BE2021C533I2 - - Google Patents

Download PDF

Info

Publication number
BE2021C533I2
BE2021C533I2 BE2021C533C BE2021C533C BE2021C533I2 BE 2021C533 I2 BE2021C533 I2 BE 2021C533I2 BE 2021C533 C BE2021C533 C BE 2021C533C BE 2021C533 C BE2021C533 C BE 2021C533C BE 2021C533 I2 BE2021C533 I2 BE 2021C533I2
Authority
BE
Belgium
Application number
BE2021C533C
Other languages
French (fr)
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=34426044&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=BE2021C533(I2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of BE2021C533I2 publication Critical patent/BE2021C533I2/fr

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
BE2021C533C 2003-09-30 2021-08-30 BE2021C533I2 (enrdf_load_stackoverflow)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US50822603P 2003-09-30 2003-09-30
US56654604P 2004-04-29 2004-04-29
EP17153156.9A EP3211085B1 (en) 2003-09-30 2004-09-30 Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor

Publications (1)

Publication Number Publication Date
BE2021C533I2 true BE2021C533I2 (enrdf_load_stackoverflow) 2024-01-12

Family

ID=34426044

Family Applications (1)

Application Number Title Priority Date Filing Date
BE2021C533C BE2021C533I2 (enrdf_load_stackoverflow) 2003-09-30 2021-08-30

Country Status (17)

Country Link
US (13) US7906111B2 (enrdf_load_stackoverflow)
EP (7) EP2345731B1 (enrdf_load_stackoverflow)
JP (9) JP5054975B2 (enrdf_load_stackoverflow)
CN (4) CN1856576B (enrdf_load_stackoverflow)
AU (2) AU2004278684B2 (enrdf_load_stackoverflow)
BE (1) BE2021C533I2 (enrdf_load_stackoverflow)
CA (3) CA2939103C (enrdf_load_stackoverflow)
CY (3) CY1119833T1 (enrdf_load_stackoverflow)
DK (4) DK2292780T3 (enrdf_load_stackoverflow)
ES (5) ES2555612T3 (enrdf_load_stackoverflow)
FR (1) FR21C1037I1 (enrdf_load_stackoverflow)
HU (4) HUE054805T2 (enrdf_load_stackoverflow)
NZ (1) NZ545628A (enrdf_load_stackoverflow)
PL (2) PL3211085T3 (enrdf_load_stackoverflow)
PT (2) PT2292780T (enrdf_load_stackoverflow)
SI (2) SI2292780T1 (enrdf_load_stackoverflow)
WO (1) WO2005033321A2 (enrdf_load_stackoverflow)

