA
SG10201912495SA SG10201912495SA SG10201912495SA SG10201912495SA SG 10201912495S A SG10201912495S A SG 10201912495SA SG 10201912495S A SG10201912495S A SG 10201912495SA SG 10201912495S A SG10201912495S A SG 10201912495SA SG 10201912495S A SG10201912495S A SG 10201912495SA
Authority
SG
Singapore
Prior art keywords
treatment
compositions useful
muscular atrophy
spinal muscular
spinal
Prior art date
Application number
SG10201912495SA
Inventor
James Wilson
Christian Hinderer
Nathan Katz
Qiang Wang
Original Assignee
Univ Pennsylvania
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
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG10201912495SA publication Critical patent/SG10201912495SA/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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
SG10201912495SA 2017-02-28 2018-02-27 Compositions useful in treatment of spinal muscular atrophy SG10201912495SA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762464756P 2017-02-28 2017-02-28
US201762515902P 2017-06-06 2017-06-06
US201862618437P 2018-01-17 2018-01-17

Publications (1)

Publication Number Publication Date
SG10201912495SA true SG10201912495SA (en) 2020-02-27

Family

ID=61800665

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201907474UA SG11201907474UA (en) 2017-02-28 2018-02-27 Compositions useful in treatment of spinal muscular atrophy
SG10201912495SA SG10201912495SA (en) 2017-02-28 2018-02-27 Compositions useful in treatment of spinal muscular atrophy

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG11201907474UA SG11201907474UA (en) 2017-02-28 2018-02-27 Compositions useful in treatment of spinal muscular atrophy

Country Status (18)

Country Link
US (2) US11578341B2 (en)
EP (1) EP3589739A2 (en)
JP (2) JP7037574B2 (en)
KR (1) KR20190123752A (en)
CN (1) CN110741089A (en)
AU (1) AU2018227442A1 (en)
BR (1) BR112019016444A2 (en)
CA (1) CA3055619A1 (en)
CL (1) CL2019002473A1 (en)
CO (1) CO2019009522A2 (en)
IL (1) IL268519A (en)
JO (1) JOP20190200A1 (en)
MA (1) MA47688A (en)
MX (1) MX2019010276A (en)
PE (1) PE20191261A1 (en)
PH (1) PH12019550149A1 (en)
SG (2) SG11201907474UA (en)
WO (1) WO2018160585A2 (en)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3528852A4 (en) 2016-10-20 2020-06-03 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
AU2018227440A1 (en) 2017-02-28 2019-08-29 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor
KR20210006327A (en) * 2018-02-27 2021-01-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Novel adeno-associated virus (AAV) vectors with reduced capsid deamidation and uses thereof
CN113646005A (en) * 2018-12-21 2021-11-12 宾夕法尼亚州大学信托人 Compositions for DRG-specific reduction of transgene expression
SG11202107325TA (en) * 2019-01-04 2021-08-30 Sangamo Therapeutics Inc Methods and compositions for the treatment of fabry disease
WO2021183469A1 (en) * 2020-03-09 2021-09-16 Amprion, Inc. Cerebrospinal fluid assay control solution
BR112022022212A2 (en) 2020-05-12 2022-12-13 Univ Pennsylvania COMPOSITIONS FOR SPECIFIC DRG REDUCTION OF TRANSGENE EXPRESSION
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
CN112852882A (en) * 2021-02-04 2021-05-28 中吉智药(南京)生物技术有限公司 System and method for producing AAV gene medicine by infecting insect cell with baculovirus
WO2023061499A1 (en) * 2021-10-15 2023-04-20 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating spinal muscular atrophy
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
CN114177346B (en) * 2021-12-24 2023-05-26 中国人民解放军军事科学院军事医学研究院 Hemostatic composition and hemostatic patch and application thereof

