ATE286138T1 - Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren - Google Patents

Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren

Info

Publication number
ATE286138T1
ATE286138T1 AT99912640T AT99912640T ATE286138T1 AT E286138 T1 ATE286138 T1 AT E286138T1 AT 99912640 T AT99912640 T AT 99912640T AT 99912640 T AT99912640 T AT 99912640T AT E286138 T1 ATE286138 T1 AT E286138T1
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AT
Austria
Prior art keywords
aav
virus
gene product
contaminating
recombinant adeno
Prior art date
Application number
AT99912640T
Other languages
English (en)
Inventor
Guang-Ping Gao
James M Wilson
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Univ Pennsylvania
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Publication of ATE286138T1 publication Critical patent/ATE286138T1/de

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—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
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • 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
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • C12N2830/003—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor tet inducible
    • 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
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
    • C12N2830/006—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB tet repressible
    • 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
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AT99912640T 1998-03-20 1999-03-18 Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren ATE286138T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US7890898P 1998-03-20 1998-03-20
PCT/US1999/005870 WO1999047691A1 (en) 1998-03-20 1999-03-18 Compositions and methods for helper-free production of recombinant adeno-associated viruses

Publications (1)

Publication Number Publication Date
ATE286138T1 true ATE286138T1 (de) 2005-01-15

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
AT99912640T ATE286138T1 (de) 1998-03-20 1999-03-18 Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren

Country Status (9)

Country Link
EP (1) EP1064393B1 (de)
JP (1) JP2002506652A (de)
AT (1) ATE286138T1 (de)
AU (1) AU3097399A (de)
CA (1) CA2324225A1 (de)
DE (1) DE69922934T2 (de)
ES (1) ES2235470T3 (de)
PT (1) PT1064393E (de)
WO (1) WO1999047691A1 (de)