Families Citing this family (613)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
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
WO2006033672A2 (en) 2004-04-28 2006-03-30 The Trustees Of The University Of Pennsylvania Immunization regimen with e4-deleted adenovirus prime and e1-deleted adenovirus boost
EP1742668B1 (en) 2004-04-28 2011-02-09 The Trustees of The University of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
CN117363655A (zh) 2005-04-07 2024-01-09 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
CN101460055A (zh) 2006-04-07 2009-06-17 得克萨斯大学体系董事会 和腺伴随病毒-噬菌体颗粒相关的方法和组合物
ES2400235T3 (es) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania Método de producción escalable de AAV
EP2016174A2 (en) * 2006-04-28 2009-01-21 The Trustees of the University of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
EP3492596A1 (en) 2007-04-09 2019-06-05 University of Florida Research Foundation, Inc. Raav vector compositions having tyrosine-modified capsid proteins and methods for use
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US20090215879A1 (en) * 2008-02-26 2009-08-27 University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
ES2724122T3 (es) * 2009-04-30 2019-09-06 Univ Pennsylvania Composiciones para dirigir células de las vías respiratorias de conducción que comprenden construcciones de virus adenoasociado
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2010141706A1 (en) * 2009-06-03 2010-12-09 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
US20120244127A1 (en) 2009-10-01 2012-09-27 The Trustees Of The University Of Pennsylvania AAV Vectors Expressing SEC10 for Treating Kidney Damage
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
JP5922095B2 (ja) 2010-03-29 2016-05-24 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 薬理学的に誘導される導入遺伝子アブレーション系
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
JP5963743B2 (ja) 2010-04-23 2016-08-03 ユニバーシティ オブ マサチューセッツ Ｃｎｓターゲティングａａｖベクターおよびその使用方法
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
CN107828820B (zh) 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
EP4234571A3 (en) * 2011-02-10 2023-09-27 The University of North Carolina at Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
EP3699286A1 (en) 2011-04-20 2020-08-26 The Trustees of the University of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
CA2870511C (en) 2011-04-21 2023-08-08 University Of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
KR102121355B1 (ko) 2011-04-22 2020-06-11 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
WO2013029030A1 (en) 2011-08-24 2013-02-28 The Board Of Trustees Of The Leland Stanford Junior University New aav capsid proteins for nucleic acid transfer
EP2764119A2 (en) 2011-10-05 2014-08-13 Genelux Corporation Method for detecting replication or colonization of a biological therapeutic
WO2013063019A1 (en) 2011-10-28 2013-05-02 The Wistar Institute Of Anatomy And Biology Methods and compositions for enhancing the therapeutic effect of anti-tumor t cells
US10238755B2 (en) 2011-11-30 2019-03-26 The Wistar Institute Of Anatomy And Biology Methods and compositions for regulation of cell aging, carcinogenesis and reprogramming
EP2601968A1 (en) * 2011-12-06 2013-06-12 Deutsches Krebsforschungszentrum HPV derived polynucleic acids for therapy
WO2013103896A1 (en) 2012-01-06 2013-07-11 Mayo Foundation For Medical Education And Research Treating cardiovascular or renal diseases
US20140087362A1 (en) 2012-03-16 2014-03-27 Aladar A. Szalay Methods for assessing effectiveness and monitoring oncolytic virus treatment
WO2013158265A1 (en) 2012-04-20 2013-10-24 Genelux Corporation Imaging methods for oncolytic virus therapy
JP6385920B2 (ja) * 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
US12358954B2 (en) 2012-05-15 2025-07-15 University Of Florida Research Foundation, Incorporated Capsid-modified rAAV vector compositions and methods therefor
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US9770491B2 (en) 2012-07-11 2017-09-26 The Trustees Of The University Of Pennsylvania AAV-mediated gene therapy for RPGR X-linked retinal degeneration
BR112015002168A2 (pt) 2012-08-01 2017-11-07 Nationwide Childrens Hospital liberação intratecal de vírus 9 adeno-associado recombinante
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
US9636370B2 (en) * 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
US20140140959A1 (en) 2012-10-05 2014-05-22 Aladar A. Szalay Energy Absorbing-Based Diagnostic and Therapeutic Methods Employing Nucleic Acid Molecules Encoding Chromophore-Producing Enzymes
EP2749569A1 (en) 2012-12-28 2014-07-02 Annibale Alessandro Puca Variant of BPIFB4 protein
US8957044B2 (en) 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
CA2905952A1 (en) * 2013-03-13 2014-10-02 The Children's Hospital Of Philadelphia Adeno-associated virus vectors and methods of use thereof
CA2904960A1 (en) 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with als
CA2904396A1 (en) * 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav vectors
CN105026554B (zh) 2013-03-15 2020-08-11 宾夕法尼亚大学托管会 用于治疗mps1的组合物和方法
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
CA2907799A1 (en) * 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3054995A1 (en) * 2013-10-07 2016-08-17 Kiromic, LLC Compositions and methods for treating cardiovascular diseases using disease-specific promoter
ES2714535T3 (es) 2013-10-11 2019-05-28 Massachusetts Eye & Ear Infirmary Métodos para predecir secuencias de virus ancestrales y usos de los mismos
US10238700B2 (en) 2014-01-02 2019-03-26 Genelux Corporation Oncolytic virus adjunct therapy with agents that increase virus infectivity
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
HRP20201544T1 (hr) 2014-03-09 2021-02-19 The Trustees Of The University Of Pennsylvania Pripravci korisni u liječenju nedostatka ornitinske transkarbamilaze (otc)
WO2015137802A1 (en) 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
ES2856403T3 (es) 2014-03-18 2021-09-27 Univ Massachusetts Composiciones a base de rAAV y procedimientos para el tratamiento de la esclerosis lateral amiotrofica
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
ES2876409T3 (es) 2014-04-25 2021-11-12 Univ Pennsylvania Variantes del RLBD y su uso en composiciones para reducir los niveles de colesterol
US10975391B2 (en) 2014-04-25 2021-04-13 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
MY186389A (en) 2014-05-13 2021-07-22 Univ Pennsylvania Compositions comprising aav expressing dual antibody constructs and uses thereof
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
SG10201902574RA (en) 2014-09-24 2019-04-29 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
WO2016054557A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
SG11201702934TA (en) 2014-10-14 2017-05-30 Halozyme Inc Compositions of adenosine deaminase-2 (ada2), variants thereof and methods of using same
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
EP3690024A1 (en) 2014-11-05 2020-08-05 The Research Institute at Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
JP6401871B2 (ja) 2014-11-05 2018-10-10 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． パーキンソン病の治療のためのａａｄｃポリヌクレオチド
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
RU2749882C2 (ru) 2014-11-14 2021-06-18 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
CN107532173A (zh) 2014-11-21 2018-01-02 北卡罗来纳-查佩尔山大学 靶向中枢神经系统的aav载体
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
JP6754361B2 (ja) 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
WO2016110518A1 (en) 2015-01-07 2016-07-14 Universitat Autònoma De Barcelona Single-vector gene construct comprising insulin and glucokinase genes
AU2016206518B2 (en) 2015-01-16 2020-03-05 University Of Washington Novel micro-dystrophins and related methods of use
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
HK1249067A1 (zh) 2015-03-02 2018-10-26 Adverum Biotechnologies, Inc. 用於將多核苷酸玻璃體內遞送到視網膜視錐的組合物和方法
EP3268024B1 (en) 2015-03-10 2020-09-09 The Trustees of Columbia University in the City of New York Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression
MX388331B (es) 2015-03-24 2025-03-19 Univ California Variantes de virus adenoasociado y métodos de uso de estas
WO2016172008A1 (en) 2015-04-24 2016-10-27 University Of Massachusetts Modified aav constructions 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
AU2016256894B2 (en) 2015-05-07 2020-11-26 Massachusetts Eye And Ear Infirmary Methods of delivering an agent to the eye
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CA2985786A1 (en) * 2015-05-12 2016-11-17 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
CN108135994B (zh) 2015-05-13 2022-01-25 宾夕法尼亚州大学信托人 Aav-介导的抗-流感抗体的表达及其使用方法
US20170067028A1 (en) * 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
US10676735B2 (en) 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
US10738087B2 (en) * 2015-07-30 2020-08-11 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
JP7409773B2 (ja) 2015-07-31 2024-01-09 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 改変された細胞および治療の方法
KR102745604B1 (ko) 2015-08-06 2024-12-23 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Glp-1 및 대사성 질환 치료용 조성물에서 이의 용도
EP4177346A3 (en) 2015-08-25 2023-07-26 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
JP6877408B2 (ja) 2015-08-31 2021-05-26 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 ペット治療用ａａｖ−ｅｐｏ
WO2017053732A2 (en) 2015-09-24 2017-03-30 The Trustees Of The University Of Pennsylvania Composition and method for treating complement-mediated disease
CN108137655B (zh) 2015-09-28 2022-04-22 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
WO2017062750A1 (en) 2015-10-09 2017-04-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful in treating stargardt's disease and other ocular disorders
EP3362571A4 (en) 2015-10-13 2019-07-10 Duke University GENOM ENGINEERING WITH TYPE I CRISPR SYSTEMS IN EUKARYOTIC CELLS
WO2017066764A2 (en) * 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
EP3364996B1 (en) * 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070491A1 (en) * 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
EP3368563A1 (en) 2015-10-28 2018-09-05 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
SG11201802408RA (en) 2015-11-05 2018-05-30 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
US11167042B2 (en) 2015-12-11 2021-11-09 Massachusetts Eye And Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
IL259842B (en) * 2015-12-11 2022-08-01 California Inst Of Techn Targeting peptides for directing adeno-associated viruses -aavs-
US11015173B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
ES2918998T3 (es) 2015-12-11 2022-07-21 Univ Pennsylvania Método de purificación escalable para AAVrh10
WO2017160360A2 (en) 2015-12-11 2017-09-21 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
CA3008142A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
