WO2007120542A3 - Aav capsid library and aav capsid proteins - Google Patents

Aav capsid library and aav capsid proteins Download PDF

Info

Publication number
WO2007120542A3
WO2007120542A3 PCT/US2007/008314 US2007008314W WO2007120542A3 WO 2007120542 A3 WO2007120542 A3 WO 2007120542A3 US 2007008314 W US2007008314 W US 2007008314W WO 2007120542 A3 WO2007120542 A3 WO 2007120542A3
Authority
WO
WIPO (PCT)
Prior art keywords
aav capsid
library
proteins
capsid proteins
aav
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/US2007/008314
Other languages
French (fr)
Other versions
WO2007120542A2 (en
Inventor
Mark A Kay
Dirk Grimm
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.)
Leland Stanford Junior University
Original Assignee
Leland Stanford Junior University
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 Leland Stanford Junior University filed Critical Leland Stanford Junior University
Priority to EP07794340.5A priority Critical patent/EP2007795B1/en
Publication of WO2007120542A2 publication Critical patent/WO2007120542A2/en
Publication of WO2007120542A3 publication Critical patent/WO2007120542A3/en
Anticipated expiration legal-status Critical
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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034—Isolating an individual clone by screening libraries
    • C12N15/1082—Preparation or screening gene libraries by chromosomal integration of polynucleotide sequences, HR-, site-specific-recombination, transposons, viral vectors
    • 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
    • C12N2750/14122—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Recombinant adeno-associated viral (AAV) capsid proteins are provided. Methods for generating the recombinant adeno-associated viral capsid proteins and a library from which the capsids are selected are also provided.
PCT/US2007/008314 2006-03-30 2007-03-30 Aav capsid library and aav capsid proteins Ceased WO2007120542A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP07794340.5A EP2007795B1 (en) 2006-03-30 2007-03-30 Aav capsid proteins

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US78737106P 2006-03-30 2006-03-30
US60/787,371 2006-03-30

Publications (2)

Publication Number Publication Date
WO2007120542A2 WO2007120542A2 (en) 2007-10-25
WO2007120542A3 true WO2007120542A3 (en) 2008-07-03

Family

ID=38610075

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2007/008314 Ceased WO2007120542A2 (en) 2006-03-30 2007-03-30 Aav capsid library and aav capsid proteins

Country Status (3)

Country Link
US (5) US7588772B2 (en)
EP (1) EP2007795B1 (en)
WO (1) WO2007120542A2 (en)

