SI2292780T1 - Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba - Google Patents

Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba

Info

Publication number
SI2292780T1
SI2292780T1 SI200432415T SI200432415T SI2292780T1 SI 2292780 T1 SI2292780 T1 SI 2292780T1 SI 200432415 T SI200432415 T SI 200432415T SI 200432415 T SI200432415 T SI 200432415T SI 2292780 T1 SI2292780 T1 SI 2292780T1
Authority
SI
Slovenia
Prior art keywords
clades
aav
adeno
sequences
associated virus
Prior art date
Application number
SI200432415T
Other languages
English (en)
Slovenian (sl)
Inventor
Guangping Gao
James M. Wilson
Luk Vandenberghe
Mauricio R. Alvira
Original Assignee
The Trustees Of The University Of 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=SI2292780(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by The Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of SI2292780T1 publication Critical patent/SI2292780T1/sl

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    • 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

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  • 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)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (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)
SI200432415T 2003-09-30 2004-09-30 Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba SI2292780T1 (sl)

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
EP10182796.2A EP2292780B1 (en) 2003-09-30 2004-09-30 Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof

Publications (1)

Publication Number Publication Date
SI2292780T1 true SI2292780T1 (sl) 2017-12-29

Family

ID=34426044

Family Applications (2)

Application Number Title Priority Date Filing Date
SI200432415T SI2292780T1 (sl) 2003-09-30 2004-09-30 Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (aav) in njihova uporaba
SI200432511T SI3211085T1 (sl) 2003-09-30 2004-09-30 Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (AAV) in njihova uporaba

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SI200432511T SI3211085T1 (sl) 2003-09-30 2004-09-30 Genotipske skupine, zaporedja, vektorji, ki vsebujejo le-te z adenovirusi povezanega virusa (AAV) in njihova uporaba

Country Status (17)

Country Link
US (13) US7906111B2 (enExample)
EP (7) EP3211085B1 (enExample)
JP (9) JP5054975B2 (enExample)
CN (4) CN1856576B (enExample)
AU (2) AU2004278684B2 (enExample)
BE (1) BE2021C533I2 (enExample)
CA (3) CA2939103C (enExample)
CY (3) CY1119833T1 (enExample)
DK (4) DK2292780T3 (enExample)
ES (5) ES2648241T3 (enExample)
FR (1) FR21C1037I1 (enExample)
HU (4) HUE033158T2 (enExample)
NZ (1) NZ545628A (enExample)
PL (2) PL3211085T3 (enExample)
PT (2) PT2292780T (enExample)
SI (2) SI2292780T1 (enExample)
WO (1) WO2005033321A2 (enExample)

