ATE310829T1 - Zelllinien und konstrukte für die herstellung von e1-deletierten adenoviren in abwesenheit von replikationskompetentem adenovirus - Google Patents

Zelllinien und konstrukte für die herstellung von e1-deletierten adenoviren in abwesenheit von replikationskompetentem adenovirus

Info

Publication number
ATE310829T1
ATE310829T1 AT00974097T AT00974097T ATE310829T1 AT E310829 T1 ATE310829 T1 AT E310829T1 AT 00974097 T AT00974097 T AT 00974097T AT 00974097 T AT00974097 T AT 00974097T AT E310829 T1 ATE310829 T1 AT E310829T1
Authority
AT
Austria
Prior art keywords
cell lines
absence
constructs
producing
competent adenovirus
Prior art date
Application number
AT00974097T
Other languages
English (en)
Inventor
Guangping 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 ATE310829T1 publication Critical patent/ATE310829T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10311Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10351Methods of production or purification of viral material
    • C12N2710/10352Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AT00974097T 1999-09-29 2000-09-12 Zelllinien und konstrukte für die herstellung von e1-deletierten adenoviren in abwesenheit von replikationskompetentem adenovirus ATE310829T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US15664499P 1999-09-29 1999-09-29
PCT/US2000/040872 WO2001023597A2 (en) 1999-09-29 2000-09-12 Cell lines and constructs useful in production of e1-deleted adenoviruses in absence of replication competent adenovirus

Publications (1)

Publication Number Publication Date
ATE310829T1 true ATE310829T1 (de) 2005-12-15

Family

ID=22560427

Family Applications (1)

Application Number Title Priority Date Filing Date
AT00974097T ATE310829T1 (de) 1999-09-29 2000-09-12 Zelllinien und konstrukte für die herstellung von e1-deletierten adenoviren in abwesenheit von replikationskompetentem adenovirus

Country Status (11)

Country Link
US (3) US6365394B1 (de)
EP (1) EP1226264B9 (de)
JP (1) JP2003510089A (de)
AT (1) ATE310829T1 (de)
AU (1) AU779198B2 (de)
CA (1) CA2384382A1 (de)
CY (1) CY1105440T1 (de)
DE (1) DE60024302T2 (de)
DK (1) DK1226264T3 (de)
ES (1) ES2253270T3 (de)
WO (1) WO2001023597A2 (de)