Family Cites Families (149)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
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
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0682717A4 (en) 1993-02-05 1997-04-23 Laporte Group Australia A slag defoaming composite.
PL310327A1 (en) 1993-02-12 1995-12-11 Univ Leland Stanford Junior Adjustable transcription of target genes and other biological processes
US6972193B1 (en) 1993-02-12 2005-12-06 Board Of Trustees Of Leland Stanford Junior University Regulated transcription of targeted genes and other biological events
US20020173474A1 (en) 1993-02-12 2002-11-21 President And Fellows Of Harvard College Methods & materials involving dimerization-mediated regulation of biological events
US5830462A (en) 1993-02-12 1998-11-03 President & Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
US6140120A (en) 1993-02-12 2000-10-31 Board Of Trustees Of Leland Stanford Jr. University Regulated transcription of targeted genes and other biological events
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US6150137A (en) 1994-05-27 2000-11-21 Ariad Pharmaceuticals, Inc. Immunosuppressant target proteins
US6476200B1 (en) 1994-06-27 2002-11-05 The Johns Hopkins University Mammalian proteins that bind to FKBP12 in a rapamycin-dependent fashion
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
CA2197793C (en) 1994-08-18 2007-04-24 Dennis A. Holt New multimerizing agents
US6133456A (en) 1994-08-18 2000-10-17 Ariad Gene Therapeutics, Inc. Synthetic multimerizing agents
US6150527A (en) 1994-08-18 2000-11-21 Ariad Pharmaceuticals, Inc. Synthetic multimerizing agents
US5786464C1 (en) 1994-09-19 2012-04-24 Gen Hospital Corp Overexpression of mammalian and viral proteins
US6326166B1 (en) 1995-12-29 2001-12-04 Massachusetts Institute Of Technology Chimeric DNA-binding proteins
EP0805819B1 (en) 1994-12-29 2012-02-08 Massachusetts Institute Of Technology Chimeric dna-binding proteins
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5811524A (en) 1995-06-07 1998-09-22 Idec Pharmaceuticals Corporation Neutralizing high affinity human monoclonal antibodies specific to RSV F-protein and methods for their manufacture and therapeutic 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
WO1996041865A1 (en) 1995-06-07 1996-12-27 Ariad Gene Therapeutics, Inc. Rapamcycin-based regulation of biological events
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
JP4936345B2 (en) 1996-02-28 2012-05-23 アリアド・ファーマシューティカルズ・インコーポレイテッド Synthetic derivatives of rapamycin as a multimerizing agent for chimeric proteins with immunophilin-derived domains
US6723531B2 (en) 1996-04-05 2004-04-20 The Salk Institute For Biological Studies Method for modulating expression of exogenous genes in mammalian systems, and products related thereto
WO1998002441A2 (en) 1996-07-12 1998-01-22 Ariad Pharmaceuticals, Inc. Non immunosuppressive antifungal rapalogs
ES2563643T3 (en) 1997-04-01 2016-03-15 Illumina Cambridge Limited Nucleic acid sequencing method
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6015709A (en) 1997-08-26 2000-01-18 Ariad Pharmaceuticals, Inc. Transcriptional activators, and compositions and uses related thereto
JP2003524368A (en) 1997-08-26 2003-08-19 アリアド ジーン セラピューティクス インコーポレイテッド Fusion protein comprising a dimerization domain, a trimerization domain or a tetramerization domain and a complementary heterologous transcriptional activation domain, a transcription repression domain, a DNA binding domain or a ligand binding domain
US6479653B1 (en) 1997-08-26 2002-11-12 Ariad Gene Therapeutics, Inc. Compositions and method for regulation of transcription
IL134643A0 (en) 1997-08-27 2001-04-30 Ariad Gene Therapeutics Inc Chimeric transcriptional activators and compositions and uses related thereto
JP2002508971A (en) 1998-01-15 2002-03-26 アリアド・ジーン・セラピューティクス・インコーポレーテッド Regulation of biological events using multimeric chimeric proteins
