MX2022014256A - Redirección del tropismo de las cápsides de aav. - Google Patents

Redirección del tropismo de las cápsides de aav.

Info

Publication number
MX2022014256A
MX2022014256A MX2022014256A MX2022014256A MX2022014256A MX 2022014256 A MX2022014256 A MX 2022014256A MX 2022014256 A MX2022014256 A MX 2022014256A MX 2022014256 A MX2022014256 A MX 2022014256A MX 2022014256 A MX2022014256 A MX 2022014256A
Authority
MX
Mexico
Prior art keywords
tropism
redirection
aav capsids
capsid proteins
adeno
Prior art date
Application number
MX2022014256A
Other languages
English (en)
Inventor
Mathieu E Nonnenmacher
Jinzhao Hou
Wei Wang
Matthew Child
Shaoyong Li
Original Assignee
Voyager Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of MX2022014256A publication Critical patent/MX2022014256A/es

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • C40—COMBINATORIAL TECHNOLOGY
    • C40B—COMBINATORIAL CHEMISTRY; LIBRARIES, e.g. CHEMICAL LIBRARIES
    • C40B40/00—Libraries per se, e.g. arrays, mixtures
    • C40B40/04—Libraries containing only organic compounds
    • C40B40/06—Libraries containing nucleotides or polynucleotides, or derivatives thereof
    • C40B40/08—Libraries containing RNA or DNA which encodes proteins, e.g. gene libraries
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors
    • 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

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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)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

La descripción se refiere a composiciones, métodos y procesos para la preparación, uso y/o formulación de proteínas de la cápside de virus adenoasociados, en donde las proteínas de la cápside comprenden insertos de péptidos de direccionamiento para mejorar el tropismo a un tejido diana.
MX2022014256A 2020-05-13 2021-03-31 Redirección del tropismo de las cápsides de aav. MX2022014256A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063023927P 2020-05-13 2020-05-13
US202063122300P 2020-12-07 2020-12-07
PCT/US2021/025061 WO2021230987A1 (en) 2020-05-13 2021-03-31 Redirection of tropism of aav capsids

Publications (1)

Publication Number Publication Date
MX2022014256A true MX2022014256A (es) 2023-01-11

Family

ID=75639982

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022014256A MX2022014256A (es) 2020-05-13 2021-03-31 Redirección del tropismo de las cápsides de aav.

Country Status (14)

Country Link
US (3) US20230203102A1 (es)
EP (1) EP4149955A1 (es)
JP (1) JP2023525810A (es)
KR (1) KR20230022175A (es)
CN (1) CN116096734A (es)
AU (1) AU2021273447A1 (es)
BR (1) BR112022023106A2 (es)
CA (1) CA3182970A1 (es)
CO (1) CO2022016156A2 (es)
IL (1) IL298001A (es)
MX (1) MX2022014256A (es)
PE (1) PE20230767A1 (es)
TW (1) TW202208397A (es)
WO (1) WO2021230987A1 (es)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3409296A1 (en) * 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
EP3880823A4 (en) * 2018-11-16 2022-08-17 Asklepios Biopharmaceutical, Inc. THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR THE TREATMENT OF PUMP DISEASE
WO2021230987A1 (en) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
IL304880A (en) 2021-02-12 2023-10-01 Alnylam Pharmaceuticals Inc Superoxide dismutase 1 (SOD1) IRNA compositions and methods of using them to treat or prevent superoxide dismutase 1- (SOD1-) associated neurodegenerative diseases
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2023034997A1 (en) * 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023044483A2 (en) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
TW202334436A (zh) 2021-11-02 2023-09-01 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
WO2023092002A2 (en) * 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis and disorders associatedwith the spinal cord
WO2023092004A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023091948A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
WO2023148617A1 (en) * 2022-02-01 2023-08-10 The Broad Institute, Inc. Adeno-associated viral vectors and uses thereof
TW202346599A (zh) 2022-02-08 2023-12-01 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
WO2023194796A2 (en) * 2022-04-04 2023-10-12 Vectory B.V. Recombinant aav capsid proteins
WO2023220695A2 (en) 2022-05-13 2023-11-16 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023220476A2 (en) * 2022-05-13 2023-11-16 The Broad Institute, Inc. Adeno-associated viral vectors and uses thereof
WO2023225564A1 (en) * 2022-05-18 2023-11-23 The Broad Institute, Inc. Engineered viral capsids with increased stability and methods of use thereof
WO2023235791A1 (en) 2022-06-02 2023-12-07 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2023250388A1 (en) 2022-06-22 2023-12-28 Voyager Therapeutics, Inc. Tau binding compounds
WO2024006741A1 (en) 2022-06-28 2024-01-04 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024030976A2 (en) 2022-08-03 2024-02-08 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2024055020A2 (en) * 2022-09-09 2024-03-14 The Regents Of The University Of California Reprogramming tropism via displayed peptides tiling receptor-ligands
WO2024059739A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. Tau binding compounds
WO2024086747A1 (en) 2022-10-19 2024-04-25 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2024100633A1 (en) 2022-11-13 2024-05-16 Alexion Pharma International Operations Limited Gene therapy for frontotemporal dementia
CN117264912A (zh) * 2023-09-25 2023-12-22 中国科学院深圳先进技术研究院 一种重组腺相关病毒颗粒、重组腺相关病毒载体系统及其应用