Families Citing this family (304)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
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
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2009154452A1 (en) * 2008-06-17 2009-12-23 Amsterdam Molecular Therapeutics B.V. Parvoviral capsid with incorporated Gly-Ala repeat region
TR201813067T4 (en) * 2008-09-15 2018-09-21 Uniqure Biopharma B V The factor IX polypeptide mutant, its uses and a method for producing it.
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
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
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
EP3444346B1 (en) 2010-04-23 2022-07-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
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
US10415056B2 (en) 2010-11-10 2019-09-17 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
EP2673002A4 (en) * 2011-02-10 2015-12-30 Cedars Sinai Medical Center METHODS OF TREATING ALZHEIMER'S DISEASE BY APOA-1 MILANO
EP3318635A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
CN105755044A (en) 2011-04-22 2016-07-13 加利福尼亚大学董事会 Adeno-associated Virus Virions With Variant Capsid And Methods Of Use Thereof
ES2857773T5 (en) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Novel AAV capsid proteins for nucleic acid transfer
EP2747551B1 (en) 2011-08-26 2020-02-12 Yecuris Corporation Fumarylacetoacetate hydrolase (fah)-deficient and immunodeficient rats and uses thereof
US20140359799A1 (en) * 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
MX367100B (en) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues.
WO2013170078A1 (en) * 2012-05-09 2013-11-14 Oregon Health & Science University Adeno associated virus plasmids and vectors
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
AU2014235096B2 (en) 2013-03-15 2020-03-19 The Trustees Of The University Of Pennsylvania Compositions and methods for treating MPSI
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Modified Aav and preparations, methods and uses for gene transfer to cells, organs and tissues
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
SMT202000531T1 (en) 2014-03-09 2020-11-10 Univ Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
CA2942515C (en) 2014-03-18 2025-12-09 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
PL3628334T3 (en) 2014-03-21 2023-12-18 Genzyme Corporation Gene therapy for retinitis pigmentosa
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
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
EP3134431B1 (en) 2014-04-25 2021-04-07 The Trustees Of The University Of Pennsylvania Ldlr variants and their use in compositions for reducing cholesterol levels
ES2833230T3 (en) 2014-05-13 2021-06-14 Univ Pennsylvania Compositions Comprising AVV Expressing Double Antibody Constructs and Uses thereof
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
EP4012035B1 (en) 2014-09-16 2024-11-06 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
PL4012035T3 (en) 2014-09-16 2025-03-24 Genzyme Corporation ADENOVIRUS-RELATED VIRAL VECTORS FOR THE TREATMENT OF MYOCILLOIDAL GLAUCOMA (MYOC)
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3200830B1 (en) * 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
CN107073051B (en) 2014-10-21 2021-08-24 马萨诸塞大学 Recombinant AAV variants and their uses
RU2716991C2 (en) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Aadc polynucleotides for treating parkinson's disease
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
KR20230145206A (en) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. Modulatory polynucleotides
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US10900053B2 (en) 2014-11-21 2021-01-26 University Of Florida Research Foundation, Incorporated Genome-modified recombinant adeno-associated virus vectors
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
MX2017009336A (en) 2015-01-16 2017-11-15 Voyager Therapeutics Inc Central nervous system targeting polynucleotides.
EA201791630A1 (en) 2015-01-20 2018-04-30 Джензим Корпорейшн ANALYTICAL ULTRACENTRIFUGATION FOR DETERMINING THE CHARACTERISTICS OF RECOMBINANT VIRAL PARTICLES
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
CN107530447A (en) 2015-02-10 2018-01-02 建新公司 Enhanced delivery of viral particles to the striatum and cortex
CR20170406A (en) 2015-02-10 2017-11-14 Genzyme Corp ARNI VARIANTE
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US20180044698A1 (en) * 2015-02-19 2018-02-15 University Of Florida Research Foundation, Incorporated Site-specific integrating recombinant aav vectors for gene therapy and improved production methods
AU2016219789B2 (en) * 2015-02-20 2021-03-25 Fondazione Telethon Methods and compositions for treating genetic eye diseases
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
JP6836999B2 (en) * 2015-03-24 2021-03-03 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Ｔｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ Adeno-associated virus mutants and how to use them
TWI707951B (en) 2015-04-08 2020-10-21 美商健臻公司 Production of oversized adeno-associated vectors
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
