BR112022023106A2 - Redirecionamento de tropismo de capsídeos de aav - Google Patents

Redirecionamento de tropismo de capsídeos de aav

Info

Publication number
BR112022023106A2
BR112022023106A2 BR112022023106A BR112022023106A BR112022023106A2 BR 112022023106 A2 BR112022023106 A2 BR 112022023106A2 BR 112022023106 A BR112022023106 A BR 112022023106A BR 112022023106 A BR112022023106 A BR 112022023106A BR 112022023106 A2 BR112022023106 A2 BR 112022023106A2
Authority
BR
Brazil
Prior art keywords
tropism
redirect
aav capsid
capsid proteins
adeno
Prior art date
Application number
BR112022023106A
Other languages
English (en)
Inventor
E Nonnenmacher Mathieu
Hou Jinzhao
Wang Wei
Child Matthew
Li Shaoyong
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 BR112022023106A2 publication Critical patent/BR112022023106A2/pt

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    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Abstract

REDIRECIONAMENTO DE TROPISMO DE CAPSÍDEOS DE AAV. A presente invenção refere-se a composições, métodos e processos para a preparação, uso e/ou formulação de proteínas do capsídeo do vírus adenoassociado, em que as proteínas do capsídeo compreendem inserções peptídicas de direcionamento para tropismo potencializado para um tecido alvo.
BR112022023106A 2020-05-13 2021-03-31 Redirecionamento de tropismo de capsídeos de aav BR112022023106A2 (pt)

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
BR112022023106A2 true BR112022023106A2 (pt) 2023-01-17

Family

ID=75639982

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022023106A BR112022023106A2 (pt) 2020-05-13 2021-03-31 Redirecionamento de tropismo de capsídeos de aav

Country Status (14)

Country Link
US (2) US20230203102A1 (pt)
EP (1) EP4149955A1 (pt)
JP (1) JP2023525810A (pt)
KR (1) KR20230022175A (pt)
CN (1) CN116096734A (pt)
AU (1) AU2021273447A1 (pt)
BR (1) BR112022023106A2 (pt)
CA (1) CA3182970A1 (pt)
CO (1) CO2022016156A2 (pt)
IL (1) IL298001A (pt)
MX (1) MX2022014256A (pt)
PE (1) PE20230767A1 (pt)
TW (1) TW202208397A (pt)
WO (1) WO2021230987A1 (pt)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2359865B1 (en) * 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
CA3182970A1 (en) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
TW202246516A (zh) 2021-03-03 2022-12-01 美商航海家醫療公司 病毒蛋白之控制表現
WO2022187548A1 (en) 2021-03-03 2022-09-09 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
CA3235632A1 (en) 2021-11-02 2023-05-11 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
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
WO2023154693A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
WO2024059739A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. Tau binding compounds

