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
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
Original Assignee
Univ Pennsylvania
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Application granted granted Critical
Publication of ATE286138T1 publication Critical patent/ATE286138T1/de

Links

Classifications

    • 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

Landscapes

  • 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

ID=22146942

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 (126)

* 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
US20010034062A1 (en) * 2000-02-09 2001-10-25 Scott Koenig Antibody gene therapy with adeno-associated viral vectors
AU2001267058A1 (en) * 2000-06-09 2001-12-17 University Of Florida Research Foundation, Inc. Aav vector-based compositions and therapeutic uses thereof
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
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
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.
MXPA04004876A (es) 2001-11-21 2004-07-30 Univ Pennsylvania Secuencias de acido nucleico y de aminoacido de adenovirus de simio, vectores que contienen los mismos y metodos de uso.
EP1463805B1 (de) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Sequenzen des adeno-assoziierten virus (aav) serotyp 9, diese enthaltende vektoren und verwendungen dafür
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
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
KR101662574B1 (ko) 2007-11-28 2016-10-05 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 유인원 ｅ 아데노바이러스 ｓａｄｖ-39, -25.2, -26, -30, -37, 및 -38
CN102131920B (zh) 2007-11-28 2013-11-06 宾夕法尼亚大学托管会 猿猴亚家族C腺病毒SAdV-40、-31和-34及其应用
EP2325298B1 (de) 2008-03-04 2016-10-05 The Trustees Of The University Of Pennsylvania AFFENADENOVIREN SAdV-36, -42.1, -42.2, AND -44 UND DEREN NUTZUNG
WO2010045659A1 (en) 2008-10-17 2010-04-22 American Gene Technologies International Inc. Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
EP2774985B1 (de) 2008-10-31 2016-12-14 The Trustees Of The University Of Pennsylvania Simian-Adenovirus SAdV-43 und seine Verwendungen
SI3988660T1 (sl) 2009-05-02 2025-09-30 Genzyme Corporation Genska terapija za nevrodegenerativne motnje
US8846031B2 (en) 2009-05-29 2014-09-30 The Trustees Of The University Of Pennsylvania Simian adenovirus 41 and uses thereof
EP2643465B1 (de) 2010-11-23 2016-05-11 The Trustees Of The University Of Pennsylvania Affen-adenovirus a1321 der subfamilie e und verwendungen davon
AU2013262626B2 (en) 2012-05-18 2018-11-29 The Trustees Of The University Of Pennsylvania Subfamily E simian adenoviruses A1302, A1320, A1331 and A1337 and uses thereof
BR112016020688B1 (pt) 2014-03-09 2024-01-30 The Trustees Of The University Of Pennsylvania Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos
WO2016200997A1 (en) * 2015-06-10 2016-12-15 America Gene Technologies International, Inc. Non-integrating viral delivery system and methods of use thereof
US11980663B2 (en) 2015-07-08 2024-05-14 American Gene Technologies International Inc. HIV pre-immunization and immunotherapy
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
IL297238B2 (en) 2016-01-15 2024-08-01 American Gene Tech Int Inc Methods and preparations for gamma-delta T-cell activation
US10137144B2 (en) 2016-01-15 2018-11-27 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
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
WO2017213697A1 (en) * 2016-06-08 2017-12-14 American Gene Technologies International Inc. Non-integrating viral delivery system and methods related thereto
CA3028982A1 (en) 2016-07-08 2018-01-11 American Gene Technologies International Inc. Hiv pre-immunization and immunotherapy
WO2018017882A1 (en) 2016-07-21 2018-01-25 American Gene Technologies International Inc. Viral vectors for treating parkinson's disease
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
US12559769B2 (en) 2017-06-16 2026-02-24 American Gene Technologies International Inc. Methods and compositions for the activation of tumor cytotoxicity via human gamma-delta t-cells
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
EP3762500A1 (de) 2018-03-06 2021-01-13 Voyager Therapeutics, Inc. Aus insektenzelle hergestellte teilweise selbstkomplementäre aav-genome
CA3097192A1 (en) 2018-04-27 2019-10-31 Voyager Therapeutics, Inc. Methods for measuring the potency of aadc viral vectors
WO2019217483A1 (en) * 2018-05-07 2019-11-14 Spark Therapeutics, Inc. Plasmid free aav vector producing cell lines
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2019222444A2 (en) 2018-05-16 2019-11-21 Voyager Therapeutics, Inc. Directed evolution
US20210207167A1 (en) 2018-05-16 2021-07-08 Voyager Therapeutics, Inc. Aav serotypes for brain specific payload delivery