EP3288594B1 (en) 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
EA201792500A1 (en) 2015-05-13 2018-04-30 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания AAV-MEDIATED EXPRESSION OF ANTIBODIES AGAINST FLU AND METHODS OF THEIR USE
BR112017024514A2 (en) 2015-05-16 2018-07-24 Genzyme Corporation genetic editing of deep intronic mutations
MX390848B (en) 2015-06-23 2025-03-21 Childrens Hospital Philadelphia MODIFIED FACTOR IX, AND COMPOSITIONS, METHODS AND USES FOR GENE TRANSFER TO CELLS, ORGANS AND TISSUES.
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
WO2017024198A1 (en) 2015-08-06 2017-02-09 The Trustees Of The University Of Pennsylvania Glp-1 and use thereof in compositions for treating metabolic diseases
WO2017035416A2 (en) 2015-08-25 2017-03-02 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
CA2995733A1 (en) 2015-08-31 2017-03-09 The Trustees Of The University Of Pennsylvania Aav-epo for treating companion animals
CN108291216B (en) 2015-09-24 2022-09-16 宾夕法尼亚州大学信托人 Compositions and methods for treating complement-mediated diseases
ES2865487T3 (en) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Methods and compositions for viral vectors that evade antibodies
US11273227B2 (en) 2015-10-09 2022-03-15 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating Stargardt's disease and other ocular disorders
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
CN108779167A (en) 2015-10-28 2018-11-09 宾夕法尼亚州大学信托人 Intrathecal administration of adeno-associated viral vectors for gene therapy
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. ASSAYS FOR DETECTION OF NEUTRALIZING ANTIBODIES OF VAA
AU2016366560A1 (en) 2015-12-11 2018-06-28 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11028372B2 (en) 2015-12-11 2021-06-08 The Trustees Of The University Of Pennsylvania Scalable purification method for AAVRH10
ES2934848T3 (en) 2015-12-11 2023-02-27 Univ Pennsylvania Scalable purification method for AAV8
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3387138B1 (en) 2015-12-11 2022-01-26 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
CA3008264A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JP7061067B2 (en) 2015-12-14 2022-04-27 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 Composition for the treatment of Crigler-Najer syndrome
MX2018007330A (en) 2015-12-15 2019-03-14 Genzyme Corp Adeno-associated viral vectors for treating mucolipidosis type ii.
CA3011939A1 (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
CA3012195A1 (en) 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
SG10201912761UA (en) 2016-04-15 2020-02-27 The Trustees Of The Univ Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
US11401527B2 (en) 2016-04-17 2022-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11364308B2 (en) 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
KR20240056729A (en) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. Modulatory polynucleotides
KR102427379B1 (en) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 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
US11174469B2 (en) 2016-06-29 2021-11-16 Crispr Therapeutics Ag Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders
EP4484443A3 (en) 2016-06-29 2025-03-26 CRISPR Therapeutics AG Materials and methods for treatment of friedreich ataxia and other related disorders
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
CN109843914B (en) 2016-07-06 2024-03-15 沃泰克斯药物股份有限公司 Materials and methods for treating pain-related conditions
JP7305534B2 (en) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド Materials and methods for treating pain-related disorders
WO2018009814A1 (en) 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
JP7490211B2 (en) 2016-07-19 2024-05-27 デューク ユニバーシティ Therapeutic Applications of CPF1-Based Genome Editing
WO2018022511A1 (en) 2016-07-25 2018-02-01 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
CA3033856A1 (en) 2016-08-15 2018-02-22 Genzyme Corporation Methods for detecting aav
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CA3038292A1 (en) 2016-09-28 2018-04-05 Cohbar, Inc. Therapeutic mots-c related peptides
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
US20190328846A1 (en) 2016-12-01 2019-10-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Pharmaceutical compositions for the treatment of retinal degenerative diseases
JP2020510648A (en) 2017-02-20 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 Gene therapy to treat familial hypercholesterolemia
EP3585895A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Compositions and methods for gene editing
EP3585807A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
EP3585898A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
WO2018154459A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
EP3585900B1 (en) 2017-02-22 2022-12-21 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
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
PT3589730T (en) 2017-02-28 2024-02-28 Univ Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
UY37620A (en) 2017-02-28 2018-08-31 Univ Pennsylvania VACCINES AGAINST THE FLU MEDIATED BY NEW NOVEDOSA