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
JP2002503667A (en) 1998-02-13 2002-02-05 プレジデント・アンド・フェローズ・オブ・ハーバード・カレッジ Novel dimerizing agents, their production and use
US6333318B1 (en) 1998-05-14 2001-12-25 The Salk Institute For Biological Studies Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto
EP1080218A1 (en) 1998-05-27 2001-03-07 University of Florida Method of preparing recombinant adeno-associated virus compositions by using an iodixanol gradient
US6258603B1 (en) 1998-06-17 2001-07-10 Rohm And Haas Company Ligands for modulating the expression of exogenous genes via an ecdysone receptor complex
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US7109317B1 (en) 1998-11-06 2006-09-19 President And Fellows Of Harvard College FK506-based regulation of biological events
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
US7067526B1 (en) 1999-08-24 2006-06-27 Ariad Gene Therapeutics, Inc. 28-epirapalogs
ES2219388T3 (en) 1999-08-24 2004-12-01 Ariad Gene Therapeutics, Inc. 28-EPI-RAPALOGOS.
US6780639B1 (en) 1999-08-24 2004-08-24 Universite Libre De Bruxelles Antibiotic inducible/repressible genetic construct for gene therapy or gene immunization
JP3961737B2 (en) 2000-02-29 2007-08-22 株式会社小糸製作所 Vehicular lamp and manufacturing method thereof
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
DE60122685T2 (en) 2000-03-22 2007-08-23 Rheogene Holdings Inc. INDUCED GENE EXPRESSION SYSTEM BASED ON ECDYSON RECEPTORS
WO2001091803A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
DK1456346T3 (en) 2001-02-20 2012-05-29 Intrexon Corp NEW INDUCERABLE Gene EXPRESSION SYSTEM BASED ON ECDYSON RECEPTOR / INVERTEBRATE-RETINOID-X RECEPTOR
AU2002248500B2 (en) 2001-02-20 2007-12-13 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
EP1572862B1 (en) 2001-02-20 2012-08-01 Intrexon Corporation Chimeric retinoid x receptors and their use in a novel ecdysone receptor-based inducible gene expression system
WO2002066612A2 (en) 2001-02-20 2002-08-29 Rheogene, Inc. Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
IL158487A0 (en) 2001-05-18 2004-05-12 Wisconsin Alumni Res Found Method for the synthesis of dna sequences
NZ578982A (en) 2001-11-13 2011-03-31 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
ES2717377T3 (en) 2001-12-17 2019-06-20 Univ Pennsylvania Sequences of serotype 8 of adeno-associated virus (AAV), vectors containing them and uses thereof
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US8005620B2 (en) 2003-08-01 2011-08-23 Dna Twopointo Inc. Systems and methods for biopolymer engineering
WO2005013090A2 (en) 2003-08-01 2005-02-10 Dna Twopointo Inc. Systems and methods for biopolymer engineering
EP3910063A1 (en) 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US20060246079A1 (en) 2003-11-14 2006-11-02 Morrow Phillip R Neutralizing human antibodies to anthrax toxin
US7935510B2 (en) 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
EP2359865B1 (en) 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
PL2420256T3 (en) 2005-05-02 2016-12-30 Gene therapy for neurometabolic disorders
US7456683B2 (en) 2005-06-09 2008-11-25 Panasonic Corporation Amplitude error compensating device and quadrature skew error compensating device
DK3308788T3 (en) 2005-06-23 2019-01-02 Biogen Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN2 SPLASH
US8877187B2 (en) 2005-07-25 2014-11-04 Avianax, Llc Therapeutic antibodies for treatment and prophylaxis of transmittable viral diseases
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
HUE044182T2 (en) 2006-06-07 2019-10-28 Genzyme Corp Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
WO2008042420A2 (en) 2006-10-03 2008-04-10 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
JP2010520748A (en) 2007-02-20 2010-06-17 アナプティスバイオ インコーポレイティッド Somatic hypermutation system