Families Citing this family (643)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL161827A0 (en) 2001-11-13 2005-11-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby
CA2469785C (en) 2001-12-17 2014-04-22 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
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
EP3211085B1 (en) * 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
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
WO2006078279A2 (en) 2004-04-28 2006-07-27 The Trustees Of The University Of Pennsylvania Sequential delivery of immunogenic molecules via adenovirus and adeno-associated virus-mediated administrations
CN104293835B (zh) 2005-04-07 2017-07-04 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
US20070274908A1 (en) 2006-04-07 2007-11-29 The Board Of Regents Of The University Of Texas System Methods and compositions related to adenoassociated virus-phage particles
WO2007127264A2 (en) 2006-04-28 2007-11-08 The Trustees Of The University Of Pennsylvania Scalable production method for aav
WO2008027084A2 (en) * 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
DK2191001T3 (en) 2007-04-09 2016-09-19 Univ Florida RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF
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
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
HUE044522T2 (hu) * 2009-04-30 2019-10-28 Univ Pennsylvania Adeno-asszociált vírus konstrukciókat tartalmazó légúti vezetõ sejtek célzására szolgáló kompozíciók
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US9133479B2 (en) * 2009-06-03 2015-09-15 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
MX342858B (es) 2010-03-29 2016-10-13 The Trustees Of The Univ Of Pennsylvania * Sistema de ablacion transgenica inducida farmacologicamente.
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
CA2833912C (en) 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders
ES2605305T3 (es) 2010-04-23 2017-03-13 University Of Massachusetts Vectores de AAV que se dirigen al SNC y métodos de uso de los mismos
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
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
WO2012057363A1 (ja) * 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
CA2826273C (en) 2011-02-10 2021-11-02 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
JP6224459B2 (ja) 2011-02-17 2017-11-01 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 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
EP2699688A1 (en) 2011-04-20 2014-02-26 The Trustees Of The University Of Pennsylvania Regimens and compositions for aav-mediated passive immunization of airborne pathogens
EP3318634A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations
CN105755044A (zh) 2011-04-22 2016-07-13 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及其使用方法
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
EP2748185A1 (en) 2011-08-24 2014-07-02 The Board of Trustees of The Leland Stanford Junior University New aav capsid proteins for nucleic acid transfer
US8859256B2 (en) 2011-10-05 2014-10-14 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
US9611305B2 (en) 2012-01-06 2017-04-04 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
EP2847337A4 (en) * 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
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
CN105120901A (zh) 2012-07-11 2015-12-02 宾夕法尼亚大学托管会 Rpgr x染色体连锁视网膜退化的aav介导的基因治疗
EP3415167B1 (en) 2012-08-01 2024-05-29 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
EP3738974A1 (en) * 2012-09-28 2020-11-18 The University of North Carolina at Chapel Hill Aav vectors targeted to oligodendrocytes
WO2014055960A1 (en) 2012-10-05 2014-04-10 Genelux Corporation 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
WO2014159247A1 (en) 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with als
CN110885854A (zh) * 2013-03-15 2020-03-17 北卡罗来纳-查佩尔山大学 双重聚糖结合aav载体的方法和组合物
PL2984166T3 (pl) 2013-03-15 2021-01-11 The Trustees Of The University Of Pennsylvania Kompozycje do leczenia mpsi
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
WO2015038958A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CN106029107A (zh) * 2013-10-07 2016-10-12 基罗米克有限责任公司 使用疾病特异性启动子治疗心血管疾病的组合物和方法
EP3459965B1 (en) 2013-10-11 2020-12-02 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
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
HUE051311T2 (hu) 2014-03-09 2021-03-01 Univ Pennsylvania Ornitin transzkarbamiláz (TC) deficiencia kezelésében alkalmas készítmények
WO2015137802A1 (en) 2014-03-10 2015-09-17 Uniqure Ip B.V. Further improved aav vectors produced in insect cells
WO2015142941A1 (en) 2014-03-17 2015-09-24 Avalanche Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
EP3750907A3 (en) 2014-03-18 2021-04-28 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
EP2933335A1 (en) 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
BR112016024379A2 (pt) 2014-04-25 2017-10-10 Univ Pennsylvania variantes ldlr e seu uso em composições para reduzir os níveis de colesterol
JP6983511B2 (ja) 2014-04-25 2021-12-17 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 脳中の転移乳癌および他の癌を処置するための方法および組成物
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
PE20170261A1 (es) 2014-05-13 2017-04-12 Univ Pennsylvania Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usos
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
US10711270B2 (en) 2014-10-03 2020-07-14 University Of Massachusetts High efficiency library-identified AAV vectors
CN108064282A (zh) 2014-10-14 2018-05-22 哈洛齐梅公司 腺苷脱氨酶-2(ada2)、其变体的组合物及使用其的方法
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
SG10202007103TA (en) 2014-11-05 2020-09-29 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
AU2015342997B2 (en) 2014-11-05 2021-11-18 Research Institute At Nationwide Children's Hospital Methods and materials for producing recombinant viruses in eukaryotic microalgae
IL284949B2 (en) 2014-11-14 2024-06-01 Voyager Therapeutics Inc Modulatory polynucleotides
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
EP4344741A3 (en) * 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
WO2016094783A1 (en) 2014-12-12 2016-06-16 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
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
JP6832280B2 (ja) 2015-01-16 2021-02-24 ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ 新規のマイクロジストロフィンおよび使用の関連する方法
WO2016126857A1 (en) 2015-02-03 2016-08-11 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
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
SG11201707116QA (en) 2015-03-10 2017-09-28 Univ Columbia Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression
EP3277819B1 (en) 2015-03-24 2021-03-03 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
EP3288594B1 (en) 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
WO2016179496A1 (en) 2015-05-07 2016-11-10 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
EP3294894B8 (en) * 2015-05-12 2019-09-25 The U.S.A. 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
EP3307310A2 (en) 2015-05-13 2018-04-18 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-influenza antibodies and methods of use thereof
US20170067028A1 (en) * 2015-05-15 2017-03-09 Douglas J. Ballon Radiolabeling of adeno associated virus
WO2016196324A1 (en) 2015-05-29 2016-12-08 University Of Floridia Research Foundation, Inc. Methods for diagnosing huntington's disease
EP3325018B1 (en) 2015-07-22 2025-01-15 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
WO2017019994A2 (en) * 2015-07-30 2017-02-02 Massachusetts Eye And Ear Infirmary Ancestral virus sequences and uses thereof
IL257105B (en) 2015-07-31 2022-09-01 Univ Minnesota Modified cells and methods of therapy
JP6978407B2 (ja) 2015-08-06 2021-12-08 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 Ｇｌｐ−１及び代謝性疾患を治療するための組成物におけるその使用
EP4345455A3 (en) 2015-08-25 2024-07-17 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
CN108137664B (zh) 2015-08-31 2021-11-26 宾夕法尼亚州大学信托人 用于治疗伴侣动物的aav-epo
EP3359663A4 (en) 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE
AU2016332821B2 (en) 2015-09-28 2022-02-17 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
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
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome 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
WO2017070476A2 (en) * 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
WO2017070516A1 (en) * 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017070491A1 (en) * 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