WO2017106202A2 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
CN109072254A (zh) * 2015-12-14 2018-12-21 宾夕法尼亚州大学信托人 用于治疗脊髓性肌萎缩的腺相关病毒载体
JP7061067B2 (ja) 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 クリグラー・ナジャー症候群の処置のための組成物
EP3411059A4 (en) 2016-02-02 2019-10-16 University Of Massachusetts METHOD FOR INCREASING THE EFFECTIVENESS OF THE SYSTEMIC DELIVERY OF AVV GENES TO THE CENTRAL NERVOUS SYSTEM
BR112018015751A2 (pt) 2016-02-03 2019-02-05 Univ Pennsylvania terapia gênica para tratamento de mucopolissacaridose tipo i
JP2019504888A (ja) 2016-02-05 2019-02-21 エモリー ユニバーシティ 一本鎖アデノ随伴ウイルス９または自己相補型アデノ随伴ウイルス９の脳脊髄液への注射
EP4094780A3 (en) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
CA3019847A1 (en) 2016-04-04 2017-10-12 University Of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
IL262366B2 (en) 2016-04-15 2024-07-01 Alpine Immune Sciences Inc Immunomodulatory proteins and CD80 variants and their uses
EP3443108A4 (en) * 2016-04-15 2019-11-20 The Trustees of The University of Pennsylvania NOVEL AAV8 MUTANT CAPSIDES AND COMPOSITIONS THEREOF
CA3019199A1 (en) 2016-04-15 2017-10-19 Alpine Immune Sciences, Inc. Icos ligand variant immunomodulatory proteins and uses thereof
CN109562191A (zh) 2016-04-15 2019-04-02 宾夕法尼亚州大学信托人 用于治疗血友病a的基因疗法
KR20250036262A (ko) 2016-04-15 2025-03-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
EP3452103A1 (en) 2016-04-15 2019-03-13 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
WO2017184463A1 (en) 2016-04-17 2017-10-26 The Trustees Of The University Of Pennsylvania Compositions and methods useful for prophylaxis of organophosphates
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017192699A1 (en) * 2016-05-03 2017-11-09 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
CR20180589A (es) 2016-05-13 2019-02-27 4D Molecular Therapeutics Inc Cápsulas variantes de virus adenoasociados y métodos de uso de estas
JP7220080B2 (ja) 2016-05-18 2023-02-09 ボイジャー セラピューティクス インコーポレイテッド ハンチントン病治療組成物及び方法
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
WO2017218450A1 (en) 2016-06-13 2017-12-21 The University Of North Carolina At Chapel Hill Optimized cln1 genes and expression cassettes and their use
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
EP3481958A4 (en) 2016-07-08 2019-12-25 The Trustees of The University of Pennsylvania METHOD AND COMPOSITIONS FOR TREATING DISTURBANCES AND DISEASES WITH RDH12
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University 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
EP3491008A2 (en) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
WO2018022945A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd112 variant immunomodulatory proteins and uses thereof
CA3032120A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd155 variant immunomodulatory proteins and uses thereof
US11471488B2 (en) 2016-07-28 2022-10-18 Alpine Immune Sciences, Inc. CD155 variant immunomodulatory proteins and uses thereof
CN114672516B (zh) 2016-07-29 2024-11-26 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
JP2019524162A (ja) 2016-08-18 2019-09-05 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 モジュラーＡＡＶ送達システムによるＣＲＩＳＰＲ−Ｃａｓゲノム編集
US11298041B2 (en) 2016-08-30 2022-04-12 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
CN110506119A (zh) * 2016-10-13 2019-11-26 马萨诸塞大学 Aav衣壳设计
AU2017346885B2 (en) 2016-10-18 2021-10-21 Intima Bioscience, Inc. Tumor infiltrating lymphocytes and methods of therapy
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
EP3872180A1 (en) 2016-10-20 2021-09-01 Alpine Immune Sciences, Inc. Secretable variant immunomodulatory proteins and engineered cell therapy
WO2018083606A1 (en) 2016-11-01 2018-05-11 Novartis Ag Methods and compositions for enhancing gene editing
EP3548065B1 (en) 2016-12-01 2022-11-09 INSERM - Institut National de la Santé et de la Recherche Médicale Pharmaceutical compositions for the treatment of retinal degenerative diseases
CA3048044A1 (en) 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating wilson's disease
WO2018126112A1 (en) 2016-12-30 2018-07-05 The Trustees Of The University Of Pennsylvania Gene therapy for treating phenylketonuria
WO2018134168A1 (en) 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
EP3576760A2 (en) 2017-02-01 2019-12-11 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
AU2018219895C1 (en) 2017-02-09 2024-10-03 Indapta Therapeutics, Inc. Engineered Natural Killer (NK) cells and compositions and methods thereof
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated PROTEINS OF MODIFIED AAV CAPSIDES AND THEIR USES
US11518991B2 (en) 2017-02-24 2022-12-06 Georg-August-Universitaet Goettingen Stiftung Oeffentlichen Rechts, Universitaetsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated RASAL1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including RASAL1 in a subject
JP2020510430A (ja) 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 Ａａｖベクターに基づくインフルエンザワクチン
PE20191362A1 (es) * 2017-02-28 2019-10-01 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
EP3589738A4 (en) 2017-03-01 2021-01-06 The Trustees of The University of Pennsylvania Gene therapy for ocular disorders
WO2018158397A1 (en) 2017-03-02 2018-09-07 Genethon Method for removing anti-aav antibodies from a blood-derived composition
KR20250083578A (ko) 2017-03-16 2025-06-10 알파인 이뮨 사이언시즈, 인코포레이티드 Cd80 변이체 면역조절 단백질 및 그의 용도
IL320149A (en) 2017-03-16 2025-06-01 Alpine Immune Sciences Inc Pd-l1 variant immunomodulatory proteins and uses thereof
EP3596115A1 (en) 2017-03-16 2020-01-22 Alpine Immune Sciences, Inc. Pd-l2 variant immunomodulatory proteins and uses thereof
SG10202110530WA (en) 2017-03-24 2021-11-29 Lankenau Inst Medical Res Methods and compositions for inducible extracellular membrane capture of monoclonal immunoglobulins secreted by hybridomas
US20210108197A1 (en) 2017-03-31 2021-04-15 Staidson (Beijing) Biopharmaceuticals Co., Ltd. Shrna expression cassette, polynucleotide sequence carrying same, and application thereof
WO2018189208A1 (en) 2017-04-10 2018-10-18 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
BR112019021569A2 (pt) 2017-04-14 2020-05-12 Regenxbio Inc. Precursor de iduronato-2-sulfatase humana recombinante glicosilada (ids), método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo ii (mps ii)
CA3060250A1 (en) * 2017-04-17 2018-10-25 University Of Florida Research Foundation, Incorporated Regulation of ran protein translation by pkr and eif2a-p pathways
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
JP2020518258A (ja) 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． 筋萎縮性側索硬化症（ａｌｓ）治療組成物および方法
JP7327803B2 (ja) 2017-05-09 2023-08-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症（ａｌｓ）を処置する方法
CA3061968A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses
IL270422B2 (en) 2017-05-11 2025-04-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis
WO2018218359A1 (en) 2017-05-31 2018-12-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating peroxisomal disorders
EP3638316A4 (en) 2017-06-14 2021-03-24 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR EYE DISORDERS
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2019006418A2 (en) * 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
WO2019006182A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF
BR112020000063A2 (pt) 2017-07-06 2020-07-14 The Trustees Of The University Of Pennsylvania terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i
EP4450078A3 (en) 2017-07-07 2025-01-08 Genethon Novel polynucleotides encoding a human fkrp protein
US11497576B2 (en) 2017-07-17 2022-11-15 Voyager Therapeutics, Inc. Trajectory array guide system
KR20200032722A (ko) 2017-07-27 2020-03-26 노파르티스 아게 부분절단효소 내성 trem2 변이체
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN110709511A (zh) 2017-08-28 2020-01-17 加利福尼亚大学董事会 腺相关病毒衣壳变体及其使用方法
MY199445A (en) 2017-09-20 2023-10-28 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
EP3684937A4 (en) 2017-09-22 2021-06-02 University of Massachusetts NEW DUAL SOD1 EXPRESSION VECTORS AND RELATED USES
US11903910B2 (en) 2017-09-26 2024-02-20 University Of Florida Research Foundation, Incorporated Use of metformin and analogs thereof to reduce RAN protein levels in the treatment of neurological disorders
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
EP4124658A3 (en) 2017-10-16 2023-04-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
KR20250034197A (ko) 2017-10-18 2025-03-10 알파인 이뮨 사이언시즈, 인코포레이티드 변이체 icos 리간드 면역조절 단백질 및 관련 조성물 및 방법
CN111727201A (zh) 2017-10-18 2020-09-29 再生生物股份有限公司 完全人源翻译后修饰的抗体治疗剂
MX2020003945A (es) 2017-10-18 2020-11-09 Regenxbio Inc Tratamiento de enfermedades oculares y cancer de colon metastasico con vegf-trap humano modificado post-traduccionalmente .
US11723988B2 (en) 2017-10-20 2023-08-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
JP7184894B2 (ja) 2017-11-27 2022-12-06 ４ディー モレキュラー セラピューティクス インコーポレイテッド アデノ随伴ウイルス変異キャプシドおよび血管新生の阻害のための使用
KR20200104864A (ko) 2017-11-30 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 뮤코다당류증 iiib형에 대한 유전자 요법
CA3083416A1 (en) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iii a
SG11202005618WA (en) 2017-12-19 2020-07-29 Akouos Inc Aav-mediated delivery of therapeutic antibodies to the inner ear
AU2019205273B2 (en) 2018-01-03 2024-04-04 Alpine Immune Sciences, Inc. Multi-domain immunomodulatory proteins and methods of use thereof
CN111601620A (zh) 2018-01-17 2020-08-28 阿瑞纳斯治疗公司 用于21-羟化酶缺乏症的腺相关病毒基因疗法
PE20212076A1 (es) 2018-02-01 2021-10-26 Homology Medicines Inc Composiciones de virus adeno-asociado para restaurar la funcion del gen pah y metodos de uso de las mismas
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
IL276859B1 (en) * 2018-02-27 2025-08-01 Univ Pennsylvania Novel adeno-associated virus (AAV) vectors, AAV vectors with reduced capsid deamidation and uses therefor
MX2020008932A (es) * 2018-02-27 2020-10-01 Univ Pennsylvania Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
AU2019234918B2 (en) * 2018-03-16 2025-08-14 Research Institute At Nationwide Children's Hospital Increasing tissue specific gene delivery by capsid modification
JP7425738B2 (ja) 2018-04-03 2024-01-31 ギンコ バイオワークス インコーポレイテッド 眼組織を標的とするウイルスベクター
CA3094311A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
KR20210007963A (ko) 2018-04-03 2021-01-20 스트라이드바이오 인코포레이티드 항체-회피 바이러스 벡터
WO2019193119A1 (en) 2018-04-05 2019-10-10 Genethon HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM
EP3775266A4 (en) 2018-04-05 2021-06-30 Massachusetts Eye and Ear Infirmary METHOD OF MANUFACTURING AND USING COMBINATIONAL BAR-CODED NUCLEIC ACID LIBRARIES WITH DEFINED VARIATION
US20210100917A1 (en) 2018-04-18 2021-04-08 The Trustees Of Columbia University In The City Of New York Gene therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes
AU2019261646A1 (en) 2018-04-27 2020-11-12 Spacecraft Seven, Llc Gene therapy for CNS degeneration