Family Cites Families (135)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5625048A (en) 1994-11-10 1997-04-29 The Regents Of The University Of California Modified green fluorescent proteins
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5741657A (en) 1995-03-20 1998-04-21 The Regents Of The University Of California Fluorogenic substrates for β-lactamase and methods of use
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
JP2001500015A (ja) 1996-09-06 2001-01-09 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The Aav4 vector and uses thereof
KR100503701B1 (ko) 1996-11-20 2005-07-26 인트로겐 테라페티스, 인코퍼레이티드 아데노바이러스 벡터를 생산하고 정제하는 개선된 방법
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP1015619A1 (en) 1997-09-19 2000-07-05 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
CA2303768C (en) 1997-09-19 2009-11-24 The Trustees Of The University Of Pennsylvania Methods and vector constructs useful for production of recombinant aav
CA2324225A1 (en) 1998-03-20 1999-09-23 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6984517B1 (en) 1998-05-28 2006-01-10 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector and uses thereof
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
FR2786478B1 (fr) 1998-11-30 2000-12-22 Commissariat Energie Atomique Confinement du cesium et/ou du rubidium dans des ceramiques apatitiques
JP4693244B2 (ja) 1999-03-18 2011-06-01 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 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
CA2375098A1 (en) 1999-06-02 2000-12-14 Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
AU1071701A (en) 1999-09-29 2001-04-30 Trustees Of The University Of Pennsylvania, The Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
EP1373527A1 (en) 2001-04-06 2004-01-02 CropDesign N.V. The use of double and opposite recombination sites for the single step cloning of two dna segments
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
US6845866B2 (en) 2002-10-10 2005-01-25 N. Henning Zieger Dispenser
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
SI2277996T1 (sl) 2003-05-21 2014-12-31 Genzyme Corporation Postopki za izdelavo pripravkov rekombinantnih AAV virionov v bistvu brez praznih kapsid
ES2862206T3 (es) 2003-06-19 2021-10-07 Genzyme Corp Viriones de AAV con inmunorreactividad disminuida y sus usos
EP1636370B1 (en) 2003-06-20 2014-04-16 The Trustees of The University of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
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
EP2298926A1 (en) 2003-09-30 2011-03-23 The Trustees of The University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
SI1945779T1 (sl) 2005-10-20 2013-07-31 Uniqure Ip B.V. Izboljĺ ani aav vektorji proizvedeni v celicah insekta
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
US8945918B2 (en) 2006-08-24 2015-02-03 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
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
EP2312608B1 (en) 2008-07-09 2015-01-14 Kabushiki Kaisha Toshiba Target for x-ray tube, x-ray tube using the same, x-ray inspection system, and method for producing target for x-ray tube
DE102008060558B4 (de) 2008-12-04 2013-09-26 Eduard Buzetzki Verfahren zum katalytischen Cracken von Pflanzenölen und tierischen Fetten
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
US9163261B2 (en) 2010-02-22 2015-10-20 Koteswara Rao KOLLIPARA Adeno-associated virus 2/8—micro RNA-101 therapy for liver cancer
EP2547781A4 (en) 2010-03-17 2014-04-23 Anaptysbio Inc PROCESS FOR PRODUCING TRANSCRIPTION PRODUCTS USING CRYPTIC SPLICE SITES