CA3003435A1 (en) 2015-10-28 2017-05-04 The Trustees Of The University Of Pennsylvania Intrathecal administration of adeno-associated-viral vectors for gene therapy
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
JP6767483B2 (ja) 2015-11-05 2020-10-14 バンブー セラペウティクス インク 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター
WO2017096162A1 (en) 2015-12-02 2017-06-08 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
EP3387117B1 (en) 2015-12-11 2022-11-23 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
SG11201804814YA (en) 2015-12-11 2018-07-30 Massachusetts Eye & Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2017100704A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
EP3400304B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
EP3795180B1 (en) 2015-12-14 2025-11-05 The Trustees of The University of Pennsylvania Gene therapy for ocular disorders
US20180353624A1 (en) * 2015-12-14 2018-12-13 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
WO2017106345A1 (en) 2015-12-14 2017-06-22 The Trustees Of The University Of Pennsylvania Composition for treatment of crigler-najjar syndrome
WO2017136536A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
SG11201806270XA (en) 2016-02-03 2018-08-30 Univ Pennsylvania Gene therapy for treating mucopolysaccharidosis type 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
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
US10940161B2 (en) 2016-04-04 2021-03-09 University Of Florida Research Foundation, Incorporated Manipulation of EIF3 to modulate repeat associated non-ATG (RAN) translation
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN
US11446398B2 (en) 2016-04-11 2022-09-20 Obsidian Therapeutics, Inc. Regulated biocircuit systems
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
SG11201808812RA (en) * 2016-04-15 2018-11-29 Univ Pennsylvania Novel aav8 mutant capsids and compositions containing same
CA3019425A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Gene therapy for treating hemophilia a
KR20190008237A (ko) 2016-04-15 2019-01-23 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
KR20250095754A (ko) 2016-04-15 2025-06-26 알파인 이뮨 사이언시즈, 인코포레이티드 Icos 리간드 변이체 면역조절 단백질 및 그의 용도
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
JP7046828B2 (ja) 2016-04-15 2022-04-04 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 滲出型加齢性黄斑変性の治療のための組成物
EP3443000B1 (en) 2016-04-15 2025-11-12 Alpine Immune Sciences, Inc. Cd80 variant immunomodulatory proteins and uses thereof
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
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
AU2017261249B2 (en) * 2016-05-03 2021-05-06 Children's Medical Research Institute Adeno-associated virus polynucleotides, polypeptides and virions
RU2725286C2 (ru) 2016-05-13 2020-06-30 4Д Молекьюлар Терапьютикс Инк. Варианты капсидов аденоассоциированного вируса и способы их применения
EP3458588B1 (en) 2016-05-18 2025-10-29 Voyager Therapeutics, Inc. Modulatory polynucleotides
AU2017268382B2 (en) 2016-05-18 2023-09-28 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
CA3024536A1 (en) 2016-06-13 2017-12-21 The University Of North Carolina At Chapel Hill Optimized cln1 genes and expression cassettes and their use
US11882815B2 (en) 2016-06-15 2024-01-30 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
KR102526506B1 (ko) 2016-07-08 2023-05-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
US11883470B2 (en) 2016-07-25 2024-01-30 The Trustees Of The University Of Pennsylvania Compositions comprising a lecithin cholesterol acyltransferase variant and uses thereof
IL305149A (en) * 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
WO2018022946A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd155 variant immunomodulatory proteins and uses thereof
WO2018022945A1 (en) 2016-07-28 2018-02-01 Alpine Immune Sciences, Inc. Cd112 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
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
WO2018035503A1 (en) 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
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
KR20190075964A (ko) * 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
CN110520530A (zh) 2016-10-18 2019-11-29 明尼苏达大学董事会 肿瘤浸润性淋巴细胞和治疗方法
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
CN110352245A (zh) 2016-10-20 2019-10-18 高山免疫科学股份有限公司 可分泌变体免疫调节蛋白和工程化细胞疗法
EP3535396A1 (en) 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
JP7065852B2 (ja) 2016-12-01 2022-05-12 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 網膜変性疾患の処置のための医薬組成物
US11473106B2 (en) 2016-12-30 2022-10-18 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
WO2018144709A2 (en) 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
CN110546265A (zh) 2017-02-09 2019-12-06 因达普塔治疗公司 工程化自然杀伤(nk)细胞及其组合物和方法
US11554147B2 (en) 2017-02-20 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3054131C (en) 2017-02-21 2021-05-25 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
WO2018154096A1 (en) 2017-02-24 2018-08-30 Georg-August-Universität Göttingen Stiftung Öffentlichen Rechts, Universitätsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated rasal,1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including rasal1 in a subject
EP3589730B1 (en) 2017-02-28 2023-12-27 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CN110573523A (zh) 2017-02-28 2019-12-13 宾夕法尼亚州大学信托人 基于aav载体的流感疫苗
US11564996B2 (en) 2017-03-01 2023-01-31 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
EP3589311A1 (en) 2017-03-02 2020-01-08 Genethon Method for removing anti-aav antibodies from a blood-derived composition
AU2018236224B2 (en) 2017-03-16 2024-01-04 Alpine Immune Sciences, Inc. PD-L1 variant immunomodulatory proteins and uses thereof
JP7386083B2 (ja) 2017-03-16 2023-11-24 アルパイン イミューン サイエンシズ インコーポレイテッド Ｃｄ８０バリアント免疫調節タンパク質及びその使用
AU2018235835A1 (en) 2017-03-16 2019-09-05 Alpine Immune Sciences, Inc. PD-L2 variant immunomodulatory proteins and uses thereof
US11946074B2 (en) 2017-03-24 2024-04-02 Lankenau Institute For Medical Research Methods and compositions for inducible extracellular membrane capture of monoclonal immunoglobulins secreted by hybridomas
EP3613856A4 (en) 2017-03-31 2020-12-23 Staidson(Beijing) Biopharmaceuticals Co., Ltd. SHORT RNA EXPRESSION CASSETTE IN HAIRPIN, POLYNUCLEOTIDIAL SEQUENCE WEARING IT AND CORRESPONDING APPLICATION
EP3610016A1 (en) 2017-04-10 2020-02-19 Genethon Antisense targeting dynamin 2 and use for the treatment of centronuclear myopathies and neuropathies
SG11201909361SA (en) 2017-04-14 2019-11-28 Regenxbio Inc Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2 sulfatase (ids) produced by human neural or glial cells
JP7526455B2 (ja) * 2017-04-17 2024-08-01 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． ＰＫＲおよびｅＩＦ２Ａ－Ｐ経路によるＲＡＮタンパク質翻訳の調節
US11879133B2 (en) 2017-04-24 2024-01-23 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
CN111108198A (zh) 2017-05-05 2020-05-05 沃雅戈治疗公司 治疗亨廷顿病的组合物和方法
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CA3059213A1 (en) 2017-05-09 2018-11-15 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
JP7330899B2 (ja) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー ウイルスのアセンブリ活性化タンパク質（ａａｐ）依存性を改変する方法および組成物
IL270422B2 (en) 2017-05-11 2025-04-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis
US11793887B2 (en) 2017-05-31 2023-10-24 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 DISEASES
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
KR102766238B1 (ko) 2017-06-30 2025-02-13 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
JP2020530307A (ja) * 2017-06-30 2020-10-22 インティマ・バイオサイエンス，インコーポレーテッド 遺伝子治療のためのアデノ随伴ウイルスベクター
KR20200053468A (ko) 2017-07-06 2020-05-18 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 I형 점액다당류증을 치료하기 위한 유전자 요법
PL3648783T3 (pl) 2017-07-07 2024-11-18 Genethon Nowe polinukleotydy kodujące ludzkie białko fkrp
AU2018302016A1 (en) 2017-07-17 2020-02-06 The Regents Of The University Of California Trajectory array guide system
EP3658577A1 (en) 2017-07-27 2020-06-03 Novartis AG Sheddase resistant trem2 variants
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
BR112020003571A2 (pt) 2017-08-28 2020-08-25 The Regents Of The University Of California variantes capsidiais de vírus adenoassociado e métodos de uso das mesmas
MX2020003176A (es) 2017-09-20 2020-08-20 4D Molecular Therapeutics Inc Variantes de capsides de virus adenoasociados y metodos de uso de estas.