US12365865B2 (en) 2018-04-29 2025-07-22 Regenxbio Inc. Scalable clarification process for recombinant AAV production
WO2019212922A1 (en) 2018-04-29 2019-11-07 Regenxbio Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
KR20210006352A (ko) 2018-05-08 2021-01-18 루트거스, 더 스테이트 유니버시티 오브 뉴 저지 Aav-상용성 라미닌-링커 중합 단백질
JP2021522811A (ja) 2018-05-09 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド フェニルケトン尿症の治療方法
JP7438981B2 (ja) 2018-05-11 2024-02-27 マサチューセッツ アイ アンド イヤー インファーマリー アデノ随伴ウイルスの肝臓特異的向性
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2019268330A1 (en) 2018-05-15 2020-11-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of Parkinson's disease
WO2019221992A1 (en) 2018-05-15 2019-11-21 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
AR126019A1 (es) 2018-05-30 2023-09-06 Novartis Ag Anticuerpos frente a entpd2, terapias de combinación y métodos de uso de los anticuerpos y las terapias de combinación
KR20240060879A (ko) 2018-06-04 2024-05-08 카리디 바이오테라퓨틱스, 인크. 바이러스 치료법의 강화를 위한 세포-기반 비히클
US11505782B2 (en) 2018-06-04 2022-11-22 Calidi Biotherapeutics, Inc. Cell-based vehicles for potentiation of viral therapy
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
WO2019241315A1 (en) 2018-06-12 2019-12-19 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
IL319002A (en) 2018-06-14 2025-04-01 Regenxbio Inc Anion exchange chromatography for recombinant AAV production
US12065476B2 (en) 2018-06-15 2024-08-20 Alpine Immune Sciences, Inc. PD-1 variant immunomodulatory proteins and uses thereof
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
CN112512596A (zh) 2018-07-12 2021-03-16 火箭制药有限公司 治疗danon病的基因治疗载体
US11510999B2 (en) 2018-07-17 2022-11-29 Helixmith Co., Ltd Treatment of neuropathy with DNA constructs expressing IGF-1 isoforms
WO2020018691A1 (en) * 2018-07-18 2020-01-23 The General Hospital Corporation Modified t cells and methods of their use
MX2021000810A (es) 2018-07-24 2021-04-28 Voyager Therapeutics Inc Sistemas y metodos para producir formulaciones de terapia genetica.
US20210277418A1 (en) * 2018-08-03 2021-09-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
JP2021533757A (ja) 2018-08-10 2021-12-09 リジェネクスバイオ インコーポレイテッド 組換えａａｖ生成のためのスケーラブルな方法
EP3844276A2 (en) 2018-08-28 2021-07-07 Actym Therapeutics, Inc. Engineered immunostimulatory bacterial strains and uses thereof
AU2019333315B2 (en) 2018-08-30 2022-09-29 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with myocardin and ASCL1
AU2019346447A1 (en) 2018-09-26 2021-04-29 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
KR20210068068A (ko) 2018-09-28 2021-06-08 보이저 테라퓨틱스, 인크. 조작된 프로모터를 갖는 프라탁신 발현 구축물 및 그의 사용 방법
AU2019351815A1 (en) 2018-10-01 2021-05-06 The Trustees Of The University Of Pennsylvania Compositions useful for treating GM1 gangliosidosis
CN112752583A (zh) 2018-10-01 2021-05-04 宫崎彻 神经变性疾病治疗剂
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
TW202028458A (zh) * 2018-10-05 2020-08-01 美商航海家醫療公司 編碼腺相關病毒(aav)生產蛋白之經基因工程化核酸構築體
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
US20210324483A1 (en) 2018-10-15 2021-10-21 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
CN113166781A (zh) 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体
US20210363509A1 (en) 2018-10-22 2021-11-25 University Of Rochester Genome Editing by Directed Non-Homologous DNA Insertion Using a Retroviral Integrase-Cas9 Fusion Protein
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
WO2020097269A1 (en) 2018-11-06 2020-05-14 Calidi Biotherapeutics, Inc. Enhanced systems for cell-mediated oncolytic viral therapy
KR20210093954A (ko) 2018-11-16 2021-07-28 아스텔라스세이야쿠 가부시키가이샤 유트로핀 유전자를 표적으로 하여 근이영양증을 치료하는 방법
CN113891934A (zh) 2018-11-21 2022-01-04 因达普塔治疗公司 扩增天然杀伤（nk）细胞子集的方法以及相关组合物和方法
US20220001028A1 (en) * 2018-11-30 2022-01-06 Novartis Ag Aav viral vectors and uses thereof
WO2020113141A2 (en) 2018-11-30 2020-06-04 Alpine Immune Sciences, Inc. Cd86 variant immunomodulatory proteins and uses thereof
US20220089670A1 (en) 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
DK3906066T5 (da) 2019-01-04 2024-08-05 Ultragenyx Pharmaceutical Inc Geneterapikonstruktioner til behandling af wilsons sygdom
MX2021008487A (es) 2019-01-14 2021-11-12 Univ Rochester Escisión y poliadenilación del arn nuclear dirigido con crispr-cas.
MX2021008542A (es) 2019-01-18 2021-11-12 Voyager Therapeutics Inc Métodos y sistemas para la producción de partículas de aav.
EP3917566A4 (en) 2019-01-31 2022-10-26 Oregon Health & Science University METHODS OF USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS
SG11202108504YA (en) 2019-02-22 2021-09-29 Univ Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
US20220154210A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
JP2022520875A (ja) 2019-02-25 2022-04-01 ノバルティス アーゲー Ｂｉｅｔｔｉクリスタリン網膜症を治療するための組成物及び方法
MX2021010266A (es) 2019-02-26 2021-09-23 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
CN113748124A (zh) 2019-02-27 2021-12-03 阿克蒂姆治疗有限公司 工程化以定植肿瘤、肿瘤驻留免疫细胞和肿瘤微环境的免疫刺激性细菌
US12024709B2 (en) 2019-02-27 2024-07-02 Actym Therapeutics, Inc. Immunostimulatory bacteria engineered to colonize tumors, tumor-resident immune cells, and the tumor microenvironment
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
CA3130196A1 (en) 2019-03-25 2020-10-01 Isabelle Richard Production of large-sized quasidystrophins using overlapping aav vectors
EP3947700A4 (en) 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
AU2020253462A1 (en) 2019-04-03 2021-10-28 Regenxbio Inc. Gene therapy for eye pathologies
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
EP3953483B1 (en) 2019-04-11 2023-10-04 REGENXBIO Inc. Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions
JP7630443B2 (ja) 2019-04-19 2025-02-17 レジェンクスバイオ インコーポレーテッド アデノ随伴ウイルスベクター製剤及び方法
TW202106708A (zh) 2019-04-24 2021-02-16 美商銳進科斯生物股份有限公司 全人類轉譯後修飾之抗體治療劑
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
MX2021013268A (es) * 2019-04-29 2021-11-17 Univ Pennsylvania Nuevas capsides de aav y composiciones que las contienen.
BR112021021908A2 (pt) 2019-05-03 2022-02-01 Univ Pennsylvania Composições úteis no tratamento da leucodistrofia metacromática
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
EP3969061A1 (en) 2019-05-14 2022-03-23 BioMarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
MX2021014478A (es) 2019-05-28 2022-01-06 Astellas Pharma Inc Metodo para tratar distrofia muscular por direccionamiento del gen dmpk.
KR20220016150A (ko) 2019-05-30 2022-02-08 솔리드 바이오사이언시즈 인크. 재조합 헤르페스비랄레스 벡터
US20230241248A1 (en) 2019-06-27 2023-08-03 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
AU2020298575A1 (en) 2019-07-02 2022-02-24 M6P Therapeutics (Switzerland) GmbH Vector compositions and methods of using same for treatment of lysosomal storage disorders
EP3997225A1 (en) 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
US20230137971A1 (en) 2019-07-11 2023-05-04 Tenaya Therapeutics Inc. Cardiac cell reprogramming with micrornas and other factors
ES2964907T3 (es) 2019-07-11 2024-04-10 Centre Nat Rech Scient Virus adenoasociado modificado químicamente
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
US10557149B1 (en) 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
US10801042B1 (en) 2019-07-15 2020-10-13 Vigene Biosciences, Inc. Use of ion concentrations to increase the packaging efficiency of recombinant adeno-associated virus
WO2021016453A1 (en) 2019-07-23 2021-01-28 University Of Rochester Targeted rna cleavage with crispr-cas
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
EP4004214A1 (en) 2019-07-26 2022-06-01 RegenxBio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
US20220290182A1 (en) 2019-08-09 2022-09-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
JP7641644B2 (ja) 2019-08-16 2025-03-07 株式会社モダリス Ｌａｍａ１遺伝子を標的とした筋ジストロフィーの治療方法
WO2021034920A1 (en) * 2019-08-20 2021-02-25 St. Jude Children's Research Hospital, Inc. SCHIZOPHRENIA-RELATED MICRODELETION GENE 2510002D24Rik IS ESSENTIAL FOR SOCIAL MEMORY
CN114502197A (zh) 2019-08-26 2022-05-13 再生生物股份有限公司 用全人经翻译后修饰的抗VEGF Fab治疗糖尿病性视网膜病变
WO2021040736A1 (en) 2019-08-30 2021-03-04 Obsidian Therapeutics, Inc. Tandem cd19 car-based compositions and methods for immunotherapy
US20220307013A1 (en) 2019-08-30 2022-09-29 The Regents Of The University Of California Gene fragment overexpression screening methodologies, and uses thereof
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021046451A1 (en) 2019-09-06 2021-03-11 Obsidian Therapeutics, Inc. Compositions and methods for dhfr tunable protein regulation
CA3150330A1 (en) 2019-09-13 2021-03-18 Peter D. YURCHENCO Aav-compatible laminin-linker polymerization proteins
TW202124446A (zh) 2019-09-18 2021-07-01 瑞士商諾華公司 與ｅｎｔｐｄ２抗體之組合療法
WO2021053559A1 (en) 2019-09-18 2021-03-25 Novartis Ag Entpd2 antibodies, combination therapies, and methods of using the antibodies and combination therapies
WO2021053124A1 (en) 2019-09-19 2021-03-25 Genethon Gene therapy expression system alleviating cardiac toxicity of fkrp
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
WO2021071835A1 (en) 2019-10-07 2021-04-15 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
JP7640538B2 (ja) 2019-10-08 2025-03-05 トラスティーズ オブ ボストン カレッジ 複数の異なる非天然アミノ酸を含有するタンパク質、並びにそのようなタンパク質の製造方法及び使用方法
WO2021071976A1 (en) 2019-10-08 2021-04-15 Exhaura, Ltd. Compositions and methods for ocular therapy
EP4045093A4 (en) 2019-10-14 2024-02-07 Duke University Compositions comprising novel prokaryotic sodium channels and associated methods
JP2022551739A (ja) * 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
WO2021092300A1 (en) * 2019-11-08 2021-05-14 President And Fellows Of Harvard College Viral capsid polypeptides
US20230002451A1 (en) * 2019-11-08 2023-01-05 President And Fellows Of Harvard College Viral capsid polypeptides
EP4061943A1 (en) 2019-11-19 2022-09-28 Institut National de la Santé et de la Recherche Médicale (INSERM) Antisense oligonucleotides and their use for the treatment of cancer
BR112022009895A2 (pt) * 2019-11-28 2022-08-09 Regenxbio Inc Construtos de terapia de gene de microdistrofina e usos dos mesmos
EP4073257A1 (en) 2019-12-09 2022-10-19 UCL Business Ltd Gene therapy composition and treatment for myh7-linked cardiomyopathy
CA3164189A1 (en) 2019-12-10 2021-06-17 Takeda Pharmaceutical Company Limited Adeno associated virus vectors for the treatment of hunter disease
JP2023509443A (ja) 2019-12-31 2023-03-08 スワンバイオ セラピューティクス リミテッド 改善されたａａｖ－ａｂｃｄ１コンストラクトならびに副腎白質ジストロフィー（ａｌｄ）および／または副腎脊髄ニューロパチー（ａｍｎ）の処置または予防のための使用
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
WO2021150570A1 (en) 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
AU2021212749A1 (en) 2020-01-29 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis IVA
JP2023512242A (ja) 2020-01-29 2023-03-24 レジェンクスバイオ インコーポレーテッド ヒト神経細胞またはヒトグリア細胞が産生した組換えヒトイズロン酸－２－スルファターゼ（ｉｄｓ）によるムコ多糖症ｉｉの治療