RU2019130004A (en) 2017-03-01 2021-04-01 Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания GENE THERAPY FOR EYE DISEASES
WO2018200542A1 (en) 2017-04-24 2018-11-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
JP2020518259A (en) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Huntington's disease treatment compositions and methods
JP2020518258A (en) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． Amyotrophic lateral sclerosis (ALS) treatment composition and method
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
IL316926A (en) 2017-05-11 2025-01-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinoses
EP3630986B1 (en) 2017-05-31 2026-03-25 The Trustees of The University of Pennsylvania Gene therapy for treating peroxisomal disorders
EP3634978A1 (en) 2017-06-07 2020-04-15 Adrx, Inc. Tau aggregation inhibitors
JP7766393B2 (en) 2017-06-14 2025-11-10 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 Gene Therapy for Eye Disease
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP2020534788A (en) 2017-08-28 2020-12-03 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 capsid mutant and how to use it
RU2770922C2 (en) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Capsids of adeno-associated virus variants and methods of their application
CN111448321A (en) 2017-09-22 2020-07-24 马萨诸塞大学 SOD1 dual expression vector and its use
KR102883778B1 (en) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Gene therapy for the treatment of type II mucopolysaccharidosis
CN111356763B (en) 2017-09-22 2024-03-12 建新公司 variant RNAi
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
TW202413649A (en) 2017-10-16 2024-04-01 美商航海家醫療公司 Treatment of amyotrophic lateral sclerosis (als)
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CA3082450A1 (en) 2017-11-21 2019-05-31 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
CN111770999A (en) 2017-11-27 2020-10-13 4D分子治疗有限公司 Adeno-associated virus variant capsids and use for inhibiting angiogenesis
CN111989396A (en) 2017-11-30 2020-11-24 宾夕法尼亚州大学信托人 Gene therapy for mucopolysaccharidosis type IIIB
AU2018375164B2 (en) 2017-11-30 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for Mucopolysaccharidosis IIIA
EP3727468A4 (en) 2017-12-19 2021-09-22 Akouos, Inc. AAV-MEDIATED DELIVERY OF THERAPEUTIC ANTIBODIES TO THE INNER EAR
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
WO2019123430A1 (en) 2017-12-21 2019-06-27 Casebia Therapeutics Llp Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
SI3740222T1 (en) * 2018-01-17 2023-11-30 Meiragtx Uk Ii Limited MODIFIED RAAV CAPSID PROTEIN FOR GENE THERAPY
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US20190256867A1 (en) 2018-02-01 2019-08-22 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
JP7244547B2 (en) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド Adeno-associated virus compositions and methods of use thereof for restoring F8 gene function
EP3768695A4 (en) 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSID DEAMIDATION AND THEIR USES
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
MX2020010465A (en) 2018-04-03 2021-01-08 Virus vectors for targeting ophthalmic tissues.
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020011386A (en) 2018-04-27 2021-01-29 Spacecraft Seven Llc GENE THERAPY FOR CNS DEGENERATION.
TW202005978A (en) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 Novel liver targeting adeno-associated viral vectors
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
KR20210019996A (en) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. Composition and method for the treatment of Parkinson's disease
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
WO2020014471A1 (en) 2018-07-11 2020-01-16 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of agents across the blood-brain barrier
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
KR102918544B1 (en) 2018-07-12 2026-01-27 로켓 파마슈티컬스, 리미티드 Gene therapy vector for treating Danone disease
CN112770812A (en) 2018-07-24 2021-05-07 沃雅戈治疗公司 System and method for producing gene therapy formulations
WO2020028751A2 (en) 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
KR102931619B1 (en) 2018-08-03 2026-02-26 젠자임 코포레이션 Mutant RNAi against alpha-synuclein
MA53252A (en) 2018-08-10 2021-09-15 Logicbio Therapeutics Inc NON-DISRUPTIVE GENE THERAPY FOR THE TREATMENT OF MMA
CN113966399A (en) * 2018-09-26 2022-01-21 加州理工学院 Adeno-associated virus compositions for targeted gene therapy
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CA3114175A1 (en) 2018-10-01 2020-04-09 The Trustees Of The University Of Pennsylvania Beta-galactosidase based aav for treating gm1 gangliosidosis
CN113166208B (en) 2018-10-02 2024-11-22 沃雅戈治疗公司 Redirecting the tropism of AAV capsids
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2019357039B2 (en) 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy
UY38407A (en) 2018-10-15 2020-05-29 Novartis Ag TREM2 STABILIZING ANTIBODIES
SG11202107645RA (en) 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
SG11202107761SA (en) 2019-01-28 2021-08-30 Cohbar Inc Therapeutic peptides