Families Citing this family (118)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6924128B2 (en) 1994-12-06 2005-08-02 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
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
US6893865B1 (en) 1999-04-28 2005-05-17 Targeted Genetics Corporation Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles
EP1916258B1 (de) 1999-08-09 2014-04-23 Targeted Genetics Corporation Erweiterte Expression einer einstämmigen, heterologen Nukleotidsequenz von rekombinanten viralen Vektoren durch Modellierung der Sequenz mit stammübergreifenden Basispaaren
WO2001059142A1 (en) * 2000-02-09 2001-08-16 Medimmune, Inc. Antibody gene therapy with adeno-associated viral vectors
WO2001094605A2 (en) * 2000-06-09 2001-12-13 University Of Florida Research Foundation, Inc. Recombinant aav vectors for gene therapy of obesity
DE10056210A1 (de) * 2000-11-13 2002-05-29 Arimedes Biotechnology Gmbh Virales Expressionssystem
DE10120265A1 (de) * 2001-04-25 2002-11-14 Deutsches Krebsforsch AAV-Helferplasmide zur Helfervirus-freien Verpackung und Pseudotypisierung von AAV-Vektoren
DE10137283A1 (de) * 2001-08-01 2003-02-27 Deutsches Krebsforsch AAV-Vektor-Verpackungsplasmide zur Helfervirus-freien Herstellung pseudotypisierter AAV-Partikel über Einzeltransfektion
SG10201506627PA (en) 2001-11-13 2015-10-29 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
CA2990322A1 (en) 2001-11-21 2003-06-05 The Trustees Of The University Of Pennsylvania Simian adenovirus nucleic acid and amino acid sequences, vectors containing same, and methods of use
EP1944043A1 (de) 2001-11-21 2008-07-16 The Trustees of the University of Pennsylvania Nukleinsäure- und Aminosäuresequenzen von Affen-Adenoviren, hergeleitete Vektoren und Verwendungsverfahren.
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
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
JP4811765B2 (ja) * 2004-06-22 2011-11-09 学校法人東海大学 外来遺伝子の誘導発現の制御が可能な発現ベクター
PT2220241T (pt) 2007-11-28 2016-12-26 Univ Pennsylvania Adenovírus compreendendo uma proteína hexão da cápside do adenovírus e símio sadv-39 e suas utilizações
JP5758124B2 (ja) 2007-11-28 2015-08-05 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 サルサブファミリーＣアデノウイルスＳＡｄＶ−４０、−３１および−３４ならびにそれらの用途
WO2009136977A2 (en) 2008-03-04 2009-11-12 The Trustees Of The University Of Pennsylvania Simian adenoviruses sadv-36,-42.1, -42.2, and -44 and uses thereof
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
US8940290B2 (en) 2008-10-31 2015-01-27 The Trustees Of The University Of Pennsylvania Simian adenoviruses SAdV-43, -45, -46, -47, -48, -49, and -50 and uses thereof
SG10202109219SA (en) 2009-05-02 2021-10-28 Genzyme Corp Gene therapy for neurodegenerative disorders
EP2435559A1 (de) 2009-05-29 2012-04-04 The Trustees Of The University Of Pennsylvania Affen-adenovirus 41 und verwendungen davon
AU2011332025B2 (en) 2010-11-23 2015-06-25 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1321, A1325, A1295, A1309 and A1322 and uses thereof
CN105473723A (zh) 2012-05-18 2016-04-06 宾夕法尼亚大学托管会 亚家族e猿腺病毒a1302、a1320、a1331和a1337及其用途
ES2821938T3 (es) 2014-03-09 2021-04-28 Univ Pennsylvania Composiciones útiles en el tratamiento de la deficiencia de ornitina transcarbamilasa (OTC)
US20180161455A1 (en) * 2015-06-10 2018-06-14 American Gene Technologies International, Inc. Non-integrating viral delivery system and methods of use therof
JP6890831B2 (ja) 2015-07-08 2021-06-18 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド Ｈｉｖ予備免疫化および免疫療法
EP3368054A4 (de) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. Regulierbare expression unter verwendung des adeno-assoziierten virus (aav)
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
DK3402483T3 (da) 2016-01-15 2024-01-02 American Gene Tech Int Inc Fremgangsmåder og sammensætninger til aktivering af gamma-delta-T-celler
WO2017139065A1 (en) 2016-02-08 2017-08-17 American Gene Technologies International Inc. Hiv vaccination and immunotherapy
JP7017247B2 (ja) 2016-03-09 2022-02-08 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド がんを処置するための組み合わせベクターおよび方法
WO2017213697A1 (en) * 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
AU2017292582C1 (en) 2016-07-08 2021-11-11 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
JP7176756B2 (ja) 2016-07-21 2022-11-22 アメリカン ジーン テクノロジーズ インターナショナル インコーポレイテッド パーキンソン病を処置するためのウイルスベクター
CA3057142A1 (en) 2017-04-03 2018-10-11 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
EP3662060A2 (de) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur aav-freisetzung
EP4454654A3 (de) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Behandlung von amyotropher lateralsklerose (als)
CA3077426A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
US20210010028A1 (en) 2018-03-06 2021-01-14 Voyager Therapeutics, Inc. Insect cell manufactured partial self-complementary aav genomes
EP3784780A1 (de) 2018-04-27 2021-03-03 Voyager Therapeutics, Inc. Verfahren zur messung der potenz von aadc-virusvektoren
US20220025396A1 (en) * 2018-05-07 2022-01-27 Spark Therapeutics, Inc. Plasmid free aav vector producing cell lines
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US20210207167A1 (en) 2018-05-16 2021-07-08 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
EP3807404A1 (de) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Manipulierte 5'-unübersetzte regionen (5' utr) zur herstellung von aav
EP3818161A1 (de) 2018-07-02 2021-05-12 Voyager Therapeutics, Inc. Behandlung von amyotropher lateralsklerose und rückenmarksassoziierten erkrankungen
MX2021000810A (es) 2018-07-24 2021-04-28 Voyager Therapeutics Inc Sistemas y metodos para producir formulaciones de terapia genetica.