CN115867323A (zh) 2020-02-02 2023-03-28 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂症的组成物
WO2021158982A2 (en) 2020-02-07 2021-08-12 University Of Rochester Targeted translation of rna with crispr-cas13 to enhance protein synthesis
US20230073250A1 (en) 2020-02-07 2023-03-09 University Of Rochester Ribozyme-mediated RNA Assembly and Expression
EP4103724A1 (en) 2020-02-14 2022-12-21 Ultragenyx Pharmaceutical Inc. Gene therapy for treating cdkl5 deficiency disorder
WO2021170784A1 (en) 2020-02-28 2021-09-02 INSERM (Institut National de la Santé et de la Recherche Médicale) Gene therapy for maple syrup urine disease
AU2021230476A1 (en) 2020-03-02 2022-10-20 Tenaya Therapeutics, Inc. Gene vector control by cardiomyocyte-expressed microRNAs
WO2022074464A2 (en) 2020-03-05 2022-04-14 Neotx Therapeutics Ltd. Methods and compositions for treating cancer with immune cells
CA3171066A1 (en) 2020-03-11 2021-09-16 Saar GILL Methods and composition for gene delivery using an engineered viral particle
WO2021188892A1 (en) 2020-03-19 2021-09-23 Ultragenyx Pharmaceutical Inc. Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav
EP4126936A1 (en) 2020-03-27 2023-02-08 UCB Biopharma SRL Autonomous knob domain peptides
US20240218367A1 (en) 2020-03-27 2024-07-04 University Of Rochester Targeted Destruction of Viral RNA by CRISPR-Cas13
US20230103771A1 (en) 2020-03-27 2023-04-06 University Of Rochester CRISPR-Cas13 crRNA Arrays
JP2023520402A (ja) 2020-03-31 2023-05-17 ウルトラジェニックス ファーマシューティカル インコーポレイテッド プロピオン酸血症を処置するための遺伝子治療
US20230131352A1 (en) 2020-04-01 2023-04-27 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
AU2021254272A1 (en) 2020-04-10 2022-10-27 Sola Biosciences Llc Compositions and methods for the treatment of protein aggregation disorders
JP7713956B2 (ja) 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 タウ結合化合物
KR20230004719A (ko) * 2020-04-20 2023-01-06 테나야 테라퓨틱스, 인코포레이티드 조작된 캡시드를 갖는 아데노-연관 바이러스
EP4139439A1 (en) 2020-04-22 2023-03-01 Indapta Therapeutics, Inc. Natural killer (nk) cell compositions and methods for generating same
MX2022013288A (es) 2020-04-28 2023-02-22 Sola Biosciences Llc Composiciones y metodos para el tratamiento de proteinopatias por la proteina 43 de union a acido desoxirribonucleico (adn) de respuesta transactiva (tdp-43).
AR122013A1 (es) * 2020-05-05 2022-08-03 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
AU2021271635A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
US20230212606A1 (en) 2020-05-13 2023-07-06 Akouos, Inc. Compositions and methods for treating kcnq4-associated hearing loss
TW202208405A (zh) 2020-05-13 2022-03-01 美商阿科奧斯公司 用於治療ｓｌｃ２６ａ４相關聽力損失之組合物及方法
KR20230022175A (ko) 2020-05-13 2023-02-14 보이저 테라퓨틱스, 인크. Aav 캡시드의 향성 방향변경
WO2021230385A1 (en) 2020-05-15 2021-11-18 Astellas Pharma Inc. Method for treating muscular dystrophy by targeting utrophin gene
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
CN116096737A (zh) 2020-06-05 2023-05-09 索拉生物科学有限公司 用于治疗突触核蛋白病的组合物和方法
US20230220069A1 (en) 2020-06-17 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of gene therapy patients
EP4168052A2 (en) 2020-06-19 2023-04-26 Genethon Gene therapy expression system allowing an adequate expression in the muscles and in the heart of sgcg
EP4176064A1 (en) * 2020-07-03 2023-05-10 Genethon Method for engineering novel hybrid aav capsids through hypervariable regions swapping
US20230241249A1 (en) * 2020-07-07 2023-08-03 The Board Of Regents Of The University Of Texas System Adeno-associated virus vector for dwarf open reading frame
WO2022011262A1 (en) 2020-07-10 2022-01-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
CN116194587A (zh) 2020-07-10 2023-05-30 吉尼松公司 一种新的肌肉特异性启动子
CN116438311A (zh) 2020-07-13 2023-07-14 宾夕法尼亚州大学信托人 可用于治疗夏科-马里-图思病的组合物
JP2023534464A (ja) 2020-07-15 2023-08-09 ユニバーシティ オブ ロチェスター ｄＣａｓ１３－ＲＮａｓｅ融合タンパク質による標的ＲＮＡの切断
IL299928A (en) 2020-07-27 2023-03-01 Voyager Therapeutics Inc Preparations and methods for treating neurological disorders associated with glucosylcremidase beta deficiency
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
CN111876432B (zh) * 2020-07-29 2022-10-28 舒泰神（北京）生物制药股份有限公司 一组肝靶向新型腺相关病毒的获得及其应用
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
CN116406305A (zh) 2020-08-17 2023-07-07 麻省理工学院 Shank3基因治疗方法
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
WO2022046815A1 (en) 2020-08-24 2022-03-03 The Trustees Of The University Of Pennsylvania Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
CN116887867A (zh) 2020-08-26 2023-10-13 宾夕法尼亚州大学信托人 用于治疗grn相关成人发作性神经退行性病变的重组腺相关病毒
KR20230061474A (ko) 2020-09-04 2023-05-08 고쿠리츠다이가쿠호진 고베다이가쿠 소형화 시티딘 데아미나아제를 포함하는 이중쇄 dna의 개변용 복합체
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
US20230383278A1 (en) 2020-09-18 2023-11-30 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
KR20230082614A (ko) 2020-10-07 2023-06-08 리젠엑스바이오 인크. 유전자 요법을 안구 전달하기 위한 아데노-연관 바이러스
CA3198372A1 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
US20230414788A1 (en) 2020-10-07 2023-12-28 Regenxbio Inc. Formulations for suprachoroidal administration such as gel formulations
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
JP2023544803A (ja) 2020-10-07 2023-10-25 レジェンクスバイオ インコーポレーテッド Ｃｌｎ２疾患の眼症状に対する遺伝子療法
US20230372538A1 (en) 2020-10-07 2023-11-23 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
US20230365955A1 (en) 2020-10-09 2023-11-16 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
AU2021359874A1 (en) 2020-10-18 2023-05-25 The Trustees Of The University Of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
TW202233841A (zh) 2020-10-28 2022-09-01 美商銳進科斯生物股份有限公司 用於眼適應症之載體化抗ＴＮＦ－α抗體
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
WO2022094255A2 (en) 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
US20240384292A1 (en) 2020-10-29 2024-11-21 Regenxbio Inc. Vectorized tnf-alpha antagonists for ocular indications
AU2021372262B9 (en) 2020-11-02 2025-05-15 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
JP2023548816A (ja) 2020-11-03 2023-11-21 ファイザー・インク 陰イオン交換クロマトグラフィーによるａａｖベクターの精製方法
EP4255500A1 (en) 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
MX2023006444A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen.
TW202237644A (zh) 2020-12-01 2022-10-01 美商阿科奧斯公司 用於治療前庭神經鞘瘤相關症狀之抗ｖｅｇｆ抗體構築體及相關方法
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
MX2023007315A (es) 2020-12-16 2023-08-22 Regenxbio Inc Método para producir una partícula de virus adenoasociado recombinante.
AU2021404944A1 (en) 2020-12-23 2023-07-06 Pfizer Inc. Methods for purification of aav vectors by affinity chromatography
JP2024500786A (ja) 2020-12-29 2024-01-10 アコーオス インコーポレイテッド Ｃｌｒｎ１関連難聴及び／または視力低下を治療するための組成物及び方法
US20240309076A1 (en) 2020-12-29 2024-09-19 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
TW202241479A (zh) 2020-12-30 2022-11-01 美商安迅生物製藥公司 遞送外來抗原之溶瘤病毒及表現靶向外來抗原之嵌合受體之經工程化免疫細胞之組合療法
JP2024504613A (ja) 2021-01-14 2024-02-01 センティ バイオサイエンシズ インコーポレイテッド 分泌性ペイロード調節
EP4281568A1 (en) 2021-01-21 2023-11-29 RegenxBio Inc. Improved production of recombinant polypeptides and viruses
EP4281551A4 (en) * 2021-01-25 2025-06-04 Trames Bio, Inc. ADENO-ASSOCIATED VIRUS CAPSIDS AND MANIPULATED LIGAND-GATED ION CHANNELS FOR THE TREATMENT OF FOCAL EPILEPSY AND NEUROPATHIC PAIN
US20240091380A1 (en) 2021-02-01 2024-03-21 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022169774A1 (en) * 2021-02-02 2022-08-11 University Of Massachusetts Inverted terminal repeats from aav serotypes 8 and rh.39 in gene therapy vectors
WO2022170146A1 (en) 2021-02-05 2022-08-11 Maze Therapeutics, Inc. Vectors comprising stuffer polynucleotide sequences
EP4291215A2 (en) * 2021-02-09 2023-12-20 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2022173605A2 (en) 2021-02-10 2022-08-18 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
PE20240806A1 (es) 2021-02-26 2024-04-18 Takeda Pharmaceuticals Co Composicion y metodos para el tratamiento de la enfermedad de fabry
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4308711A1 (en) 2021-03-19 2024-01-24 Adrenas Therapeutics, Inc. Gene therapies for 21-hydroxylase deficiency
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
WO2022216749A1 (en) 2021-04-05 2022-10-13 Solid Biosciences Inc. Recombinant herpesvirales vector
EP4323520A1 (en) 2021-04-12 2024-02-21 The Trustees of The University of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
JP2024515715A (ja) 2021-04-23 2024-04-10 ユニバーシティ オブ ロチェスター レトロウイルスインテグラーゼ－Ｃａｓ融合タンパク質を使用した指向性非相同ＤＮＡ挿入によるゲノム編集及び治療の方法
BR112023021971A2 (pt) 2021-04-23 2024-02-20 Univ Pennsylvania Composições com motivos de direcionamento específicos do cérebro e composições contendo os mesmos
JP2024515902A (ja) 2021-04-26 2024-04-10 アレクシオン ファーマ インターナショナル オペレーションズ リミテッド 組織向性が改善されたアデノ随伴ウイルスベクターカプシド
JP2024517143A (ja) 2021-04-26 2024-04-19 リジェネックスバイオ インコーポレイテッド ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与
EP4334454A2 (en) 2021-05-04 2024-03-13 RegenxBio Inc. Novel aav vectors and methods and uses thereof
EP4337267A1 (en) 2021-05-11 2024-03-20 RegenxBio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
JP2024520815A (ja) * 2021-06-08 2024-05-24 エヌエフ２ セラピューティクス， インコーポレイテッド 神経線維腫性障害を処置するための組成物および方法
CN117957324A (zh) * 2021-06-18 2024-04-30 迪诺治疗公司 衣壳变体及其使用方法
CA3224755A1 (en) 2021-06-22 2022-12-29 Pfizer Inc. Production of adeno-associated virus vector in insect cells
WO2022272056A2 (en) * 2021-06-24 2022-12-29 University Of Utah Research Foundation Compositions and methods for treating pgm1 deficiency
EP4362957A1 (en) 2021-07-01 2024-05-08 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
CA3224488A1 (en) * 2021-07-08 2023-01-12 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
CA3226452A1 (en) 2021-07-19 2023-01-26 New York University Auf1 combination therapies for treatment of muscle degenerative disease
CN118076744A (zh) * 2021-07-23 2024-05-24 杜克大学 腺相关病毒组合物及其使用方法
KR20240056500A (ko) 2021-07-30 2024-04-30 토루 미야자키 허혈성 질환에 대한 치료제
WO2023019226A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
JP2024534771A (ja) 2021-08-11 2024-09-26 サナ バイオテクノロジー，インコーポレイテッド 補体媒介性炎症反応を低減するための同種異系細胞療法のための遺伝子改変細胞
US20250127820A1 (en) 2021-08-11 2025-04-24 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
WO2023019229A1 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified primary cells for allogeneic cell therapy
KR20240078666A (ko) * 2021-08-25 2024-06-04 캔브리지 파마슈티컬스, 인크. 간-향성 캡시드 단백질 및 산 알파-글루코시다제(gaa)를 포함하는 aav 입자 및 폼페 질환을 치료하기 위한 이의 용도
AU2022332298A1 (en) * 2021-08-25 2024-03-14 Canbridge Pharmaceuticals, Inc. Aav particles comprising a liver-tropic capsid protein and alpha-galactosidase and their use to treat fabry disease
IL311096A (en) 2021-08-31 2024-04-01 Scout Bio Inc Antigen binding molecules and their uses
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
PE20241065A1 (es) 2021-09-30 2024-05-13 Akouos Inc Composiciones y metodos para el tratamiento de la perdida de audicion asociada a kcnq4