CN110437317B (en) * 2019-01-30 2023-05-02 上海科技大学 Adeno-associated virus with mutated capsid protein and use thereof
WO2020160508A1 (en) 2019-01-31 2020-08-06 Oregon Health & Science University Methods for using transcription-dependent directed evolution of aav capsids
BR112021015817A2 (en) 2019-02-22 2021-10-13 The Trustees Of The University Of Pennsylvania RECOMBINANT ADENO-ASSOCIATED VIRUS FOR ADULT-ONSET NEURODEGENERATION TREATMENT ASSOCIATED WITH GRN
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
SG11202108044YA (en) 2019-02-25 2021-09-29 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
EP3931337A1 (en) 2019-02-26 2022-01-05 The Trustees of The University of Pennsylvania Compositions useful in treatment of krabbe disease
US20220047618A1 (en) 2019-02-28 2022-02-17 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
CN113795279B (en) 2019-03-04 2024-12-31 宾夕法尼亚州大学信托人 Neuroprotective gene therapy targeting the AKT pathway
AR118465A1 (en) 2019-03-21 2021-10-06 Stridebio Inc RECOMBINANT ADENO-ASSOCIATED VIRUS VECTORS
KR102934700B1 (en) * 2019-03-28 2026-03-09 더 제너럴 하스피탈 코포레이션 Engineered adeno-associated (AAV) vectors for transgene expression
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
KR20220020261A (en) 2019-05-03 2022-02-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Compositions useful for the treatment of ichthyosis leukodystrophy
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
WO2020223933A1 (en) * 2019-05-08 2020-11-12 中国科学院脑科学与智能技术卓越创新中心 Aav mutant that efficiently infects supporting cells
TW202104592A (en) * 2019-05-14 2021-02-01 美商拜奧馬林製藥公司 Methods of redosing gene therapy vectors
MA56393A (en) 2019-06-28 2022-05-04 Bayer Healthcare Llc MATERIALS AND METHODS OF REGULATING GENE EDITING
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
WO2021025995A1 (en) 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP4450514A3 (en) 2019-10-14 2025-04-09 Duke University Compositions comprising novel prokaryotic sodium channels and associated methods
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (en) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド Adeno-associated viral vectors for the treatment of Niemann-Pick disease type C
AU2020401116A1 (en) 2019-12-10 2022-07-21 Takeda Pharmaceutical Company Limited Adeno associated virus vectors for the treatment of Hunter disease
IL293658A (en) * 2019-12-10 2022-08-01 Homology Medicines Inc Adeno-associated virus compositions and methods of use thereof
CA3162020A1 (en) 2019-12-19 2021-06-24 James M. Wilson Compositions for treating friedreich's ataxia
WO2021138559A1 (en) 2019-12-31 2021-07-08 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
CN115279400B (en) 2020-01-10 2026-04-28 布里格姆妇女医院 Methods and compositions for delivering immunotherapeutic agents across the blood-brain barrier to treat brain cancer
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
AU2021213735A1 (en) 2020-01-30 2022-09-22 Umoja Biopharma, Inc. Bispecific transduction enhancer
KR20220145838A (en) 2020-02-02 2022-10-31 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Compositions useful for treating GM1 gangliosiosis
CN111349148A (en) * 2020-03-13 2020-06-30 辉大(上海)生物科技有限公司 Adeno-associated virus vector and application thereof
IL296732A (en) 2020-03-27 2022-11-01 UCB Biopharma SRL Autonomous knob domain peptides
WO2021202651A1 (en) 2020-04-01 2021-10-07 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
JP7713956B2 (en) 2020-04-15 2025-07-28 ボイジャー セラピューティクス インコーポレイテッド Tau-binding compounds
MX2022014258A (en) 2020-05-12 2023-02-22 Univ Pennsylvania Compositions for drg-specific reduction of transgene expression.
CA3177924A1 (en) 2020-05-12 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
TWI900553B (en) 2020-05-13 2025-10-11 美商航海家醫療公司 Redirection of tropism of aav capsids
WO2021231538A2 (en) 2020-05-13 2021-11-18 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
IL298128A (en) 2020-05-13 2023-01-01 Akouos Inc Compositions and methods for treating slc26a4-associated hearing loss
TW202208632A (en) 2020-05-27 2022-03-01 美商同源醫藥公司 Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
AU2021292200A1 (en) 2020-06-17 2023-02-02 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
CA3187635A1 (en) * 2020-07-03 2022-01-06 Genethon Method for engineering novel hybrid aav capsids through hypervariable regions swapping
IL299762A (en) 2020-07-13 2023-03-01 Univ Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (en) 2020-07-27 2023-04-11 Voyager Therapeutics Inc COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO BETA-GLYCOSYLCERAMIDASE DEFICIENCY
KR20230043869A (en) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Placophilin-2 (PKP2) gene therapy using AAV vectors
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CA3190399A1 (en) 2020-08-24 2022-03-03 James M. Wilson Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
EP4204014A1 (en) 2020-08-26 2023-07-05 The Trustees of The University of Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
MX2023003805A (en) 2020-10-01 2023-04-12 Genzyme Corp Human pah expression cassette for treatment of pku by liver-directed gene replacement therapy.