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
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
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
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
IL128780A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US20030215422A1 (en) 1996-09-11 2003-11-20 John A. Chiorini Aav4 vector and uses thereof
CN1244215B (zh) 1996-11-20 2010-11-03 荷兰克鲁塞尔公司 改进的腺病毒载体生产和纯化方法
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
CA2304168A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
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
JP2002506652A (ja) 1998-03-20 2002-03-05 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を含まない製造のための組成物及び方法
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
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
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
WO2000075353A1 (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
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
JP2003523320A (ja) 1999-09-29 2003-08-05 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 ウイルスベクターの迅速なｐｅｇ改変方法、高められた遺伝子形質導入のための組成物、高められた物理的安定性を伴う組成物、およびそのための用途
WO2002081711A1 (en) 2001-04-06 2002-10-17 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
EP2573170B1 (en) 2001-12-17 2017-12-20 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
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
EP2277996B1 (en) 2003-05-21 2014-09-03 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
ES2629380T3 (es) 2003-06-19 2017-08-09 Genzyme Corporation Viriones de AAV con inmunorreactividad reducida y usos de los mismos
US7491508B2 (en) 2003-06-20 2009-02-17 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
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
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
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
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
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
CA2566286A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere 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
WO2007046703A2 (en) 2005-10-20 2007-04-26 Amsterdam Molecular Therapeutics B.V. Improved aav vectors produced in insect cells
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
ATE553206T1 (de) 2006-08-24 2012-04-15 Virovek Inc Expression von genen mit überlappenden offenen leserastern in insektenzellen, verfahren und zusammensetzungen dafür
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un 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
WO2010005001A1 (ja) 2008-07-09 2010-01-14 株式会社 東芝 Ｘ線管用ターゲットおよびそれを用いたｘ線管、ｘ線検査装置ならびにｘ線管用ターゲットの製造方法
DE102008060558B4 (de) 2008-12-04 2013-09-26 Eduard Buzetzki Verfahren zum katalytischen Cracken von Pflanzenölen und tierischen Fetten
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel 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
CA2793309A1 (en) 2010-03-17 2011-09-22 Anaptysbio, Inc. Method of producing transcripts using cryptic splice sites
WO2011133890A1 (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
DK2673289T3 (da) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
CA2833870C (en) 2011-04-22 2020-03-10 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv 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
AU2013323270B2 (en) 2012-09-28 2017-09-28 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
US9938541B2 (en) 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
DK2954051T3 (da) 2013-02-08 2019-07-08 Univ Pennsylvania Modificeret kapsid til genoverførsel til behandling af nethinden
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
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
WO2015164757A1 (en) 2014-04-25 2015-10-29 Oregon Health & Science University Methods of viral neutralizing antibody epitope mapping
EP3137497B1 (en) 2014-05-02 2021-04-07 Genzyme Corporation Aav vectors for retinal and cns gene therapy
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
RU2668395C9 (ru) 2014-07-11 2018-10-29 Хуавей Текнолоджиз Ко., Лтд. Сетевое устройство, устройство пользователя и способы трансляции сигнала доступа
DK3198018T3 (da) 2014-09-24 2021-03-01 Hope City Vektorvarianter af adenoassocieret virus til højeffektiv genomredigering og fremgangsmåder dertil
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
US20180030096A1 (en) 2015-02-03 2018-02-01 University Of Florida Research Foundation, Inc. Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof
JP6873907B2 (ja) 2015-02-20 2021-05-19 ユニバーシティー オブ アイオワ リサーチ ファウンデーション 遺伝性眼疾患を治療するための方法及び組成物
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
CA2982129A1 (en) 2015-04-08 2016-10-13 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
AU2016332821B2 (en) 2015-09-28 2022-02-17 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
EP3374494A4 (en) 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. CRISPR COMPOSITIONS AND METHODS OF USE 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
SG11201804713VA (en) 2015-12-11 2018-07-30 California Inst Of Techn TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
WO2017143100A1 (en) 2016-02-16 2017-08-24 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
EP3423154B1 (en) 2016-03-01 2021-05-05 Intraop Medical Corporation Low energy electron beam radiation system that generates electron beams with precisely controlled and adjustable penetration depth useful for therapeutic applications
EP3827812A1 (en) 2016-07-29 2021-06-02 The Regents of the University of California Adeno-associated virus virions with variant capsid and methods of use thereof
WO2018035213A1 (en) 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US20200316221A1 (en) 2016-10-13 2020-10-08 University Of Massachusetts Aav capsid designs
US20230048025A1 (en) 2016-12-22 2023-02-16 Oregon Health & Science University Adeno associated viral vectors
US11680275B2 (en) * 2017-06-06 2023-06-20 University Of Massachusetts Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome
KR20200022434A (ko) 2017-06-27 2020-03-03 리제너론 파마슈티칼스 인코포레이티드 유전 물질을 인간 세포 내로 표적화하여 도입하기 위한 향성-변형 재조합 바이러스 벡터 및 이의 용도
EP3645021A4 (en) 2017-06-30 2021-04-21 Intima Bioscience, Inc. ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
NZ763018A (en) 2017-09-20 2023-02-24 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
WO2019068854A1 (en) 2017-10-06 2019-04-11 Ospedale San Raffaele S.R.L. GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP7393766B2 (ja) 2018-02-28 2023-12-07 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 抗体回避ウイルスベクターのための方法および組成物
CN112351787A (zh) 2018-05-04 2021-02-09 俄勒冈健康与科学大学 人抗aav2衣壳多克隆抗体表位
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
EP3793615A2 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Directed evolution of aav to improve tropism for cns
WO2019222441A1 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
WO2020014471A1 (en) * 2018-07-11 2020-01-16 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of agents across the blood-brain barrier
EP3830107A2 (en) 2018-08-03 2021-06-09 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
WO2020072683A1 (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
CN113646005A (zh) 2018-12-21 2021-11-12 宾夕法尼亚州大学信托人 用于drg特异性降低转基因表达的组合物
CA3128205A1 (en) 2019-01-30 2020-08-06 The Broad Institute, Inc. Systems for evolved adeno-associated viruses (aavs) for targeted delivery
EP3917566A4 (en) 2019-01-31 2022-10-26 Oregon Health & Science University METHODS OF USING DIRECTED, TRANSCRIPTION-DEPENDENT EVOLUTION OF AAV CAPSIDS
EA202192501A1 (ru) 2019-03-21 2021-12-16 Страйдбайо, Инк. Рекомбинантные аденоассоциированные вирусные векторы
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
AU2020253435A1 (en) 2019-04-01 2021-11-18 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
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
KR20220007056A (ko) 2019-04-11 2022-01-18 캘리포니아 인스티튜트 오브 테크놀로지 뇌에서 증진된 특이성을 갖는 바이러스 조성물
US20200370137A1 (en) 2019-04-26 2020-11-26 Sangamo Therapeutics, Inc. Engineering aav
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
WO2020223280A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
US20220281922A1 (en) 2019-08-02 2022-09-08 Voyager Therapeutics, Inc. Aav variants with enhanced tropism
US20230131352A1 (en) 2020-04-01 2023-04-27 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
CA3182970A1 (en) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
US20230048732A1 (en) 2020-05-26 2023-02-16 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
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CA3235632A1 (en) 2021-11-02 2023-05-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023092004A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of tau-related disorders
WO2023091948A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
WO2023154693A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof

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