EP2125886A2 (en) 2007-03-13 2009-12-02 Humabs LLC Antibodies against h5n1 strains of influenza a virus
WO2008124934A1 (en) 2007-04-17 2008-10-23 National Research Council Of Canada Constructs for enhancement of gene expression in muscle
US7879326B2 (en) 2007-06-15 2011-02-01 The Board Of Trustees Of The Leland Stanford Junior University Human neutralizing monoclonal antibodies to H5N1 influenza A virus
KR20100097691A (en) 2007-11-12 2010-09-03 테라클론 사이언시스, 아이엔씨. Compositions and methods for the therapy and diagnosis of influenza
NZ586611A (en) 2007-12-06 2012-09-28 Dana Farber Cancer Inst Inc Antibodies against influenza virus and methods of use thereof
ITTO20080204A1 (en) 2008-03-17 2009-09-18 Pomona Biotechnologies Llc MONOCLONAL ANTIBODIES ACTIONS TO REACT WITH A PLURALITY OF SUBTIPS OF THE INFLUENTIAL VIRUS A
EP2698380A1 (en) 2008-03-28 2014-02-19 Sea Lane Biotechnologies, LLC Neutralizing molecules to viral antigens
ITTO20080398A1 (en) 2008-05-27 2009-11-28 Pomona Biotechnologies Llc MONOCLONAL ANTIBODIES HAVING PROPERTY OF HOSUBLICTIC CROSS-NEUTRALIZATION FOR INFLUENTIAL VIRUSES OF SUBTIPLE TYPE H1
EP2296700A2 (en) 2008-06-03 2011-03-23 Vaxin, Inc. Intranasal administration of receptor-binding ligands or genes encoding such ligands as a therapeutic regimen for mitigating infections caused by respiratory pathogens
AU2009275226B2 (en) 2008-07-25 2015-05-14 Institute For Research In Biomedicine Neutralizing anti-influenza a virus antibodies and uses thereof
US8871207B2 (en) 2008-07-25 2014-10-28 Humabs, LLC Neutralizing anti-influenza A virus antibodies and uses thereof
CA3006395C (en) 2008-11-07 2022-05-31 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US8211631B2 (en) 2008-12-18 2012-07-03 Wisconsin Alumni Research Foundation In vitro model of spinal muscular atrophy
JP5879256B2 (en) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション Gene therapy for neurodegenerative disorders
KR101790354B1 (en) 2009-05-11 2017-10-25 얀센 백신스 앤드 프리벤션 비.브이. Human binding molecules capable of neutralizing influenza virus h3n2 and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
IT1395961B1 (en) 2009-06-01 2012-11-02 Pomona Biotechnologies Llc MONOCLONAL ANTIBODIES AS A MEDICATION FOR THERAPEUTIC AND / OR PROFILACTIC TREATMENT OF INFLUENCIAL VIRUS INFECTIONS (H1N1) OF SWINE ORIGIN (S-OIV)
EP3643783A1 (en) 2009-06-17 2020-04-29 Biogen MA Inc. Compositions and methods for modulation of smn2 splicing in a subject
WO2010151673A1 (en) 2009-06-25 2010-12-29 Medimmune, Llc Swine influenza hemagglutinin variants
US8598331B2 (en) 2009-09-28 2013-12-03 The University Of British Columbia CLDN5 mini-promoters
MX342858B (en) 2010-03-29 2016-10-13 The Trustees Of The Univ Of Pennsylvania * Pharmacologically induced transgene ablation system.
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
EP2678433B1 (en) 2011-02-22 2017-05-03 California Institute of Technology Delivery of proteins using adeno-associated virus (aav) vectors
WO2012145572A1 (en) 2011-04-20 2012-10-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
US20140127749A1 (en) 2011-04-21 2014-05-08 Arizona Borad Of Regents, A Body Corporate Of The State Of Arizona Acting For And On Behalf Of Arizo Methods of protein production and compositions thereof
KR20200084048A (en) 2011-06-08 2020-07-09 샤이어 휴먼 지네틱 테라피즈 인크. Lipid nanoparticle compositions and methods for mrna delivery
FR2977562B1 (en) 2011-07-06 2016-12-23 Gaztransport Et Technigaz SEALED AND THERMALLY INSULATING TANK INTEGRATED IN A CARRIER STRUCTURE
MY166282A (en) 2011-07-14 2018-06-25 Crucell Holland Bv Human binding molecules capable of neutralizing influenza a viruses of phylogenetic group 1 and phylogenetic group 2 and influenza b viruses
EP2814841A4 (en) 2012-01-31 2015-06-17 Univ Osaka Human monoclonal antibodies broadly protective against influenza b virus and methods of using the same