AU2018338188B2 (en) 2017-09-22 2025-02-20 University Of Massachusetts SOD1 dual expression vectors and uses thereof
WO2019060662A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II
CA3075908A1 (en) 2017-09-25 2019-03-28 University Of Florida Research Foundation, Incorporated Immunoassays for detection of ran proteins
JP7350337B2 (ja) 2017-09-26 2023-09-26 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 神経障害の処置においてｒａｎタンパク質レベルを低下させるためのメトホルミンおよびそのアナログの使用
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
CN111479924B (zh) 2017-10-16 2024-06-14 沃雅戈治疗公司 肌萎缩性侧索硬化症(als)的治疗
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
SG11202003479TA (en) 2017-10-18 2020-05-28 Regenxbio Inc Fully-human post-translationally modified antibody therapeutics
TW202500579A (zh) 2017-10-18 2025-01-01 美商艾爾潘免疫科學有限公司 變異型icos 配位體免疫調節蛋白及相關組合物及方法
AU2018350990A1 (en) 2017-10-18 2020-05-21 Regenxbio Inc. Treatment of ocular diseases and metastatic colon cancer with human post-translationally modified VEGF-Trap
EP3697448A1 (en) 2017-10-20 2020-08-26 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
CA3083472A1 (en) 2017-11-27 2019-05-31 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and use for inhibiting angiogenesis
JP7384797B2 (ja) 2017-11-30 2023-11-21 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 ムコ多糖症ｉｉｉｂ型のための遺伝子療法
US11555206B2 (en) 2017-11-30 2023-01-17 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis IIIA
EA202091509A1 (ru) 2017-12-19 2020-09-22 Акуос, Инк. Aav-опосредованная доставка терапевтических антител во внутреннее ухо
SG11202006148UA (en) 2018-01-03 2020-07-29 Alpine Immune Sciences Inc Multi-domain immunomodulatory proteins and methods of use thereof
WO2019143803A1 (en) 2018-01-17 2019-07-25 Adrenas Therapeutics, Inc. Adeno-associated virus gene therapy for 21-hydroxylase deficiency
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
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
CN112041437A (zh) 2018-02-19 2020-12-04 同源药物公司 用于恢复f8基因功能的腺相关病毒组合物和其使用方法
IL276859B2 (en) * 2018-02-27 2025-12-01 Univ Pennsylvania Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
CN120519403A (zh) * 2018-02-27 2025-08-22 宾夕法尼亚州大学信托人 新颖腺相关病毒(aav)载体、具有降低的衣壳脱酰胺化的aav载体及其用途
CN111819286B (zh) * 2018-03-16 2024-05-24 国家儿童医院研究所 通过衣壳修饰增加组织特异性的基因递送
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
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
EP3775173A1 (en) 2018-04-05 2021-02-17 Genethon Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
WO2019195701A1 (en) 2018-04-05 2019-10-10 Massachusetts Eye And Ear Infirmary Methods of making and using combinatorial barcoded nucleic acid libraries having defined variation
WO2019204303A2 (en) 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
AU2019255745A1 (en) 2018-04-18 2020-11-19 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
US12378576B2 (en) 2018-04-27 2025-08-05 Spacecraft Seven, Llc Gene therapy for CNS degeneration
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
US12365865B2 (en) 2018-04-29 2025-07-22 Regenxbio Inc. Scalable clarification process for recombinant AAV production
AU2019265663B2 (en) 2018-05-08 2024-06-13 Rutgers, The State University Of New Jersey AAV-compatible laminin-linker polymerization proteins
MX2020011937A (es) 2018-05-09 2021-01-29 Biomarin Pharm Inc Metodos de tratamiento de fenilcetonuria.
WO2019217911A1 (en) 2018-05-11 2019-11-14 Massachusetts Eye And Ear Infirmary Liver-specific tropism of adeno-associated viruses
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
WO2019221992A1 (en) 2018-05-15 2019-11-21 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
TW202015742A (zh) 2018-05-15 2020-05-01 美商航海家醫療公司 投遞腺相關病毒（ａａｖ）之組成物和方法
MA52631A (fr) 2018-05-15 2021-03-24 Voyager Therapeutics Inc Compositions et méthodes pour le traitement de la maladie de parkinson
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
US20220403001A1 (en) 2018-06-12 2022-12-22 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
IL279193B2 (en) 2018-06-14 2025-07-01 Regenxbio Inc Anion exchange chromatography for recombinant AAV production
WO2019241758A1 (en) 2018-06-15 2019-12-19 Alpine Immune Sciences, Inc. Pd-1 variant immunomodulatory proteins and uses thereof
GB201811368D0 (en) 2018-07-11 2018-08-29 Ucb Biopharma Sprl Antibody
WO2020014523A1 (en) 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
AU2019304569B2 (en) 2018-07-17 2023-07-06 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
JP2021530548A (ja) 2018-07-24 2021-11-11 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. 遺伝子治療製剤を生産するための系および方法
WO2020028751A2 (en) 2018-08-03 2020-02-06 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2020033842A1 (en) 2018-08-10 2020-02-13 Regenxbio Inc. Scalable method for recombinant aav production
CA3109804A1 (en) 2018-08-16 2020-02-20 The Regents Of The University Of California Chemically and photochemically initiated cell membrane blebbing to induce cell vesicle production, modifications thereof, and uses thereof
WO2020047161A2 (en) 2018-08-28 2020-03-05 Actym Therapeutics, Inc. Engineered immunostimulatory bacterial strains and uses thereof
JP7450945B2 (ja) 2018-08-30 2024-03-18 テナヤ セラピューティクス， インコーポレイテッド ミオカルディンおよびａｓｃｌ１を用いた心細胞リプログラミング
AU2019346447A1 (en) 2018-09-26 2021-04-29 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
JP2022513318A (ja) 2018-10-01 2022-02-07 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
US20230040603A1 (en) 2018-10-01 2023-02-09 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
CA3114869C (en) 2018-10-01 2023-10-03 Toru Miyazaki Therapeutic agent for neurodegenerative disease
EP3861010A1 (en) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
EP3861113A1 (en) 2018-10-04 2021-08-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
CA3115248A1 (en) * 2018-10-05 2020-04-09 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding aav production proteins
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
TW202028468A (zh) 2018-10-15 2020-08-01 美商航海家醫療公司 用於桿狀病毒/Sf9系統中rAAV之大規模生產的表現載體
CN112912518A (zh) 2018-10-15 2021-06-04 再生生物股份有限公司 用于测量复制缺陷型病毒载体和病毒的感染性的方法
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
BR112021007503A2 (pt) 2018-10-22 2021-11-03 Univ Rochester Proteína de fusão, molécula de ácido nucleico, método para edição de material genético, e, sistemas para edição de material genético e para distribuição de componentes de edição de genoma
WO2020086742A1 (en) 2018-10-24 2020-04-30 Obsidian Therapeutics, Inc. Er tunable protein regulation
CA3116192C (en) 2018-11-06 2025-09-23 Calidi Biotherapeutics (Nevada), Inc. IMPROVED SYSTEMS FOR CELL-MEDIATED ONCOLYTIC VIRAL THERAPY
MX2021005720A (es) 2018-11-16 2021-07-21 Astellas Pharma Inc Metodo para tratar la distrofia muscular mediante el direccionamiento del gen de utrofina.
EP3884041A2 (en) 2018-11-21 2021-09-29 Indapta Therapeutics, Inc. Methods for expansion of natural killer (nk) cell subset and related compositions and methods
MX2021006359A (es) * 2018-11-30 2021-08-11 Novartis Ag Vectores virales de vaa y sus usos.
JP7713886B2 (ja) 2018-11-30 2025-07-28 アルパイン イミューン サイエンシズ インコーポレイテッド Ｃｄ８６バリアント免疫調節タンパク質およびその使用
BR112021009913A2 (pt) 2018-12-05 2021-08-17 Abeona Therapeutics, Inc. vetor viral adeno-associado recombinante para inserção gênica
US20220089670A1 (en) 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
BR112021013140A2 (pt) 2019-01-04 2021-11-30 Ultragenyx Pharmaceutical Inc Construções de terapia gênica para tratar doença de wilson
AU2020208346B2 (en) 2019-01-14 2026-01-29 University Of Rochester Targeted nuclear RNA cleavage and polyadenylation with CRISPR-cas
SG11202107645RA (en) 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
EP3917566A4 (en) 2019-01-31 2022-10-26 Oregon Health & Science University METHODS OF USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS
US12416017B2 (en) 2019-02-22 2025-09-16 The Trustees Of The University Of Pennsylvania Recombinant adeno-associated virus for treatment of GRN-associated adult-onset neurodegeneration
JP2022521025A (ja) 2019-02-25 2022-04-04 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
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
KR20210132095A (ko) 2019-02-26 2021-11-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 크라베병의 치료에 유용한 조성물
KR20220004959A (ko) 2019-02-27 2022-01-12 액팀 테라퓨틱스, 인코퍼레이티드 종양, 종양-상주 면역 세포, 및 종양 미세환경을 콜로니화하기 위해 조작된 면역자극성 박테리아
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