WO2019241486A1 (en) 2018-06-13 2019-12-19 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
US20210254103A1 (en) 2018-07-02 2021-08-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
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
WO2020072849A1 (en) 2018-10-04 2020-04-09 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
CA3125770A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
US20220243225A1 (en) 2019-04-29 2022-08-04 Voyager Therapeutics, Inc. SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS
EP3966227A1 (de) 2019-05-07 2022-03-16 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur vektorisierten vermehrung von proteinzerstörung, -expression und/oder -regulierung
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
CA3180222A1 (en) 2020-04-15 2021-10-21 Voyager Therapeutics, Inc. Tau binding compounds
GB202005732D0 (en) 2020-04-20 2020-06-03 Synpromics Ltd Regulatory nucleic acid sequences
US12630842B2 (en) 2020-05-13 2026-05-19 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
WO2022079083A1 (en) 2020-10-15 2022-04-21 F. Hoffmann-La Roche Ag Nucleic acid constructs for va rna transcription
KR20230078805A (ko) 2020-11-02 2023-06-02 바이오마린 파머수티컬 인크. 아데노-연관 바이러스의 농축 공정
EP4291665A1 (de) * 2021-02-11 2023-12-20 Lonza Houston Inc. Herstellung von adeno-assoziiertem virus (aav)
JPWO2022172986A1 (de) * 2021-02-12 2022-08-18
WO2022187473A2 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
WO2022187548A1 (en) 2021-03-03 2022-09-09 Voyager Therapeutics, Inc. Controlled expression of viral proteins
JP2024515626A (ja) 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドａａｖビリオン
US20240384242A1 (en) 2021-09-03 2024-11-21 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
JP2024534894A (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
WO2023034989A1 (en) 2021-09-03 2023-03-09 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
EP4396201A1 (de) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav-kapsid-zusammensetzungen und verfahren zur freisetzung
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin 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
EP4426716A1 (de) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav-kapsidvarianten und verwendungen davon
EP4433169A1 (de) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Aav-kapsidvarianten und verwendungen davon
CA3240342A1 (en) 2021-12-16 2023-06-22 Gopal SAPKOTA Targeted degradation of alpha-synuclein
US20250346920A1 (en) 2022-01-25 2025-11-13 Voyager Therapeutics, Inc. Baculovirus expression system
JP2025507338A (ja) 2022-02-08 2025-03-18 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
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
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
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
JP2025535203A (ja) 2022-09-15 2025-10-23 ボイジャー セラピューティクス インコーポレイテッド タウ結合化合物
UY40442A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav
UY40443A (es) 2022-09-22 2024-02-15 Biomarin Pharm Inc Tratamiento de la miocardiopatía con vectores de genoterapia con aav
WO2024145474A2 (en) 2022-12-29 2024-07-04 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
JP2026515815A (ja) 2023-05-03 2026-05-19 ボイジャー セラピューティクス インコーポレイテッド Ｃｄｋｌ５欠損に関連する障害の治療のための組成物及び方法
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
IL324036A (en) 2023-05-03 2025-12-01 Voyager Therapeutics Inc Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
WO2024229173A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
JP2026515970A (ja) 2023-05-04 2026-05-19 ボイジャー セラピューティクス インコーポレイテッド Ａａｖカプシドバリアント及びその使用
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
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025038800A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin 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
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
WO2025122548A1 (en) 2023-12-05 2025-06-12 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
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
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
AR134555A1 (es) 2023-12-05 2026-01-28 Voyager Therapeutics Inc Composiciones y métodos para el tratamiento de trastornos relacionados con la deficiencia de frataxina
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
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
WO2026096599A1 (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
WO2026096611A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering cyclin-dependent kinase-like 5 protein
WO2026102000A1 (en) 2024-11-06 2026-05-15 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
NL2039429B1 (en) 2024-12-20 2026-07-13 Academisch Ziekenhuis Leiden Pabpn1 isoforms for use in therapy