WO2013132007A1 (en) 2012-03-08 2013-09-12 Crucell Holland B.V. Human binding molecules capable of binding to and neutralizing influenza b viruses and uses thereof
JP2015516143A (en) * 2012-04-02 2015-06-08 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Modified polynucleotides for the production of proteins associated with human disease
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
PL2839014T3 (en) 2012-04-18 2021-08-23 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
WO2013182683A1 (en) 2012-06-08 2013-12-12 Ethris Gmbh Pulmonary delivery of messenger rna
EP3536795A3 (en) 2012-06-21 2019-12-11 Association Institut de Myologie Widespread gene expression
DK2880053T3 (en) 2012-08-01 2020-05-11 Ikaika Therapeutics Llc Relief of tissue damage and fibrosis via anti-LTBP4 antibody
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
AU2013388083B2 (en) * 2013-05-01 2019-08-22 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
EP3024497B1 (en) 2013-07-26 2021-01-13 University of Iowa Research Foundation Methods and compositions for treating brain diseases
JP6893785B2 (en) * 2013-10-22 2021-06-23 トランスレイト バイオ， インコーポレイテッド CNS delivery of mRNA and its usage
US20160243260A1 (en) 2013-10-24 2016-08-25 Uniqure Ip B.V. Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins
US20170183396A1 (en) 2014-02-19 2017-06-29 Jody Berry Ebola monoclonal antibodies
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
CA2946392A1 (en) 2014-04-25 2015-10-29 James M. Wilson Ldlr variants and their use in compositions for reducing cholesterol levels
WO2015164723A1 (en) 2014-04-25 2015-10-29 The Trustees Of The University Of Pennsylvania Methods and compositions for treating metastatic breast cancer and other cancers in the brain
CN114231563A (en) 2014-05-02 2022-03-25 建新公司 AAV vectors for retinal and CNS gene therapy
MA44179B1 (en) 2014-05-13 2020-10-28 Univ Pennsylvania Compositions comprising an adeno-associated virus expressing double antibody constructs and their uses
DK3198018T3 (en) 2014-09-24 2021-03-01 Hope City VECTOR VARIANTS OF ADENO ASSOCIATED VIRUS FOR HIGH-EFFECTIVE GENERATING AND METHODS THEREOF
JP2017531642A (en) 2014-10-03 2017-10-26 マサチューセッツ インスティテュート オブ テクノロジー Antibodies that bind to Ebola glycoprotein and uses thereof
CN107207611B (en) 2015-02-05 2021-11-09 扬森疫苗与预防公司 Binding molecules against influenza hemagglutinin and uses thereof
CN108135994B (en) 2015-05-13 2022-01-25 宾夕法尼亚州大学信托人 AAV-mediated expression of anti-influenza antibodies and methods of use thereof
CN108779167A (en) 2015-10-28 2018-11-09 宾夕法尼亚州大学信托人 Intrathecal application adeno-associated virus vector is used for gene therapy
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP4215605A1 (en) 2015-12-11 2023-07-26 The Trustees of The University of Pennsylvania Scalable purification method for aav8
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017106326A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Aav-anti pcsk9 antibody constructs and uses thereof
WO2017106244A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Compositions and methods for regulatable antibody expression
MA44119A (en) 2015-12-14 2018-10-31 Univ Pennsylvania ADENO-ASSOCIATED VIRAL VECTORS FOR USE IN THE TREATMENT OF SPINAL AMYOTROPHY
MA43968A (en) 2016-02-03 2018-12-12 Univ Pennsylvania GENE THERAPY TO TREAT MUCOPOLYSACCHARIDOSIS TYPE I
KR102425289B1 (en) 2016-08-15 2022-07-27 젠자임 코포레이션 AAV detection method
US20190240328A1 (en) 2016-09-24 2019-08-08 The Trustees Of The University Of Pennsylvania Novel humanized anti-ebola antibodies useful in preventing ebola infections
JP2020510430A (en) 2017-02-28 2020-04-09 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 Influenza vaccine based on AAV vector
AU2018227440A1 (en) 2017-02-28 2019-08-29 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clade f vector and uses therefor