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
EP3861107A1 (de) 2018-10-05 2021-08-11 Voyager Therapeutics, Inc. Gentechnisch hergestellte nukleinsäurekonstrukte, die aav-produktionsproteine codieren
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3867389A1 (de) 2018-10-15 2021-08-25 Voyager Therapeutics, Inc. Expressionsvektoren zur massenproduktion von raav im baculovirus/sf9-system
TW202039858A (zh) 2019-01-18 2020-11-01 美商航海家醫療公司 用於生產aav顆粒之方法及系統
WO2020223274A1 (en) 2019-04-29 2020-11-05 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
WO2020227515A1 (en) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
EP4010465A1 (de) 2019-08-09 2022-06-15 Voyager Therapeutics, Inc. Zellkulturmedium zur verwendung bei der herstellung von gentherapieprodukten in bioreaktoren
EP4022070A1 (de) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Kontrollierte expression viraler proteine
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
WO2021202651A1 (en) 2020-04-01 2021-10-07 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
EP4135841A1 (de) 2020-04-15 2023-02-22 Voyager Therapeutics, Inc. Tau-bindende verbindungen
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
CA3182970A1 (en) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
EP4192514A1 (de) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Zellkulturmedium zur verwendung bei der herstellung von gentherapieprodukten in bioreaktoren
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
MX2023004052A (es) 2020-10-15 2023-05-03 Hoffmann La Roche Constructos de acido nucleico para transcripcion de arn asociado a un virus (arn va).
EP4237545A1 (de) 2020-11-02 2023-09-06 BioMarin Pharmaceutical Inc. Verfahren zur anreicherung von adeno-assoziiertem virus
EP4291665A1 (de) * 2021-02-11 2023-12-20 Lonza Houston Inc. Herstellung von adeno-assoziiertem virus (aav)
EP4293118A4 (de) * 2021-02-12 2025-01-22 FUJIFILM Corporation Kit zur herstellung von adeno-assoziiertem virus und verwendung des kits
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4323015A4 (de) 2021-04-16 2025-10-22 Askbio Inc Rationale polyploide aav-virionen, die die blut-hirn-schranke durchqueren und eine reduzierte humorale antwort hervorrufen
US20240376496A1 (en) 2021-09-03 2024-11-14 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034990A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
JP2024534888A (ja) 2021-09-03 2024-09-26 ビオマリン プハルマセウトイカル インコーポレイテッド Ａａｖカプシド組成物及び送達方法
US20250122530A1 (en) 2021-09-03 2025-04-17 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
JP2024533174A (ja) 2021-09-03 2024-09-12 ビオマリン プハルマセウトイカル インコーポレイテッド Ａａｖカプシド組成物及び送達方法
US20250223376A1 (en) 2021-09-20 2025-07-10 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
JP2024540221A (ja) 2021-11-02 2024-10-31 ボイジャー セラピューティクス インコーポレイテッド Ａａｖカプシドバリアント及びその使用
WO2023091948A1 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
KR20240123828A (ko) 2021-12-16 2024-08-14 유니버시티 오브 던디 알파-시누클레인의 표적화 분해
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
KR20240161976A (ko) 2022-02-08 2024-11-13 보이저 테라퓨틱스, 인크. Aav 캡시드 변이체 및 이의 용도
GB202201713D0 (en) 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
IL316719A (en) 2022-06-02 2024-12-01 Voyager Therapeutics Inc Adeno-associated virus capsid variants and their uses
EP4547690A1 (de) 2022-06-28 2025-05-07 Voyager Therapeutics, Inc. Aav-kapsidvarianten und verwendungen davon
TW202424201A (zh) 2022-07-06 2024-06-16 美商航海家醫療公司 Ａａｖ殼體變異體及其用途
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CA3266790A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. TAU PROTEIN BINDING COMPOUNDS
UY40442A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav
KR20250108100A (ko) 2022-09-22 2025-07-15 디나코 아게 Aav 유전자 치료법 벡터를 이용한 심근병증의 치료
IL320511A (en) 2022-12-29 2025-06-01 Voyager Therapeutics Inc Compositions and methods for regulating MAPT
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024229173A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
CN121079310A (zh) 2023-05-03 2025-12-05 沃雅戈治疗公司 用于治疗与cdkl5缺乏相关的病症的组合物及方法
WO2024229164A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
TW202444917A (zh) 2023-05-04 2024-11-16 美商航海家醫療公司 Ａａｖ衣殼變異體及其用途
WO2025038430A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025038802A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025038800A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
WO2025038795A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
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
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
TW202540436A (zh) 2023-12-05 2025-10-16 美商航海家醫療公司 用於治療與共濟蛋白(frataxin)缺陷相關之病症之組合物及方法
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
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
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors
WO2026028089A1 (en) 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5436146A (en) * 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US6686200B1 (en) * 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
US5872005A (en) * 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
ATE237352T1 (de) * 1995-06-07 2003-05-15 Univ North Carolina Helfervirus-freie herstellung von aav
US6004797A (en) * 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
ATE465267T1 (de) * 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen

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WO1999047691A1 (en) 1999-09-23
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CA2324225A1 (en) 1999-09-23
DE69922934T2 (de) 2005-12-08
DE69922934D1 (de) 2005-02-03
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AU3097399A (en) 1999-10-11
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