AU2021358781A1 (en) 2020-10-07 2023-05-18 Tern Therapeutics, Llc Gene therapy for ocular manifestations of cln2 disease
AU2021356684A1 (en) 2020-10-09 2023-05-11 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2022082109A1 (en) 2020-10-18 2022-04-21 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
AU2021375404A1 (en) 2020-11-03 2023-06-08 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
TW202237850A (en) 2020-12-01 2022-10-01 賓州大學委員會 Novel compositions with tissue-specific targeting motifs and compositions containing same
IL303239A (en) 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
IL303317A (en) 2020-12-01 2023-07-01 Akouos Inc Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of AAV vectors by affinity chromatography
IL304006A (en) 2020-12-29 2023-08-01 Akouos Inc Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
AR124981A1 (en) 2021-02-26 2023-05-24 Takeda Pharmaceuticals Co COMPOSITION AND METHODS FOR THE TREATMENT OF FABRY DISEASE
US20240307553A1 (en) 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
IL307633A (en) 2021-04-12 2023-12-01 Univ Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
US20240181084A1 (en) 2021-04-23 2024-06-06 University Of Rochester Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas Fusion Protein and Methods of Treatment
CA3216004A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2022269466A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
CA3227103A1 (en) 2021-07-30 2023-02-02 Matthew P. GEMBERLING Compositions and methods for modulating expression of frataxin (fxn)
US20240252684A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
KR20240107201A (en) 2021-08-31 2024-07-08 스카우트 바이오, 인코포레이티드 Antigen-binding molecules and uses thereof
JP2024537775A (en) 2021-09-30 2024-10-16 アコーオス インコーポレイテッド Compositions and methods for treating KCNQ4-associated hearing loss
EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
US20240408157A1 (en) 2021-12-02 2024-12-12 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023133574A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
CN119317718A (en) 2022-01-25 2025-01-14 宾夕法尼亚州大学信托人 AAV capsids for improved cardiac transduction and liver targeting
TW202342525A (en) 2022-02-02 2023-11-01 美商阿科奧斯公司 Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
WO2023166425A1 (en) 2022-03-01 2023-09-07 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions
CN119301262A (en) 2022-04-01 2025-01-10 武田药品工业株式会社 Gene therapy for diseases with CNS manifestations
IL316038A (en) 2022-04-04 2024-11-01 Univ California Genetic complementation compositions and methods
WO2023196862A1 (en) 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
EP4504797A1 (en) 2022-04-06 2025-02-12 The Trustees of The University of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
US20250270261A1 (en) 2022-04-21 2025-08-28 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins for nucleic acid tranfer
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
CA3261865A1 (en) 2022-07-12 2024-01-18 Tune Therapeutics, Inc. Compositions, systems, and methods for targeted transcriptional activation
AU2023331251A1 (en) 2022-08-25 2025-02-27 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
EP4634394A2 (en) 2022-12-17 2025-10-22 The Trustees of The University of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
IT202200026595A1 (en) 2022-12-22 2024-06-22 Fond Telethon Ets NEW INHIBITORS OF EPIGENETIC REGULATORS
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024186275A1 (en) * 2023-03-09 2024-09-12 Agency For Science, Technology And Research Novel compositions of adeno-associated viruses engineered for enhanced tissue transduction and specificity
CN121586778A (en) 2023-04-26 2026-02-27 赛诺菲 Host cell lines and methods for identifying and using such host cell lines
WO2024229309A2 (en) 2023-05-03 2024-11-07 Manifold Biotechnologies, Inc. Methodsand compositions for high-throughput protein delivery, screening, and detection
KR20260010739A (en) 2023-05-11 2026-01-21 유니버시티 하스피탈즈 클리블랜드 메디컬 센터 Anti-anxiety treatment
WO2024241176A1 (en) 2023-05-24 2024-11-28 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of diseases with central nervous system disorders
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
TW202516019A (en) 2023-06-29 2025-04-16 賓州大學委員會 Mutant aav with central nervous system targeting motifs and compositions containing same
AU2024301706A1 (en) 2023-07-21 2026-02-05 Crispr Therapeutics Ag Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene
IL326390A (en) 2023-08-10 2026-04-01 Univ Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
US20250197862A1 (en) 2023-12-15 2025-06-19 Genzyme Corporation ARTIFICIAL microRNAs TARGETING TAU
WO2025160429A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
WO2025160434A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease
TW202548022A (en) 2024-03-03 2025-12-16 美商帕西奇生物公司 Recombinant adeno-associated virus for treatment of neurodegenerative disorders
WO2025186726A1 (en) 2024-03-05 2025-09-12 Crispr Therapeutics Ag Modulating expression of agt (angiotensinogen) gene
WO2026022064A1 (en) * 2024-07-22 2026-01-29 F. Hoffmann-La Roche Ag Novel aav rep orfs and rep polypeptides

