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

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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
Authority
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 (123)

* 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
US6989264B2 (en) 1997-09-05 2006-01-24 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
US20010034062A1 (en) * 2000-02-09 2001-10-25 Scott Koenig Antibody gene therapy with adeno-associated viral vectors
EP1290206A2 (de) * 2000-06-09 2003-03-12 University of Florida Rekombinante aav-vektoren für die gentherapie von fettleibigkeit
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
SG10202108118RA (en) 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
HU230364B1 (hu) 2001-11-21 2016-03-29 The Trustees Of The University Of Pennsylvania Simian adenovírus nukleinsav és aminosav-szekvencia, azt tartalmazó vektorok, és eljárások annak alkalmazására
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
KR101761691B1 (ko) 2007-11-28 2017-07-26 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 유인원 e 아데노바이러스 sadv-39, -25.2, -26, -30, -37, 및 -38
WO2009105084A2 (en) 2007-11-28 2009-08-27 The Trustees Of The University Of Pennsylvania Simian subfamily c adenoviruses sadv-40, -31, and-34 and uses thereof
EP2250255A2 (de) 2008-03-04 2010-11-17 The Trustees of the University of Pennsylvania Simian-adenoviren sadv-36, -42.1, -42.2 und -44 sowie anwendungen davon
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
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
WO2010138675A1 (en) 2009-05-29 2010-12-02 The Trustees Of The University Of Pennsylvania Simian adenovirus 41 and uses thereof
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
SG10201609511XA (en) 2012-05-18 2016-12-29 Univ Pennsylvania Subfamily e simian adenoviruses a1302, a1320, a1331 and a1337 and uses thereof
JP6822841B2 (ja) 2014-03-09 2021-01-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物
US20180161455A1 (en) * 2015-06-10 2018-06-14 American Gene Technologies International, Inc. Non-integrating viral delivery system and methods of use therof
WO2017007994A1 (en) 2015-07-08 2017-01-12 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
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
CN111569090A (zh) 2016-01-15 2020-08-25 美国基因技术国际有限公司 用于活化γ-δ T细胞的方法和组合物
JP7153332B2 (ja) 2016-02-08 2022-10-14 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ワクチン接種および免疫療法
ES2911448T3 (es) 2016-03-09 2022-05-19 American Gene Tech Int Inc Vectores combinados y métodos para el tratamiento del cáncer
US11976292B2 (en) * 2016-06-08 2024-05-07 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
EP3481418A4 (de) 2016-07-08 2020-03-11 American Gene Technologies International Inc. Hiv-vorimmunisierung und -immuntherapie
US11583562B2 (en) 2016-07-21 2023-02-21 American Gene Technologies International Inc. Viral vectors for treating Parkinson's disease
JP2020512815A (ja) 2017-04-03 2020-04-30 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 フェニルケトン尿症を処置するための組成物および方法
WO2018232359A1 (en) 2017-06-16 2018-12-20 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
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
EP4454654A3 (de) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Behandlung von amyotropher lateralsklerose (als)
US11434502B2 (en) 2017-10-16 2022-09-06 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
US12129476B2 (en) 2018-04-27 2024-10-29 Voyager Therapeutics, Inc. Methods for measuring the potency of AADC viral vectors
JP2021522813A (ja) * 2018-05-07 2021-09-02 スパーク セラピューティクス インコーポレイテッドSpark Therapeutics, Inc. プラスミドを用いないaavベクター産生細胞株
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3794126A1 (de) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von morbus parkinson
EP3793686A1 (de) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Aav-serotypen zur gehirnspezifischen payload-übertragung
EP3793615A2 (de) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Gerichtete entwicklung von aav zur verbesserung des tropismus für zns
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CA3103963A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
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
EP3861010A1 (de) 2018-10-02 2021-08-11 Voyager Therapeutics, Inc. Umlenkung des tropismus von aav-kapsiden
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
JP2022512621A (ja) 2018-10-05 2022-02-07 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産生タンパク質をコードする操作された核酸コンストラクト
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
MX2021008542A (es) 2019-01-18 2021-11-12 Voyager Therapeutics Inc Métodos y sistemas para la producción de partículas de aav.
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