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
JP4108746B2 (ja) * 1995-06-07 2008-06-25 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル ヘルパーウイルスを含まないａａｖ産生
US6004797A (en) * 1995-11-09 1999-12-21 Avigen, Inc. Adenovirus helper-free recombinant AAV Virion production
EP1696036B1 (de) * 1996-09-06 2010-04-21 The Trustees of The University of Pennsylvania Rekombinante AAV zur Herstellung eines Medikaments für die Gentherapie von Muskelzellen

Also Published As

Publication number Publication date
EP1064393B1 (de) 2004-12-29
PT1064393E (pt) 2005-04-29
DE69922934D1 (de) 2005-02-03
DE69922934T2 (de) 2005-12-08
WO1999047691A1 (en) 1999-09-23
ES2235470T3 (es) 2005-07-01
EP1064393A1 (de) 2001-01-03
AU3097399A (en) 1999-10-11
CA2324225A1 (en) 1999-09-23
JP2002506652A (ja) 2002-03-05

Similar Documents

Publication Publication Date Title
DE69922934D1 (de) Zusammensetzungen und verfahren zur helfer-freien herstellung von rekombinante adeno-assoziierten viren
ATE349542T1 (de) Zusammensetzungen und verfahren zur herstellung von helfer-freien rekombinanten adeno- assoziierten viren
Robert et al. Manufacturing of recombinant adeno‐associated viruses using mammalian expression platforms
Chejanovsky et al. Mutagenesis of an AUG codon in the adeno-associated virus rep gene: effects on viral DNA replication
DE69631480D1 (de) Helfersystem zur wirkungssteigerung der aav-vektorproduktion
CA2303768A1 (en) Methods and vector constructs useful for production of recombinant aav
CA2349838A1 (en) Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
JP2005525086A5 (de)
GB2595404A (en) Inducible AAV system comprising cumate operator sequences
DE69737107D1 (de) Ein verbessertes verfahren zur produktion und reinigung von adenoviralen vektoren
NZ600121A (en) A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
US20220090130A1 (en) MODIFIED BACULOVIRUS SYSTEM FOR IMPROVED PRODUCTION OF CLOSED-ENDED DNA (ceDNA)
KR960010864A (ko) 동물 세포의 형질 감염을 위한 재조합 dna 바이러스 벡터
DE69941823D1 (de) Eine verbesserte methode zur herstellung und reinigung von adenoviralen vektoren
EP4317424A1 (de) Vektor, verfahren zur herstellung von linearer kovalenter bindungsverschlossener dna damit, parvovirusvektorherstellungsverfahren und parvovirusvektorproduzierende zelle
JP2020519294A5 (de)
US20220242917A1 (en) Compositions and methods for producing adeno-associated viral vectors
Cao et al. Recombinant adeno‐associated virus production evaluation in Chinese hamster ovary cells
WO2026030520A1 (en) Engineered producer cell and methods of producing and using the same
US7148341B2 (en) AAV DNA comprising helper virus sequences
JP2023546116A5 (de)
WO2024163979A2 (en) Engineered aav polypeptides
US20210108227A1 (en) Parvovirus Vector Production
RU2846879C1 (ru) МОДИФИЦИРОВАННАЯ БАКУЛОВИРУСНАЯ СИСТЕМА ДЛЯ УЛУЧШЕННОГО ПОЛУЧЕНИЯ ДНК С ЗАМКНУТЫМИ КОНЦАМИ (ceDNA)
EP4671264A1 (de) Aav plasmide die eine zusätzliche protein kodierende sequenz enthalten

Legal Events

Date Code Title Description
UEP Publication of translation of european patent specification

Ref document number: 1064393

Country of ref document: EP

REN Ceased due to non-payment of the annual fee