AU2020232692A1 (en) 2019-03-04 2021-09-30 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the AKT pathway
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy Recombinant adeno-associated virus vectors
JP7635139B2 (ja) 2019-03-25 2025-02-25 ジェネトン オーバーラップａａｖベクターを使用する大きいサイズのクアシジストロフィンの産生
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
EP3946467A1 (en) 2019-04-03 2022-02-09 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
EP4310495A3 (en) 2019-04-11 2024-04-24 REGENXBIO Inc. Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions
WO2020214929A1 (en) 2019-04-19 2020-10-22 Regenxbio Inc. Adeno-associated virus vector formulations and methods
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
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
PH12021552608A1 (en) 2019-04-29 2022-06-20 Univ Pennsylvania Novel aav capsids and compositions containing same
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
SG11202111867XA (en) 2019-05-03 2021-11-29 Univ Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
WO2020232044A1 (en) 2019-05-14 2020-11-19 Biomarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
SG11202111427RA (en) 2019-05-28 2021-11-29 Astellas Pharma Inc Method for treating muscular dystrophy by targeting dmpk gene
JP2022534119A (ja) 2019-05-30 2022-07-27 ソリッド・バイオサイエンシーズ・インコーポレーテッド 組換えヘルペスウイルスベクター
CA3144864A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
WO2021003442A1 (en) 2019-07-02 2021-01-07 M6P Therapeutics Vector compositions and methods of using same for treatment of lysosomal storage disorders
WO2021005223A1 (en) 2019-07-10 2021-01-14 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
WO2021005210A1 (en) 2019-07-11 2021-01-14 Centre National De La Recherche Scientifique Chemically-modified adeno-associated virus
EP3997226A1 (en) 2019-07-11 2022-05-18 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
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
US10653731B1 (en) 2019-07-15 2020-05-19 Vigene Biosciences Inc. Recombinantly-modified adeno-associated virus (rAAV) having improved packaging efficiency
EP4004206A1 (en) 2019-07-23 2022-06-01 University of Rochester Targeted rna cleavage with crispr-cas
JP2022544004A (ja) 2019-07-26 2022-10-17 リジェネックスバイオ インコーポレイテッド 操作された核酸調節エレメントならびにその使用方法
WO2021021674A1 (en) 2019-07-26 2021-02-04 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
AU2020334462A1 (en) 2019-08-16 2022-02-24 Modalis Therapeutics Corporation Method for treating muscular dystrophy by targeting LAMA1 gene
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
WO2021041373A1 (en) 2019-08-26 2021-03-04 Regenxbio Inc. Treatment of diabetic retinopathy with fully-human post-translationally modified anti-vegf fab
US20230092895A1 (en) 2019-08-30 2023-03-23 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
US20220340643A1 (en) 2019-09-13 2022-10-27 Rutgers, The State University Of New Jersey Aav-compatible laminin-linker polymerization proteins
WO2021053559A1 (en) 2019-09-18 2021-03-25 Novartis Ag Entpd2 antibodies, combination therapies, and methods of using the antibodies and combination therapies
TW202124446A (zh) 2019-09-18 2021-07-01 瑞士商諾華公司 與ｅｎｔｐｄ２抗體之組合療法
CN114521143B (zh) 2019-09-19 2025-03-14 吉尼松公司 减轻fkrp心脏毒性的基因治疗表达系统
JP7620988B2 (ja) 2019-09-20 2025-01-24 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 血清および組織ライセートからのｒａｎタンパク質に対する抗体の検出
EP4038194A1 (en) 2019-10-04 2022-08-10 Ultragenyx Pharmaceutical Inc. Methods for improved therapeutic use of recombinant aav
BR112022006718A2 (pt) 2019-10-07 2022-07-12 Regenxbio Inc Composição farmacêutica e métodos de vetor de vírus adeno-associado
CN115003820A (zh) 2019-10-08 2022-09-02 爱科霍拉有限公司 用于眼部治疗的组合物和方法
WO2021072129A2 (en) 2019-10-08 2021-04-15 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
US20240108754A1 (en) 2019-10-14 2024-04-04 Duke University Compositions comprising novel prokaryotic sodium channels and associated methods
AU2020367532A1 (en) * 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
EP4055034A4 (en) * 2019-11-08 2024-05-29 President And Fellows Of Harvard College Viral capsid polypeptides
EP4055030A4 (en) * 2019-11-08 2024-05-22 President And Fellows Of Harvard College VIRAL CAPSID POLYPEPTIDES
WO2021099394A1 (en) 2019-11-19 2021-05-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides and their use for the treatment of cancer
JP2023503637A (ja) * 2019-11-28 2023-01-31 リジェネックスバイオ インコーポレイテッド マイクロジストロフィン遺伝子治療コンストラクト及びその使用
IL293397A (en) * 2019-12-04 2022-07-01 Sangamo Therapeutics Inc New preparations and methods for the production of recombinant aav
WO2021116138A1 (en) 2019-12-09 2021-06-17 Ucl Business Ltd Gene therapy composition and treatment for myh7-linked cardiomyopathy
IL293684A (en) 2019-12-10 2022-08-01 Takeda Pharmaceuticals Co Adeno associated virus vectors for the treatment of hunter disease
WO2021138559A1 (en) 2019-12-31 2021-07-08 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
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
JP2023511382A (ja) 2020-01-22 2023-03-17 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα－Ｌ－イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
BR112022015036A2 (pt) 2020-01-29 2022-10-11 Regenxbio Inc Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
KR20220148162A (ko) 2020-01-29 2022-11-04 리젠엑스바이오 인크. 인간 신경 또는 신경아교 세포에 의해 생성된 재조합 인간 이두로네이트-2-설파타아제 (ids)를 사용한 점액다당류증 ii의 치료
US20230190966A1 (en) 2020-02-02 2023-06-22 The Trustees Of The University Of Pennsylvania Compositions useful for treating gm1 gangliosidosis
WO2021158964A1 (en) 2020-02-07 2021-08-12 University Of Rochester Ribozyme-mediated rna assembly and expression
JP2023513211A (ja) 2020-02-07 2023-03-30 ユニバーシティ オブ ロチェスター タンパク質合成を増強するためのＣＲＩＳＰＲ－Ｃａｓ１３による標的ＲＮＡ翻訳
BR112022015921A2 (pt) 2020-02-14 2022-10-04 Ultragenyx Pharmaceutical Inc Terapia gênica para tratar o transtorno de deficiência de cdkl5
US20230136699A1 (en) 2020-02-28 2023-05-04 INSERM (Institut National de la Santé et de la Recherche Médicale) Gene therapy for maple syrup urine disease
BR112022017551A2 (pt) 2020-03-02 2022-11-16 Tenaya Therapeutics Inc Controle de vetor genético por micrornas expressos por cardiomiócito
JP2023517011A (ja) 2020-03-05 2023-04-21 ネオティーエックス セラピューティクス リミテッド 免疫細胞を用いて癌を治療するための方法および組成物
KR20220153604A (ko) 2020-03-11 2022-11-18 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 가공된 바이러스 입자를 사용하는 유전자 전달을 위한 방법 및 조성물
JP2023518415A (ja) * 2020-03-19 2023-05-01 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 組換えａａｖにおいてｃａｐおよびｒｅｐ配列の逆パッケージングを低減するための組成物および方法
WO2021195519A1 (en) 2020-03-27 2021-09-30 University Of Rochester Targeted destruction of viral rna by crispr-cas13
EP4127170A1 (en) 2020-03-27 2023-02-08 University of Rochester Crispr-cas13 crrna arrays
JP2023519576A (ja) 2020-03-27 2023-05-11 ユーシービー バイオファルマ エスアールエル 自律ノブドメインペプチド
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
US20230226223A1 (en) 2020-04-10 2023-07-20 Sola Biosciences Llc Compositions and Methods for the Treatment of Protein Aggregation Disorders
AU2021257848A1 (en) 2020-04-15 2022-12-01 Voyager Therapeutics, Inc. Tau binding compounds
AU2021259422A1 (en) * 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US20230190801A1 (en) 2020-04-22 2023-06-22 Indapta Therapeutics, Inc. Natural killer (nk) cell compositions and methods for generating same
WO2021222168A2 (en) 2020-04-28 2021-11-04 Sola Biosciences Llc Compositions and methods for the treatment of tdp-43 proteinopathies
AR122013A1 (es) 2020-05-05 2022-08-03 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso
AU2021271635A1 (en) 2020-05-12 2023-01-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
TW202208406A (zh) 2020-05-13 2022-03-01 美商阿科奧斯公司 用於治療ｋｃｎｑ４相關性聽力損失之組成物及方法
US20230203102A1 (en) 2020-05-13 2023-06-29 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
US20230201372A1 (en) 2020-05-13 2023-06-29 Akouos, Inc. Compositions and methods for treating slc26a4-associated hearing loss
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
JP2023529371A (ja) 2020-06-05 2023-07-10 ソラ・バイオサイエンシズ・エルエルシー シヌクレイノパチーの処置のための組成物および方法
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER 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
JP2023532704A (ja) * 2020-07-03 2023-07-31 ジェネトン 超可変領域スワッピングを通じて新規のハイブリッドａａｖカプシドを操作する方法
IL299638A (en) * 2020-07-07 2023-03-01 Univ Texas Adeno-linked virus vector for DWORF polypeptide