Also Published As

Publication number Publication date
AU2018227442A1 (en) 2019-08-29
CO2019009522A2 (en) 2019-09-18
BR112019016444A2 (en) 2020-04-07
US20200056205A1 (en) 2020-02-20
US11578341B2 (en) 2023-02-14
WO2018160585A2 (en) 2018-09-07
WO2018160585A3 (en) 2018-10-25
EP3589739A2 (en) 2020-01-08
KR20190123752A (en) 2019-11-01
CN110741089A (en) 2020-01-31
MX2019010276A (en) 2019-10-15
IL268519A (en) 2019-09-26
US20230265459A1 (en) 2023-08-24
CL2019002473A1 (en) 2019-11-29
MA47688A (en) 2020-01-08
PE20191261A1 (en) 2019-09-18
JP2022078208A (en) 2022-05-24
JP7037574B2 (en) 2022-03-16
PH12019550149A1 (en) 2020-06-08
JOP20190200A1 (en) 2019-08-27
JP2020510428A (en) 2020-04-09
CA3055619A1 (en) 2018-09-07
SG11201907474UA (en) 2019-09-27

Similar Documents

Publication Publication Date Title
IL268519A (en) Compositions useful in treatment of spinal muscular atrophy
HK1256089A1 (en) Compositions for treating spinal muscular atrophy
IL268406A (en) Compositions and methods for the treatment of hemoglobinopathies
IL259877A (en) Adeno-associated viral vectors useful in treatment of spinal muscular atrophy
EP3606562A4 (en) Compositions and methods for treating spinal muscular atrophy
EP3341391A4 (en) Compositions and methods for treatment of pain
IL262830A (en) Compositions and methods for treating spinal muscular atrophy
IL251351B (en) Compositions for use in the treatment and prophylaxis of surgical site infections
IL276180A (en) Methods of treating spinal muscular atrophy
IL261277A (en) Combination therapies for treatment of spinal muscular atrophy
EP3781214A4 (en) Compositions and methods for treating spinal muscular atrophy
IL272036A (en) Methods and compositions for treatment of pain using capsaicin
HK1258502A1 (en) Compositions and methods for treatment of homocystinuria
PL3283064T3 (en) Derivatives used in the treatment of muscle atrophy
EP3518951C0 (en) Compositions for use in the treatment of osteoporis and bone fractures
IL273850A (en) Compositions and methods for treatment of fibrosis
EP3609430C0 (en) Improvements in or relating to dental treatment instruments
EP3784261A4 (en) Compositions and methods for prophylaxis or treatment of pain
PL3352747T3 (en) Compositions for treatment of pain in patients who underwent elective arthroplasty
GB201810925D0 (en) Compositions and methods of treatment
GB201710491D0 (en) Composition and methods of treatment
GB201713757D0 (en) Improvements in or relating to dental treatment instruments
AU2015904350A0 (en) Composition and methods for the prevention and treatment of muscle atrophy