EP4409010A1 (en) 2021-10-02 2024-08-07 The Trustees of The University of Pennsylvania Novel aav capsids and compositions containing same
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
EP4413147A4 (en) 2021-10-08 2025-07-30 Dyno Therapeutics Inc CAPSID VARIANTS AND METHODS OF USE THEREOF
CN118488962A (zh) 2021-10-08 2024-08-13 索拉生物科学有限公司 用于治疗p53介导的癌症的组合物和方法
WO2023060221A2 (en) 2021-10-08 2023-04-13 Sola Biosciences Llc Compositions and methods for the treatment of proteopathies
CA3235145A1 (en) 2021-10-12 2023-04-20 Bridgebio Gene Therapy Llc Methods and compositions for treating leukodystrophies
JP2024540972A (ja) 2021-10-20 2024-11-06 ユニバーシティ オブ ロチェスター 加齢性白質喪失の若返り治療
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
US20230270818A1 (en) 2021-11-02 2023-08-31 University Of Rochester Tcf7l2 mediated remyelination in the brain
AU2022379918A1 (en) 2021-11-02 2024-04-18 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023087019A2 (en) 2021-11-15 2023-05-19 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023092004A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
EP4433169A1 (en) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CN118525098A (zh) 2021-11-29 2024-08-20 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
US20230323395A1 (en) * 2021-12-15 2023-10-12 Homology Medicines, Inc. Methods and compositions for the production of adeno-associated virus
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
WO2023133595A2 (en) 2022-01-10 2023-07-13 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023135316A1 (en) 2022-01-17 2023-07-20 Dinaqor Ag Gene therapy composition and treatment for dystrophin-related cardiomyopathy
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
MX2024009186A (es) 2022-01-25 2024-09-30 Univ Pennsylvania Cápsides de aav para mejorar la transducción cardiaca y desdireccionamiento del hígado.
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
EP4473097A1 (en) 2022-02-02 2024-12-11 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2023150142A1 (en) 2022-02-02 2023-08-10 Akouos, Inc. Anti-vegf antibody constructs and related methods for treating vestibular schwannoma associated symptoms
AU2023217698A1 (en) 2022-02-08 2024-07-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
JP2025508720A (ja) 2022-02-17 2025-04-10 サナ バイオテクノロジー，インコーポレイテッド 遺伝子操作されたｃｄ４７タンパク質及びその使用関連出願の相互参照
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
WO2023164545A1 (en) 2022-02-23 2023-08-31 Massachusetts Institute Of Technology Methods for upregulating shank3 expression
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
EP4493226A1 (en) 2022-03-13 2025-01-22 RegenxBio Inc. Modified muscle-specific promoters
US20250213727A1 (en) 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
JP2025514631A (ja) 2022-04-01 2025-05-09 武田薬品工業株式会社 Ｃｎｓ症状を有する疾患の遺伝子治療
KR20250007064A (ko) 2022-04-06 2025-01-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Her2 양성 전이성 유방암 및 기타 암을 치료하기 위한 조성물 및 방법
AU2023250660A1 (en) 2022-04-06 2024-10-24 Regenxbio Inc. Pharmaceutical composition comprising a recombinant adenoassociated virus vector with an expression cassette encoding a transgene for suprachoroidal administration
TW202404651A (zh) 2022-04-06 2024-02-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如形成聚集體之調配物
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
TW202345913A (zh) 2022-04-06 2023-12-01 美商銳進科斯生物股份有限公司 用於脈絡膜上投與之調配物諸如凝膠調配物
WO2023201207A1 (en) 2022-04-11 2023-10-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
JP2025513835A (ja) 2022-04-11 2025-04-30 サントル ナショナル ドゥ ラ ルシェルシュ シアンティフィック 化学修飾型アデノ随伴ウイルス
US20250249127A1 (en) 2022-04-14 2025-08-07 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2023205610A2 (en) 2022-04-18 2023-10-26 Regenxbio Inc. Hybrid aav capsids
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
AR129215A1 (es) 2022-05-03 2024-07-31 Regenxbio Inc Anticuerpos anti-complemento y agentes del complemento vectorizados y administración de estos
TW202417633A (zh) 2022-05-03 2024-05-01 美商銳進科斯生物股份有限公司 用於眼適應症之載體化抗TNF-α抑制劑
CN118974284A (zh) 2022-05-06 2024-11-15 诺华股份有限公司 新型重组aav vp2融合多肽
EP4522757A2 (en) 2022-05-13 2025-03-19 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
KR20250039324A (ko) * 2022-05-24 2025-03-20 케이트 테라퓨틱스, 인크. Xlmtm 치료를 위한 조성물
KR20250025387A (ko) 2022-06-02 2025-02-21 보이저 테라퓨틱스, 인크. Aav 캡시드 변이체 및 이의 용도
WO2023240158A2 (en) * 2022-06-07 2023-12-14 Oregon Health & Science University Mutant adeno-associated virus (aav) capsids
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
EP4544051A2 (en) 2022-06-24 2025-04-30 Tune Therapeutics, Inc. Compositions, systems, and methods for reducing low-density lipoprotein through targeted gene repression
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
EP4547690A1 (en) 2022-06-28 2025-05-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
AU2023299307A1 (en) 2022-06-30 2025-01-09 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
AR129843A1 (es) 2022-07-06 2024-10-02 Voyager Therapeutics Inc Variantes de la cápside de aav y usos de estas
EP4558149A1 (en) 2022-07-21 2025-05-28 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
WO2024026377A1 (en) 2022-07-27 2024-02-01 Sana Biotechnology, Inc. Methods of transduction using a viral vector and inhibitors of antiviral restriction factors
AU2023320453A1 (en) 2022-08-03 2025-02-06 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
WO2024044725A2 (en) 2022-08-24 2024-02-29 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
EP4577249A1 (en) 2022-08-25 2025-07-02 Takeda Pharmaceutical Company Limited Composition for use in the treatment of fabry disease
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024064863A2 (en) 2022-09-22 2024-03-28 Biomarin Pharmaceutical Inc. Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors
KR20250108100A (ko) 2022-09-22 2025-07-15 디나코 아게 Aav 유전자 치료법 벡터를 이용한 심근병증의 치료
EP4593955A1 (en) 2022-09-30 2025-08-06 RegenxBio Inc. Treatment of ocular diseases with recombinant viral vectors encoding anti-vegf fab
WO2024077089A2 (en) * 2022-10-04 2024-04-11 Siren Biotechnology, Inc. Modified cpg dinucleotides for recombinant viral vector production
WO2024081746A2 (en) 2022-10-11 2024-04-18 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
WO2024105638A1 (en) 2022-11-18 2024-05-23 Jcr Pharmaceuticals Co., Ltd. Recombinant aav vectors and methods for treatment of hunter syndrome
TW202440939A (zh) 2022-12-17 2024-10-16 賓州大學委員會 具心臟及骨骼肌特異性靶向模體的重組aav突變載體及含有其之組成物
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
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
WO2024146935A1 (en) 2023-01-06 2024-07-11 Institut National de la Santé et de la Recherche Médicale Intravenous administration of antisense oligonucleotides for the treatment of pain
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
WO2024149844A1 (en) 2023-01-12 2024-07-18 Nantes Université Chemically-modified adeno-associated virus
WO2024163747A2 (en) 2023-02-02 2024-08-08 University Of Rochester Competitive replacement of glial cells
WO2024163979A2 (en) * 2023-02-02 2024-08-08 Codexis, Inc. Engineered aav polypeptides
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
TW202449165A (zh) 2023-02-17 2024-12-16 美商渤健麻塞諸塞州股份有限公司 用於遞送有效載荷之含ｒｇｄ之肽
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024196855A2 (en) 2023-03-17 2024-09-26 University Of Rochester Ribozyme-mediated rna assembly and expression
WO2024206226A1 (en) * 2023-03-25 2024-10-03 Duke University Compositions comprising kidney-tropic aavs and methods of use thereof
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024220389A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav2 variants and uses thereof
WO2024220463A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav5 capsid with non-canonical amino acid incorporation and uses thereof
WO2024218190A1 (en) 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Generation of adeno-associated virus capsid libraries for insect cells
WO2024218192A1 (en) 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024226761A2 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229302A1 (en) 2023-05-03 2024-11-07 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
WO2024229163A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2024229167A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2024229125A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
US20240401025A1 (en) 2023-05-03 2024-12-05 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
TW202444917A (zh) 2023-05-04 2024-11-16 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
WO2024233422A1 (en) 2023-05-05 2024-11-14 Massachusetts Institute Of Technology Shank3 gene therapy approaches
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024243236A2 (en) 2023-05-22 2024-11-28 Sana Biotechnology, Inc. Methods of delivery of islet cells and related methods
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
AR133098A1 (es) 2023-06-29 2025-08-27 The Trustees Of The Univ Of Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
WO2025017168A1 (en) 2023-07-19 2025-01-23 Genethon Novel optimized utrophin micro-genes
WO2025017169A1 (en) 2023-07-20 2025-01-23 Genethon Novel mididystrophins
WO2025035143A1 (en) 2023-08-10 2025-02-13 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of spinal muscular atrophy
WO2025042711A1 (en) 2023-08-18 2025-02-27 Eli Lilly And Company Engineered transferrin receptor binding peptides as well as methods of making and using the same
WO2025072604A1 (en) 2023-09-28 2025-04-03 University Of Rochester Rna-editing gene therapy approaches for treating myotonic dystrophy type 1 (dm1)
WO2025075963A1 (en) 2023-10-02 2025-04-10 Regenxbio Inc. Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
WO2025080780A1 (en) 2023-10-10 2025-04-17 University Of Rochester Delivery and expression of prime editing crispr systems
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025090858A1 (en) 2023-10-27 2025-05-01 Biogen Ma Inc. Methods for identifying aav capsid variants with desired characteristics
WO2025096967A1 (en) 2023-11-02 2025-05-08 Biogen Ma Inc. Redirection of aav capsids for central nervous system targeting
WO2025102034A1 (en) 2023-11-10 2025-05-15 The Trustees Of The University Of Pennsylvania Gene therapy for barth syndrome
WO2025106374A1 (en) 2023-11-13 2025-05-22 Juno Therapeutics, Inc. Aav production method
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
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025122543A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2025122536A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025129157A1 (en) 2023-12-15 2025-06-19 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of canavan disease
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025171001A1 (en) 2024-02-06 2025-08-14 Regeneron Pharmaceuticals, Inc. Compositions and methods for improved production of aav viral vectors