CA3066596A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US10415056B2 (en) 2010-11-10 2019-09-17 Fred Hutchinson Cancer Research Center Compositions and methods for generating adeno-associated viral vectors with undetectable capsid gene contamination
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
KR20220056884A (ko) 2011-04-22 2022-05-06 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 지니는 아데노-관련 바이러스 및 이의 사용 방법
US9169299B2 (en) 2011-08-24 2015-10-27 The Board Of Trustees Of The Leleand Stanford Junior University AAV capsid proteins for nucleic acid transfer
WO2013096955A1 (en) 2011-12-23 2013-06-27 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
US9644205B2 (en) 2012-04-25 2017-05-09 The Regents Of The University Of California Synthetic promoter for modulating gene expression
CN111621507A (zh) 2012-09-28 2020-09-04 北卡罗来纳-查佩尔山大学 靶向少突胶质细胞的aav载体
CN104937100B (zh) 2012-12-25 2020-04-03 宝生物工程株式会社 Aav 变体
EP2954051B1 (en) 2013-02-08 2019-03-27 The Trustees Of The University Of Pennsylvania Modified aav8 capsid for gene transfer for retinal therapies
AU2014227766B2 (en) 2013-03-15 2018-10-04 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV vectors
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
EP3134522B1 (en) 2014-04-25 2021-10-06 University of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
US10746742B2 (en) 2014-04-25 2020-08-18 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
MX2016014220A (es) 2014-05-02 2017-02-06 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc.
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2016005001A1 (en) 2014-07-11 2016-01-14 Huawei Technologies Co., Ltd. Network device, user device and methods of access signal broadcast
ES2856090T3 (es) 2014-09-24 2021-09-27 Hope City Variantes de vectores de virus adenoasociados para la edición genómica de alta eficacia y sus métodos
RU2738421C2 (ru) 2014-10-21 2020-12-14 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
US10472650B2 (en) 2015-02-20 2019-11-12 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
US10081659B2 (en) 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
EP3280451B8 (en) 2015-04-08 2019-09-25 The United States of America, as represented by the Secretary, Department of Health and Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
US20180216133A1 (en) 2015-07-17 2018-08-02 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
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
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
US10240145B2 (en) 2015-11-25 2019-03-26 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas-mediated genome editing to treat EGFR-mutant lung cancer
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
EP3417055B1 (en) 2016-02-16 2021-10-13 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
WO2017151763A1 (en) 2016-03-01 2017-09-08 Intraop Medical Corporation Low energy electron beam radiation system that generates electron beams with precisely controlled and adjustable penetration depth useful for therapeutic applications
CN116286986A (zh) 2016-07-29 2023-06-23 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
EP3500676A4 (en) 2016-08-16 2020-07-01 The University of North Carolina at Chapel Hill METHOD AND COMPOSITIONS FOR TARGETED GENE TRANSFER
US20200316221A1 (en) 2016-10-13 2020-10-08 University Of Massachusetts Aav capsid designs
EP3558393A4 (en) 2016-12-22 2020-11-11 Oregon Health & Science University ADENO-ASSOCIATED VIRAL VECTORS
BR112019025732A2 (pt) * 2017-06-06 2020-06-30 University Of Massachusetts vetores de aav auto-reguladores para expressão segura de mecp2 na síndrome de rett
DK3645551T3 (da) 2017-06-27 2024-05-06 Regeneron Pharma Tropisme-modificerede rekombinante virusvektorer og anvendelser deraf til targetteret indføring af genetisk materiale i menneskeceller