EP4179091A1 (en) 2020-07-10 2023-05-17 Institut National De La Sante Et De La Recherche Medicale - Inserm Methods and compositions for treating epilepsy
WO2022008711A1 (en) 2020-07-10 2022-01-13 Genethon A novel muscle-specific promoter
AU2021309645A1 (en) 2020-07-13 2023-02-09 The Trustees Of The University Of Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease
WO2022015916A1 (en) 2020-07-15 2022-01-20 University Of Rochester Targeted rna cleavage with dcasl3-rnase fusion proteins
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
MX2023000815A (es) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de trastornos neurologicos relacionados con la deficiencia de glucosilceramidasa beta.
CN116121275B (zh) * 2020-07-29 2024-11-29 北京三诺佳邑生物技术有限责任公司 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
EP4196494A1 (en) 2020-08-17 2023-06-21 Massachusetts Institute of Technology Shank3 gene therapy approaches
KR20230068444A (ko) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 레트 증후군의 치료를 위한 아데노 관련 바이러스 벡터
CA3190399A1 (en) 2020-08-24 2022-03-03 James M. Wilson Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
JP2023540054A (ja) 2020-08-26 2023-09-21 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 Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス
JPWO2022050413A1 (enExample) 2020-09-04 2022-03-10
US20250326861A1 (en) 2020-09-15 2025-10-23 Regenxbio Inc. Vectorized lanadelumab and administration thereof
EP4214242A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized antibodies for anti-viral therapy
WO2022061002A1 (en) 2020-09-18 2022-03-24 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
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
AU2021358964A1 (en) 2020-10-07 2023-05-25 Regenxbio Inc. Formulations for suprachoroidal administration such as formulations with aggregate formation
IL301647A (en) 2020-10-07 2023-05-01 Regenxbio Inc Adeno-associated viruses for ocular delivery of gene therapy
TW202228646A (zh) 2020-10-07 2022-08-01 美商銳進科斯生物股份有限公司 諸如凝膠調配物之用於脈絡膜上投與之調配物
AU2021358048A1 (en) 2020-10-07 2023-05-25 Regenxbio Inc. Formulations for suprachoroidal administration such as high viscosity formulations
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
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
TW202229560A (zh) 2020-10-09 2022-08-01 賓州大學委員會 治療法布瑞氏症之組成物及方法
CA3195553A1 (en) 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
JP2023547832A (ja) 2020-10-28 2023-11-14 リジェネックスバイオ インコーポレイテッド 眼の適応症に対するベクター化抗ＴＮＦ－α抗体
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
WO2022094255A2 (en) 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
CN117042787A (zh) * 2020-10-29 2023-11-10 宾夕法尼亚州大学信托人 Aav衣壳和含有aav衣壳的组合物
AU2021369833A1 (en) 2020-10-29 2023-06-08 Regenxbio Inc. Vectorized tnf-alpha antagonists for ocular indications
CA3196778A1 (en) 2020-11-02 2022-05-05 Biomarin Pharmaceutical, Inc. Process for enriching adeno-associated virus
US20230399656A1 (en) 2020-11-03 2023-12-14 Pfizer Inc. Methods for Purification of AAV Vectors by Anion Exchange Chromatography
AU2021390480A1 (en) 2020-12-01 2023-06-22 The Trustees Of The University Of Pennsylvania Novel compositions with tissue-specific targeting motifs and compositions containing same
EP4255500A1 (en) 2020-12-01 2023-10-11 The Trustees of The University of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
CR20230228A (es) 2020-12-01 2023-07-18 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
CN116761892A (zh) 2020-12-16 2023-09-15 再生生物股份有限公司 生产重组腺相关病毒颗粒的方法
IL303959A (en) 2020-12-23 2023-08-01 Pfizer Methods for purification of aav vectors by affinity chromatography
CA3203373A1 (en) 2020-12-29 2022-07-07 Emmanuel John Simons Compositions and methods for treating clrn1-associated hearing loss and/or vision loss
EP4271479A1 (en) 2020-12-29 2023-11-08 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
EP4277933A4 (en) 2021-01-14 2024-12-11 Senti Biosciences, Inc. SECRETABLE PAYLOAD REGULATION
EP4281568A1 (en) 2021-01-21 2023-11-29 RegenxBio Inc. Improved production of recombinant polypeptides and viruses
CA3205877A1 (en) * 2021-01-25 2022-07-28 Annahita KERAVALA Adeno-associated virus capsids and engineered ligand-gated ion channels for treating focal epilepsy and neuropathic pain
US20240091380A1 (en) 2021-02-01 2024-03-21 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
US11926842B2 (en) * 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
CA3206272A1 (en) 2021-02-05 2022-08-11 Carleton Proctor Goold Vectors comprising stuffer polynucleotide sequences
AU2022218706A1 (en) * 2021-02-09 2023-09-28 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
AU2022221284A1 (en) 2021-02-10 2023-08-17 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
MX2023009978A (es) 2021-02-26 2023-09-06 Takeda Pharmaceuticals Co Composicion y metodos para el tratamiento de la enfermedad de fabry.
EP4301768A2 (en) 2021-03-03 2024-01-10 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
BR112023018430A2 (pt) 2021-03-19 2023-11-28 Adrenas Therapeutics Inc Terapias gênicas para deficiência de 21-hidroxilase
US20240307553A1 (en) 2021-04-01 2024-09-19 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
US20240191251A1 (en) 2021-04-05 2024-06-13 Solid Biosciences Inc. Recombinant herpesvirales vector
AU2022258312A1 (en) 2021-04-12 2023-10-26 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
EP4326753A2 (en) 2021-04-23 2024-02-28 University of Rochester Genome editing by directed non-homologous dna insertion using a retroviral integrase-cas fusion protein and methods of treatment
TW202305124A (zh) 2021-04-23 2023-02-01 賓州大學委員會 具有腦特異性靶向模體的新穎構成物及含有其之組成物
IL307902A (en) 2021-04-26 2023-12-01 Regenxbio Inc Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2022229807A1 (en) 2021-04-26 2022-11-03 Pfizer Inc. Adeno-associated viral vector capsids with improved tissue tropism
US20240218397A1 (en) 2021-05-04 2024-07-04 Regenxbio Inc. Novel aav vectors and methods and uses thereof
WO2022241030A1 (en) 2021-05-11 2022-11-17 Regenxbio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
CN117813314A (zh) * 2021-06-08 2024-04-02 Nf2治疗公司 用于治疗神经纤维瘤疾患的组合物和方法
WO2022266452A1 (en) * 2021-06-18 2022-12-22 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
EP4359547A1 (en) 2021-06-22 2024-05-01 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
AU2022301302A1 (en) 2021-07-01 2024-01-25 Indapta Therapeutics, Inc. Engineered natural killer (nk) cells and related methods
KR20240032971A (ko) * 2021-07-08 2024-03-12 테나야 테라퓨틱스, 인코포레이티드 유전자 요법을 위한 최적화된 발현 카세트
WO2023004331A1 (en) 2021-07-19 2023-01-26 New York University Auf1 combination therapies for treatment of muscle degenerative disease
US20240366788A1 (en) * 2021-07-23 2024-11-07 Duke University Adeno-associated virus compositions and methods of use thereof
AU2022319373A1 (en) 2021-07-30 2024-03-14 Toru Miyazaki Therapeutic agent for ischemic disease
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
CA3227108A1 (en) 2021-08-11 2023-02-16 Xiaomeng HU Genetically modified primary cells for allogeneic cell therapy
EP4384544A1 (en) 2021-08-11 2024-06-19 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
AR126870A1 (es) * 2021-08-25 2023-11-22 Canbridge Pharmaceuticals Inc PARTÍCULAS DE AAV QUE COMPRENDEN UNA PROTEÍNA DE LA CÁPSIDE HEPATOTRÓPICA Y a-GALACTOSIDASA Y SU USO PARA TRATAR LA ENFERMEDAD DE FABRY
WO2023028567A2 (en) * 2021-08-25 2023-03-02 Canbridge Pharmaceuticals, Inc. Aav particles comprising a liver-tropic capsid protein and acid alpha-glucosidase (gaa) and their use to treat pompe disease
JP2024535706A (ja) 2021-08-31 2024-10-02 スカウト バイオ， インコーポレイテッド 抗原結合分子及びその使用
WO2023044483A2 (en) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
JP2024537775A (ja) 2021-09-30 2024-10-16 アコーオス インコーポレイテッド Ｋｃｎｑ４関連難聴を治療するための組成物及び方法
WO2023056399A1 (en) 2021-10-02 2023-04-06 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
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
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
EP4413019A2 (en) 2021-10-07 2024-08-14 RegenxBio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250001006A1 (en) 2021-10-07 2025-01-02 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
US20230151372A1 (en) * 2021-10-07 2023-05-18 Academia Sinica Composition and Method of Treatment for Heart Protection and Regeneration
JP2024537178A (ja) 2021-10-08 2024-10-10 ソラ・バイオサイエンシズ・エルエルシー ｐ５３媒介性癌の処置のための組成物および方法
CA3234664A1 (en) 2021-10-08 2023-04-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