Family Cites Families (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4886876A (en) 1983-03-31 1989-12-12 Scripps Clinic And Research Foundation Factor VIII coagulant polypeptides
EP0162067B1 (en) 1983-10-28 1992-07-15 Genetics Institute, Inc. Production of factor viii and related products
US4757006A (en) 1983-10-28 1988-07-12 Genetics Institute, Inc. Human factor VIII:C gene and recombinant methods for production
US5045455A (en) 1984-01-12 1991-09-03 Chiron Corporation Factor VIII:C cDNA cloning and expression
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
IL74909A (en) 1984-04-20 1992-01-15 Genentech Inc Preparation of functional human factor viii and dna sequences,expression vectors,transformed microorganisms and cell lines used therein
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5149637A (en) 1987-04-06 1992-09-22 Scripps Clinic & Research Foundation Recombinant Factor VIIIC fragments
IL86693A (en) 1987-06-12 1994-06-24 Stichting Centraal Lab Proteins with factor VIII activity, process for their preparation using genetically engineered cells and pharmaceutical compositions containing them
FR2619314B1 (fr) 1987-08-11 1990-06-15 Transgene Sa Analogue du facteur viii, procede de preparation et composition pharmaceutique le contenant
US5004803A (en) 1988-11-14 1991-04-02 Genetics Institute, Inc. Production of procoagulant proteins
ES2112255T3 (es) 1989-11-17 1998-04-01 Novo Nordisk As Complejos proteicos que tienen actividad del factor viii:c y su produccion.
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
WO1994011013A1 (en) 1992-11-13 1994-05-26 Duke University Chimeric blood coagulation proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
DE69433925T2 (de) 1993-06-10 2005-07-28 Bayer Corp. Vektoren und Zelllinien von Säugetieren mit erhöhter Produktivität
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5681746A (en) 1994-12-30 1997-10-28 Chiron Viagene, Inc. Retroviral delivery of full length factor VIII
AU6486196A (en) 1995-07-11 1997-02-10 Chiron Corporation Novel factor viii:c polypeptide analogs with altered protease sites
US20020088014A1 (en) * 1996-05-31 2002-07-04 Xiangming Fang Minimal adenovirus mediated recombinant vaccine
US5866552A (en) 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
JP2001500015A (ja) 1996-09-06 2001-01-09 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 Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
ATE380037T1 (de) 1996-09-06 2007-12-15 Univ Pennsylvania Verfaheren zur durch rekombinante adeno- assoziierte virus-gerichtete gentherapie
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, Department Of Health And Human Services Aav4 vector and uses thereof
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
EP1015619A1 (en) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
ATE402254T1 (de) 1998-05-28 2008-08-15 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
EP1127150B1 (en) 1998-11-05 2007-05-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6498244B1 (en) * 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2002248297A1 (en) * 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
CN1236057C (zh) * 2001-10-18 2006-01-11 华中科技大学同济医学院附属同济医院 表达人类激肽释放酶的重组腺相关病毒及其制法和用途
NZ618298A (en) * 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
CA2469053C (en) 2001-12-17 2011-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
AU2003221733A1 (en) * 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. Improved raav vectors
EP1636360A4 (en) * 2003-06-03 2006-11-08 Cell Genesys Inc COMPOSITIONS AND METHODS FOR ENHANCED EXPRESSION OF RECOMBINANT POLYPEPTIDES FROM A SINGLE VECTOR USING A PEPTIDE CLEAVAGE SITE
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
CN117363655A (zh) * 2005-04-07 2024-01-09 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US8628966B2 (en) 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
TR201908020T4 (tr) * 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
SG10201902574RA (en) * 2014-09-24 2019-04-29 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
JP2020510430A (ja) * 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 Ａａｖベクターに基づくインフルエンザワクチン
PE20191362A1 (es) 2017-02-28 2019-10-01 Univ Pennsylvania Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este