WO2019006418A2 (en) 2017-06-30 2019-01-03 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
RU2770922C2 (ru) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
WO2019068854A1 (en) 2017-10-06 2019-04-11 Ospedale San Raffaele S.R.L. GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症（ａｌｓ）の治療
EP3759218A4 (en) 2018-02-28 2021-12-08 The University of North Carolina at Chapel Hill METHODS AND COMPOSITIONS FOR ANTIBODY AVOIDING VIRAL VECTORS
WO2019213668A1 (en) 2018-05-04 2019-11-07 Oregon Health & Science University Human anti-aav2 capsid polyclonal antibody epitopes
EP3793616A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019222444A2 (en) * 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
WO2019222441A1 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
EP3820885A4 (en) * 2018-07-11 2022-04-20 The Brigham & Women's Hospital, Inc. METHODS AND COMPOSITIONS FOR ADMINISTRATION OF DRUGS ACROSS THE BLOOD-BRAIN BARRIER
EP3830107A2 (en) 2018-08-03 2021-06-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
AU2019346447A1 (en) 2018-09-26 2021-04-29 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
CA3115217A1 (en) 2018-10-02 2020-04-09 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2019401314A1 (en) 2018-12-21 2021-06-24 The Trustees Of The University Of Pennsylvania Compositions for DRG-specific reduction of transgene expression
EP3917945A4 (en) 2019-01-30 2023-02-08 The Broad Institute Inc. ADVANCED ADENO-ASSOCIATED VIRUS (AAVS) TARGETED DELIVERY SYSTEMS
CN113924115A (zh) 2019-01-31 2022-01-11 俄勒冈健康与科学大学 用于aav衣壳的使用转录依赖性定向进化的方法
TW202102525A (zh) 2019-03-21 2021-01-16 美商史崔德生物公司 重組腺相關病毒載體
EP3715358A1 (en) 2019-03-28 2020-09-30 Universität zu Köln Mutated adeno-associated virus capsid proteins, aav particle comprising the same and liver directed aav vector gene therapy
JP2022527199A (ja) 2019-04-01 2022-05-31 テナヤ セラピューティクス， インコーポレイテッド 操作されたカプシドを有するアデノ随伴ウイルス
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
WO2020210655A1 (en) 2019-04-11 2020-10-15 California Institute Of Technology Virus compositions with enhanced specificity in the brain
TW202106699A (zh) 2019-04-26 2021-02-16 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼
AU2020261435A1 (en) 2019-04-26 2021-11-25 Sangamo Therapeutics, Inc. Engineering AAV
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2021025995A1 (en) 2019-08-02 2021-02-11 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
EP4126910A1 (en) 2020-04-01 2023-02-08 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2021216456A2 (en) 2020-04-20 2021-10-28 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
EP4143305A2 (en) 2020-05-01 2023-03-08 The Broad Institute, Inc. Engineered central nervous system compositions
WO2021230987A1 (en) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2021242909A1 (en) 2020-05-26 2021-12-02 Shape Therapeutics Inc. High throughput engineering of functional aav capsids
CA3192600A1 (en) 2020-08-21 2022-02-24 Capsida, Inc. Adeno-associated virus compositions having preferred expression levels
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
TW202334436A (zh) 2021-11-02 2023-09-01 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
WO2023091948A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023092004A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023092002A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis and disorders associatedwith the spinal cord
TW202346599A (zh) 2022-02-08 2023-12-01 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途

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