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
US20230241118A1 (en) 2021-10-20 2023-08-03 University Of Rochester Rejuvenation treatment of age-related white matter loss
US20250025574A1 (en) 2021-10-28 2025-01-23 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
MX2024004936A (es) 2021-11-02 2024-05-08 Voyager Therapeutics Inc Variantes de capsides de aav y sus usos.
CA3236365A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
TW202334181A (zh) 2021-11-17 2023-09-01 美商航海家醫療公司 Ａａｖ蛋白殼變異體及其用途
US20250034559A1 (en) 2021-11-17 2025-01-30 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
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
US20240408157A1 (en) 2021-12-02 2024-12-12 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
WO2023114897A2 (en) * 2021-12-15 2023-06-22 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
TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
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
CA3248878A1 (en) 2022-01-17 2023-07-20 Dinaqor Ag GENE THERAPY COMPOSITION AND TREATMENT OF DYSTROPHIN-RELATED CARDIOMYOPATHY
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
US20250346920A1 (en) 2022-01-25 2025-11-13 Voyager Therapeutics, Inc. Baculovirus expression system
US20250144243A1 (en) 2022-01-25 2025-05-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
AR128422A1 (es) 2022-02-02 2024-05-08 Akouos Inc Construcciones de anticuerpos anti-vegf y métodos relacionados para el tratamiento de los síntomas asociados al schwannoma vestibular
US20250144235A1 (en) 2022-02-02 2025-05-08 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
MX2024009660A (es) 2022-02-08 2024-08-14 Voyager Therapeutics Inc Variantes de capside de aav y usos de las mismas.
WO2023158836A1 (en) 2022-02-17 2023-08-24 Sana Biotechnology, Inc. Engineered cd47 proteins and uses thereof
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
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
US20250207126A1 (en) 2022-03-13 2025-06-26 Regenxbio Inc. Modified muscle-specific promoters
WO2023183623A1 (en) 2022-03-25 2023-09-28 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
WO2023187728A1 (en) 2022-04-01 2023-10-05 Takeda Pharmaceutical Company Limited Gene therapy for diseases with cns manifestations
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
EP4504797A1 (en) 2022-04-06 2025-02-12 The Trustees of The University of Pennsylvania Compositions and methods for treating her2 positive metastatic breast cancer and other cancers
EP4504258A1 (en) 2022-04-06 2025-02-12 The Trustees of the University of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
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
EP4508222A1 (en) 2022-04-11 2025-02-19 Centre National de la Recherche Scientifique Chemically-modified adeno-associated viruses
EP4508062A1 (en) 2022-04-11 2025-02-19 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP4507741A1 (en) 2022-04-14 2025-02-19 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
US20250277004A1 (en) 2022-04-18 2025-09-04 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
EP4518973A1 (en) 2022-05-03 2025-03-12 RegenxBio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
TW202400803A (zh) 2022-05-03 2024-01-01 美商銳進科斯生物股份有限公司 載體化抗補體抗體與補體劑及其投與
KR20250007619A (ko) 2022-05-06 2025-01-14 노파르티스 아게 신규 재조합 aav vp2 융합 폴리펩티드
US20250295809A1 (en) 2022-05-13 2025-09-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
JP2025518052A (ja) * 2022-05-24 2025-06-12 ケイト セラピューティクス， インコーポレイテッド Ｘｌｍｔｍを処置するための組成物
WO2023235791A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
AU2023289696A1 (en) 2022-06-24 2025-01-16 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
WO2024006741A1 (en) 2022-06-28 2024-01-04 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
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024017990A1 (en) 2022-07-21 2024-01-25 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
JP2025528068A (ja) 2022-08-03 2025-08-26 ボイジャー セラピューティクス インコーポレイテッド 血液脳関門を通過させるための組成物及び方法
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
JP2025527658A (ja) 2022-08-24 2025-08-22 リジェネックスバイオ インコーポレイテッド 組換えアデノ随伴ウイルス及びその使用
WO2024042485A1 (en) 2022-08-25 2024-02-29 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
UY40442A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav
AU2023347220A1 (en) 2022-09-22 2025-05-01 Dinaqor Ag Treatment of cardiomyopathy with aav gene therapy vectors
JP2025534347A (ja) 2022-09-30 2025-10-15 リジェネックスバイオ インコーポレイテッド 抗ｖｅｇｆ ｆａｂをコードする組換えウイルスベクターを用いる眼疾患の処置
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
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
JP2026500351A (ja) 2022-12-17 2026-01-06 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 心筋および骨格筋特異的標的化モチーフを有する組換えａａｖ変異体ベクターおよびそれを含む組成物
IT202200026595A1 (it) 2022-12-22 2024-06-22 Fond Telethon Ets Nuovi inibitori di regolatori epigenetici
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
WO2024151541A1 (en) 2023-01-09 2024-07-18 Sana Biotechnology, Inc. Type-1 diabetes autoimmune mouse
EP4649160A1 (en) 2023-01-12 2025-11-19 Nantes Université Chemically-modified adeno-associated virus
WO2024163747A2 (en) 2023-02-02 2024-08-08 University Of Rochester Competitive replacement of glial cells
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024163979A2 (en) * 2023-02-02 2024-08-08 Codexis, Inc. Engineered aav polypeptides
CN121358753A (zh) 2023-02-17 2026-01-16 渤健马萨诸塞州股份有限公司 用于递送有效载荷的含rgd的肽
EP4680741A2 (en) 2023-03-15 2026-01-21 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
EP4661965A1 (en) * 2023-03-25 2025-12-17 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
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024220463A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav5 capsid with non-canonical amino acid incorporation and uses thereof
WO2024220389A2 (en) * 2023-04-17 2024-10-24 University Of Massachusetts Aav2 variants and uses thereof
AU2024258816A1 (en) 2023-04-18 2025-10-23 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
AU2024264889A1 (en) 2023-05-03 2025-11-13 Sana Biotechnology, Inc. Methods of dosing and administration of engineered islet cells
AU2024265504A1 (en) 2023-05-03 2025-12-11 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
AU2024266138A1 (en) 2023-05-03 2025-11-13 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
WO2024229125A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
AU2024266519A1 (en) 2023-05-03 2025-12-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2024229389A1 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2024229425A1 (en) 2023-05-04 2024-11-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
WO2024233999A2 (en) 2023-05-11 2024-11-14 University Hospitals Cleveland Medical Center Anxiolytic therapy
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
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
TW202516019A (zh) 2023-06-29 2025-04-16 賓州大學委員會 具中樞神經系統靶向模體的突變aav及含有其之組成物
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
TW202535908A (zh) 2023-11-02 2025-09-16 美商渤健麻塞諸塞州股份有限公司 中樞神經系統靶向之ａａｖ衣殼之重定向
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
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
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
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
WO2025188625A1 (en) 2024-03-03 2025-09-12 Passage Bio, Inc. Recombinant adeno-associated virus for treatment of neurodegenerative disorders
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025207948A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Aav capsids for targeting human transferrin receptor
WO2025207932A1 (en) 2024-03-27 2025-10-02 Biogen Ma Inc. Aav capsids comprising anti-transferrin antibody domains
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2025235734A2 (en) 2024-05-09 2025-11-13 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
GB202407038D0 (en) 2024-05-17 2024-07-03 Axovia Therapeutics Ltd Novel Gene Therapy
WO2025242657A1 (en) 2024-05-21 2025-11-27 Shape Biopharmaceuticals Ag Lipopeptide building blocks and aggregates
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2026006341A1 (en) 2024-06-24 2026-01-02 Regenxbio Inc. Microdystrophin gene therapy administration for treatment of dystrophinopathies
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026011008A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Expression control by skeletal muscle-expressed mirnas
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions

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
JPH07106156B2 (ja) 1983-10-28 1995-11-15 ジェネティックス、インスティチュ−ト ファクタ−▲ｖｉｉｉ▼および関連生産物の製造
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
FI86885C (fi) 1984-04-20 1992-10-26 Genentech Inc Foerfarande foer framstaellning av human rekombinantfaktor viii och nukleinsyrasekvenser och vektorer anvaend daertill
US4965199A (en) 1984-04-20 1990-10-23 Genentech, Inc. Preparation of functional human factor VIII in mammalian cells using methotrexate based selection
EP0182448A3 (en) 1984-08-24 1987-10-28 Genetics Institute, Inc. Production of factor viii and related products
FI98829C (fi) 1986-01-27 1997-08-25 Chiron Corp Menetelmä rekombinoidun proteiinikompleksin valmistamiseksi, jolla on humaanitekijä VIII:C-aktiivisuutta
US5595886A (en) 1986-01-27 1997-01-21 Chiron Corporation Protein complexes having Factor VIII:C activity and production thereof
US5422260A (en) 1986-05-29 1995-06-06 Genetics Institute, Inc. -Legal Affairs Human factor VIII:c muteins
US5451521A (en) 1986-05-29 1995-09-19 Genetics Institute, Inc. Procoagulant proteins
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
EP0670332A3 (en) 1989-11-17 1995-10-25 Novo Nordisk As Protein complexes with factor VIII: C activity and their production.
SE465222C5 (sv) 1989-12-15 1998-02-10 Pharmacia & Upjohn Ab Ett rekombinant, humant faktor VIII-derivat och förfarande för dess framställning
US5661008A (en) 1991-03-15 1997-08-26 Kabi Pharmacia Ab Recombinant human factor VIII derivatives
SE468050C (sv) 1991-03-15 1998-04-27 Pharmacia & Upjohn Ab Rekombinant derivat av human faktor VIII
CA2078721A1 (en) 1991-09-24 1993-03-25 Hiroshi Yonemura Process for preparing human coagulation factor viii protein complex
AU5602394A (en) 1992-11-13 1994-06-08 Duke University Chimeric blood coagulation proteins
US5563045A (en) 1992-11-13 1996-10-08 Genetics Institute, Inc. Chimeric procoagulant proteins
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
EP0629700A3 (en) 1993-06-10 1995-02-15 Miles Inc Mammalian vector and cell lines that have increased productivity.
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
WO1997003195A1 (en) 1995-07-11 1997-01-30 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
DE69738351T2 (de) * 1996-09-06 2008-11-13 The Trustees Of The University Of Pennsylvania Verfaheren zur durch rekombinante adeno-assoziierte virus-gerichtete gentherapie
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
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
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
JP2001517454A (ja) 1997-09-19 2001-10-09 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 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株
ATE402254T1 (de) 1998-05-28 2008-08-15 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
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
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
US6498244B1 (en) * 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
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
IL161827A0 (en) 2001-11-13 2005-11-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby
EP1463805B1 (en) * 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
CA2469785C (en) 2001-12-17 2014-04-22 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
US20060292117A1 (en) * 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
WO2005017149A1 (en) * 2003-06-03 2005-02-24 Cell Genesys, Inc. Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
EP3211085B1 (en) * 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
CN104293835B (zh) * 2005-04-07 2017-07-04 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
JP6224459B2 (ja) * 2011-02-17 2017-11-01 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 組織特異性を改変し、ａａｖ９媒介遺伝子導入を改善するための組成物および方法
EP2847337A4 (en) 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
AU2015320694B2 (en) 2014-09-24 2021-11-11 City Of Hope Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
EP3589730B1 (en) 2017-02-28 2023-12-27 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor
CN110573523A (zh) * 2017-02-28 2019-12-13 宾夕法尼亚州大学信托人 基于aav载体的流感疫苗

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