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020192823A1 (en) * 2001-01-05 2002-12-19 Bartlett Jeffrey S. AAV2 vectors and methods
WO2005072364A2 (en) * 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5993800A (en) 1995-06-05 1999-11-30 Bristol-Myers Squibb Company Methods for prolonging the expression of a heterologous gene of interest using soluble CTLA4 molecules and an antiCD40 ligand
US5773289A (en) 1995-06-06 1998-06-30 University Of Pittsburgh AAV directed targeted integration
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5748745A (en) 1996-02-26 1998-05-05 Bedini Electronics, Inc. Analog vector processor and method for producing a binaural signal
DE19644500C2 (en) 1996-10-25 1998-11-05 Deutsches Krebsforsch AAV DNA with helper virus sequences
AU5588298A (en) 1996-12-02 1998-06-29 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
US6548286B1 (en) 1997-04-14 2003-04-15 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
JPH11148326A (en) 1997-11-12 1999-06-02 Fuji Heavy Ind Ltd Control device for electromagnetically driven valve
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
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
KR100312070B1 (en) * 1999-03-15 2001-11-03 박호군 Method of Selecting Enzyme Variants Including High Throughput Screening
AU7739000A (en) 1999-10-01 2001-05-10 University Of Florida Temperature-sensitive regulation of viral vector production
AU2001240018A1 (en) 2000-03-03 2001-09-17 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral compositions and methods
CA2373110A1 (en) * 2000-03-14 2001-09-20 Neurologix, Inc. Production of chimeric capsid vectors
ATE318923T1 (en) 2000-06-01 2006-03-15 Univ North Carolina DOUBLE STRANDED PARVOVIRUS VECTORS
US7201898B2 (en) 2000-06-01 2007-04-10 The University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
JP3449353B2 (en) * 2000-12-13 2003-09-22 日本電気株式会社 Communication system and transcoder alignment method
WO2002066611A2 (en) 2001-02-16 2002-08-29 The Board Of Trustees Of The Leland Stanford Junior University Minimal plasmid vectors that provide for persistent and high level gene expression and methods for using the same
CA2442144A1 (en) 2001-03-26 2002-11-21 The Board Of Trustees Of The Leland Stanford Junior University A helper dependent adenoviral vector system and methods for using the same
DE10120265A1 (en) 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV helper plasmids for helper virus-free packaging and pseudotyping of AAV vectors
DE10137283A1 (en) 2001-08-01 2003-02-27 Deutsches Krebsforsch AAV vector packaging plasmids for helper virus-free production of pseudotyped AAV particles by single transfection
MX359371B (en) 2001-11-13 2018-09-25 Univ Pennsylvania Method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby.
DK2573170T3 (en) * 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
PT1453547T (en) 2001-12-17 2016-12-28 Univ Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
US20040009151A1 (en) 2002-04-04 2004-01-15 Kay Mark A. Methods for delivering recombinant adeno-associated virus virions to the liver of a mammal
AU2003223766A1 (en) * 2002-04-30 2003-11-17 University Of North Carolina At Chapel Hill Secretion signal vectors
AU2003265855A1 (en) * 2002-08-28 2004-03-19 University Of Florida Modified aav
WO2004020605A2 (en) 2002-08-29 2004-03-11 The Board Of Trustees Of The Leland Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same
US20040203133A1 (en) 2003-01-07 2004-10-14 Anja Ehrhardt Helper dependent adenoviral vector system and methods for using the same
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
JP5136766B2 (en) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル Chimera vector
US8008468B2 (en) 2005-02-16 2011-08-30 Benitec, Inc. RNAi expression constructs with liver-specific enhancer/promoter
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US7588772B2 (en) * 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020192823A1 (en) * 2001-01-05 2002-12-19 Bartlett Jeffrey S. AAV2 vectors and methods
WO2005072364A2 (en) * 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BOWLES D E ET AL: "Marker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production", JOURNAL OF VIROLOGY, THE AMERICAN SOCIETY FOR MICROBIOLOGY, US, vol. 77, no. 1, January 2003 (2003-01-01), pages 423 - 432, XP002975999, ISSN: 0022-538X *
CHEN CHUN-LIANG ET AL: "Molecular characterization of adeno-associated viruses infecting children", JOURNAL OF VIROLOGY, vol. 79, no. 23, December 2005 (2005-12-01), pages 14781 - 14792, XP002462123, ISSN: 0022-538X *
CHOI VIVIAN W ET AL: "AAV hybrid serotypes: Improved vectors for gene delivery", CURRENT GENE THERAPY, vol. 5, no. 3, June 2005 (2005-06-01), pages 299 - 310, XP002462124, ISSN: 1566-5232 *
DATABASE UniProt [online] 11 October 2004 (2004-10-11), "Capsid protein VP1.", XP002462125, retrieved from EBI accession no. UNIPROT:Q670R0 Database accession no. Q670R0 *