Also Published As

Publication number Publication date
ES2648241T3 (es) 2017-12-29
PT2292780T (pt) 2017-11-28
DK3211085T3 (da) 2021-06-21
US20210299274A1 (en) 2021-09-30
CY2021024I1 (el) 2021-12-31
JP2015163083A (ja) 2015-09-10
JP6516713B2 (ja) 2019-05-22
HUS2100033I1 (hu) 2021-09-28
ES2874298T3 (es) 2021-11-04
US20210170050A1 (en) 2021-06-10
FR21C1037I1 (fr) 2021-10-15
EP2292779B1 (en) 2016-11-16
AU2008203421A1 (en) 2008-08-21
US10485883B2 (en) 2019-11-26
US11357868B2 (en) 2022-06-14
EP2292780A3 (en) 2011-03-30
EP1668143B1 (en) 2013-03-20
AU2004278684A1 (en) 2005-04-14
SI3211085T1 (sl) 2021-12-31
US9737618B2 (en) 2017-08-22
EP2298926A1 (en) 2011-03-23
CA2939103A1 (en) 2005-04-14
CN1856576B (zh) 2011-05-04
PT3211085T (pt) 2021-06-17
JP6673963B2 (ja) 2020-04-01
HUE054805T2 (hu) 2021-09-28
JP5431516B2 (ja) 2014-03-05
JP2007507223A (ja) 2007-03-29
JP5054975B2 (ja) 2012-10-24
JP2012175974A (ja) 2012-09-13
US20200155704A1 (en) 2020-05-21
CY1119833T1 (el) 2018-06-27
JP2017060483A (ja) 2017-03-30
CN1856576A (zh) 2006-11-01
JP5797397B2 (ja) 2015-10-21
US20190015527A1 (en) 2019-01-17
EP2292780B1 (en) 2017-08-23
WO2005033321A3 (en) 2005-06-30
CN102220376A (zh) 2011-10-19
NZ545628A (en) 2009-04-30
CA2939103C (en) 2020-03-24
DK2292780T3 (en) 2017-12-04
US10265417B2 (en) 2019-04-23
US7906111B2 (en) 2011-03-15
US20170028082A1 (en) 2017-02-02
AU2004278684B2 (en) 2011-05-12
US20150315612A1 (en) 2015-11-05
ES2616016T3 (es) 2017-06-09
US20190054188A1 (en) 2019-02-21
JP5911069B2 (ja) 2016-04-27
US10695441B2 (en) 2020-06-30
HUE033158T2 (en) 2017-11-28
PL3211085T3 (pl) 2021-11-15
DK2345731T3 (en) 2016-01-25
US20170159027A1 (en) 2017-06-08
JP2022173573A (ja) 2022-11-18
EP3211085B1 (en) 2021-03-31
SI2292780T1 (sl) 2017-12-29
US20190022249A1 (en) 2019-01-24
CN102174574A (zh) 2011-09-07
EP3211085A1 (en) 2017-08-30
EP1668143A2 (en) 2006-06-14
CA3072423A1 (en) 2005-04-14
EP2345731A1 (en) 2011-07-20
DK2292779T3 (en) 2017-02-27
CY2021024I2 (el) 2021-12-31
CY1124586T1 (el) 2021-12-31
US10722598B2 (en) 2020-07-28
AU2008203421B2 (en) 2011-08-25
JP2018108113A (ja) 2018-07-12
CN102199626B (zh) 2015-06-24
HUE035567T2 (en) 2018-05-28
WO2005033321A2 (en) 2005-04-14
US10973928B2 (en) 2021-04-13
EP2292780A2 (en) 2011-03-09
CN102212558B (zh) 2016-08-03
US20220025400A1 (en) 2022-01-27
US11357867B2 (en) 2022-06-14
EP2345731B1 (en) 2015-10-21
CN102199626A (zh) 2011-09-28
JP2014012022A (ja) 2014-01-23
CA2537793C (en) 2013-07-23
JP2020054373A (ja) 2020-04-09
PL2292780T3 (pl) 2018-05-30
EP3910063A1 (en) 2021-11-17
US20220023441A1 (en) 2022-01-27
ES2411479T3 (es) 2013-07-05
JP2011101646A (ja) 2011-05-26
ES2555612T3 (es) 2016-01-05
JP6335141B2 (ja) 2018-05-30
CN102212558A (zh) 2011-10-12
CN102174574B (zh) 2016-08-03
EP2292779A3 (en) 2011-03-30
US20070036760A1 (en) 2007-02-15
EP2292779A2 (en) 2011-03-09
CA2537793A1 (en) 2005-04-14
US20110236353A1 (en) 2011-09-29

Similar Documents

Publication Publication Date Title
BE2013C075I2 (enrdf_load_stackoverflow)
BE2013C070I2 (enrdf_load_stackoverflow)
BE2013C067I2 (enrdf_load_stackoverflow)
BE2013C038I2 (enrdf_load_stackoverflow)
BE2013C036I2 (enrdf_load_stackoverflow)
BE2011C030I2 (enrdf_load_stackoverflow)
JP2003264592A5 (enrdf_load_stackoverflow)
IN2006CH01274A (enrdf_load_stackoverflow)
BE2013C034I2 (enrdf_load_stackoverflow)
BE2012C053I2 (enrdf_load_stackoverflow)
JP2004109110A5 (enrdf_load_stackoverflow)
JP2004215137A5 (enrdf_load_stackoverflow)
JP2003215712A5 (enrdf_load_stackoverflow)
JP2004069694A5 (enrdf_load_stackoverflow)
JP2003340227A5 (enrdf_load_stackoverflow)
JP2004220896A5 (enrdf_load_stackoverflow)
JP2004073841A5 (enrdf_load_stackoverflow)
JP2004152252A5 (enrdf_load_stackoverflow)
JP2004078182A5 (enrdf_load_stackoverflow)
AU2003210772A1 (enrdf_load_stackoverflow)
AU2002351829A1 (enrdf_load_stackoverflow)
AU2002311957A1 (enrdf_load_stackoverflow)
AU2002245368A1 (enrdf_load_stackoverflow)
JP2004006806A5 (enrdf_load_stackoverflow)
AU2002239060A1 (enrdf_load_stackoverflow)