US20220290182A1 (en) 2019-08-09 2022-09-15 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2021041485A1 (en) 2019-08-26 2021-03-04 Voyager Therapeutics, Inc. Controlled expression of viral proteins
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
BR112022020753A2 (pt) 2020-04-15 2022-12-20 Voyager Therapeutics Inc Compostos de ligação a tau
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
AU2021273447B2 (en) 2020-05-13 2026-03-05 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
WO2022032153A1 (en) 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
KR20230085929A (ko) 2020-10-15 2023-06-14 에프. 호프만-라 로슈 아게 Va rna 전사를 위한 핵산 구조체
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
KR20230142712A (ko) * 2021-02-11 2023-10-11 론자 휴스턴, 아이엔씨. 아데노-연관된 바이러스(aav) 생산
EP4293118A4 (de) * 2021-02-12 2025-01-22 FUJIFILM Corporation Kit zur herstellung von adeno-assoziiertem virus und verwendung des kits
EP4301768A2 (de) 2021-03-03 2024-01-10 Voyager Therapeutics, Inc. Kontrollierte expression von viralen proteinen
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CA3216491A1 (en) 2021-04-16 2022-10-20 Asklepios Biopharmaceutical, Inc. Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
EP4396201A1 (de) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav-kapsid-zusammensetzungen und verfahren zur freisetzung
US20250122530A1 (en) 2021-09-03 2025-04-17 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
US20240376496A1 (en) 2021-09-03 2024-11-14 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240384242A1 (en) 2021-09-03 2024-11-21 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
EP4405396A2 (de) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von her2-positivem krebs
IL311861A (en) 2021-11-02 2024-06-01 Voyager Therapeutics Inc Aav capsid variants and uses thereof
US20250011372A1 (en) 2021-11-17 2025-01-09 Voyager Thrapeutics, Inc. Aav capsid variants and uses thereof
US20250059527A1 (en) 2021-12-16 2025-02-20 University Of Dundee Targeted degradation of alpha-synuclein
US20250346920A1 (en) 2022-01-25 2025-11-13 Voyager Therapeutics, Inc. Baculovirus expression system
AU2023217698A1 (en) 2022-02-08 2024-07-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
GB202201713D0 (en) 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
EP4532515A1 (de) 2022-06-02 2025-04-09 Voyager Therapeutics, Inc. Aav-kapsidvarianten und verwendungen davon
US20250215453A1 (en) 2022-06-28 2025-07-03 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
US20260085328A1 (en) 2022-09-08 2026-03-26 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN120019071A (zh) 2022-09-15 2025-05-16 沃雅戈治疗公司 Tau结合化合物
KR20250108100A (ko) 2022-09-22 2025-07-15 디나코 아게 Aav 유전자 치료법 벡터를 이용한 심근병증의 치료
TW202421788A (zh) 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用aav基因療法載體治療致心律不整性心肌病
AU2023417630A1 (en) 2022-12-29 2025-05-15 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
EP4705317A1 (de) 2023-05-03 2026-03-11 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von erkrankungen im zusammenhang mit cdkl5-mangel
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
EP4705488A1 (de) 2023-05-04 2026-03-11 Voyager Therapeutics, Inc. Aav-kapsidvarianten und verwendungen davon
WO2025038802A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025038430A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
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
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
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
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
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
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)
WO2026096603A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering syntaxin-binding protein-1
WO2026096600A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering cyclin-dependent kinase-like 5 protein
WO2026096599A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering syntaxin-binding protein-1
WO2026096611A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering cyclin-dependent kinase-like 5 protein

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
CA2221293C (en) * 1995-06-07 2012-09-18 University Of North Carolina At Chapel Hill Helper virus-free aav production
US6004797A (en) * 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
AU723497C (en) * 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy

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WO1999047691A1 (en) 1999-09-23
CA2324225A1 (en) 1999-09-23
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AU3097399A (en) 1999-10-11
DE69922934D1 (de) 2005-02-03
JP2002506652A (ja) 2002-03-05

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