Also Published As

Publication number Publication date
EP2007795B1 (en) 2016-11-16
US8906387B2 (en) 2014-12-09
US7588772B2 (en) 2009-09-15
US8067014B2 (en) 2011-11-29
EP2007795A2 (en) 2008-12-31
US20070243526A1 (en) 2007-10-18
WO2007120542A2 (en) 2007-10-25
US20120255046A1 (en) 2012-10-04
US20120066783A1 (en) 2012-03-15
US8574583B2 (en) 2013-11-05
US20150057189A1 (en) 2015-02-26
US20100047174A1 (en) 2010-02-25

Similar Documents

Publication Publication Date Title
WO2007120542A3 (en) Aav capsid library and aav capsid proteins
EP3795581A3 (en) New avv capsid proteins for nucleic acid transfer
WO2006021724A3 (en) Adeno-associated viral vector for exon skipping in a gene encoding a dispensable-domain protein
EP2277996A3 (en) Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
WO2007120533A3 (en) Minigene expression cassette
CY1117575T1 (en) AAV ISOSOMATIA WITH REDUCED IMMUNOACTIVITY AND THEIR USES
WO2002053703A3 (en) Aav2 vectors and methods
WO2005111220A3 (en) Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
WO2006104945A3 (en) Hepatitis c therapies
TWI370946B (en) Projector, projection system, program and recording medium
ZA200703838B (en) Multivalent vaccines comprising recombinant viral vectors
EP1889117A4 (en) Bouncing mode operated scanning micro-mirror
GB0419424D0 (en) Transgene optimisation
IL195683A0 (en) Recombinant viral vaccine
WO2005052143A3 (en) Novel adenoviruses, nucleic acids that code for the same and the use of said viruses
WO2006079076A3 (en) Yersinia spp. polypeptides and methods of use
WO2008121435A3 (en) Methods for generating novel stabilized proteins
EP1733039A4 (en) Improved bacterial host cell for the direct expression of peptides
WO2007106351A3 (en) Porous silicon materials and devices
WO2006054096A3 (en) Soluble bifunctional proteins
WO2006041897A3 (en) Chimeric vectors
WO2006076742A3 (en) Methods and compositions for increasing membrane permeability
WO2007053165A3 (en) Virus protein microarray and uses therefor
WO2006115619A3 (en) Techniques for creating a user-friendly computer-based fax experience
IS7775A (en) DNA vaccine, which transcribes at least two unmodified viral viral proteins

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: 07794340

Country of ref document: EP

Kind code of ref document: A2

NENP Non-entry into the national phase

Ref country code: DE

REEP Request for entry into the european phase

Ref document number: 2007794340

Country of ref document: EP

WWE Wipo information: entry into national phase

Ref document number: 2007794340

Country of ref document: EP