Families Citing this family (136)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6989264B2 (en) * 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released 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
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
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
GB9924981D0 (en) * 1999-10-21 1999-12-22 Univ Manchester Gene therapy
CN1293196C (zh) 2000-05-26 2007-01-03 大日本住友制药株式会社 具有副作用降低的特性的新型重组腺病毒载体
US20030092161A1 (en) * 2001-09-19 2003-05-15 The Trustees Of The University Of Pennsylvania Compositions and methods for production of recombinant viruses, and uses therefor
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
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
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
JP4495588B2 (ja) * 2002-04-25 2010-07-07 クルセル ホランド ベー ヴェー 安定なアデノウイルスベクターおよびその増殖方法
US7071172B2 (en) * 2002-04-30 2006-07-04 The University Of North Carolina At Chapel Hill Secretion signal vectors
CA2551026A1 (en) * 2003-12-23 2005-07-14 Schering Corporation Methods for producing a549 cell lines stable in serum-free medium suspension culture
US8420611B2 (en) * 2004-08-12 2013-04-16 Cedars-Sinai Medical Center Combined gene therapy for the treatment of macroscopic gliomas
US20060074083A1 (en) * 2004-10-05 2006-04-06 Merz Pharma Gmbh & Co. Kgaa Cyclic and acyclic propenones for treating CNS disorders
US8223817B2 (en) * 2005-12-29 2012-07-17 Telefonaktiebolaget Lm Ericsson (Publ) Method and arrangement for frequency hopping in wireless communication systems with carriers of varying bandwidth
WO2008095027A2 (en) * 2007-01-30 2008-08-07 Cedars-Sinai Medical Center Adenoviral vector comprising herpes simplex virus type 1 thymidine kinase and a transgene for increasing the expression of the transgene
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP3567106A1 (de) 2010-04-23 2019-11-13 University of Massachusetts Auf das zns abzielende aav-vektoren und verfahren zur verwendung davon
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
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
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
CN107106689A (zh) 2014-11-05 2017-08-29 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
CN112410338A (zh) 2014-11-14 2021-02-26 沃雅戈治疗公司 调节性多核苷酸
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EP3368054A4 (de) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. Regulierbare expression unter verwendung des adeno-assoziierten virus (aav)
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
KR20220108216A (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
SG11201809699XA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Modulatory polynucleotides
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
EP3613849A4 (de) 2017-04-21 2021-01-06 Geneuin-Tech Co., Ltd. Zelllinie zur herstellung von nichtreplizierendem adenovirus und herstellungsverfahren dafür
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP2020518259A (ja) 2017-05-05 2020-06-25 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. ハンチントン病治療組成物および方法
TW201907937A (zh) 2017-05-08 2019-03-01 美商葛利史東腫瘤科技公司 阿爾法病毒新抗原載體
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
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
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (de) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Behandlung von amyotropher lateralsklerose (als)
JP7653013B2 (ja) 2018-01-04 2025-03-28 アイコニック セラピューティクス リミテッド ライアビリティ カンパニー 抗組織因子抗体、抗体薬物コンジュゲート、及び関連する方法
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
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
EP3793615A2 (de) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Gerichtete entwicklung von aav zur verbesserung des tropismus für zns
EP3793686A1 (de) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Aav-serotypen zur gehirnspezifischen payload-übertragung
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
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
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
CN109536437B (zh) * 2018-12-24 2021-06-11 内蒙古必威安泰生物科技有限公司 一种维持稳定性、高滴度病毒抗原生产的悬浮细胞病毒的培养方法
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
MX2021014525A (es) 2019-05-30 2022-03-17 Gritstone Bio Inc Adenovirus modificados.
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
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
EP4192496A4 (de) 2020-08-06 2025-01-01 Gritstone bio, Inc. Multiepitop-impfstoffkassetten
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
US20230407328A1 (en) 2020-11-02 2023-12-21 Biomarin Pharmaceutical Inc. Process for enriching adeno-associated virus
EP4284335A1 (de) 2021-02-01 2023-12-06 RegenxBio Inc. Gentherapie für neuronale ceroid-lipofuscinosen
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4301768A2 (de) 2021-03-03 2024-01-10 Voyager Therapeutics, Inc. Kontrollierte expression von viralen proteinen
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
US20240376496A1 (en) 2021-09-03 2024-11-14 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
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
US20240384242A1 (en) 2021-09-03 2024-11-21 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
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
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
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
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
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
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202449167A (zh) 2023-04-26 2024-12-16 美商航海家醫療公司 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法
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
AU2024265526A1 (en) 2023-05-03 2025-12-11 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
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
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
WO2025038430A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
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
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
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
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
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)
WO2026096599A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering syntaxin-binding protein-1
WO2026096603A1 (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
WO2026096600A1 (en) 2024-10-30 2026-05-07 Neurocrine Biosciences, Inc. Compositions and methods for delivering cyclin-dependent kinase-like 5 protein

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR100470180B1 (ko) 1995-06-15 2005-02-04 크루셀 홀란드 비.브이. 유전자 요법에 사용할 수 있는 인간의 재조합아데노바이러스 패키징 시스템
US5891690A (en) 1996-04-26 1999-04-06 Massie; Bernard Adenovirus E1-complementing cell lines
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

Also Published As

Publication number Publication date
WO2001023597A2 (en) 2001-04-05
DE60024302T2 (de) 2006-08-17
CA2384382A1 (en) 2001-04-05
AU779198B2 (en) 2005-01-13
EP1226264B9 (de) 2017-04-12
DK1226264T3 (da) 2006-04-03
US20060003451A1 (en) 2006-01-05
ES2253270T3 (es) 2006-06-01
WO2001023597A9 (en) 2001-11-22
WO2001023597A3 (en) 2001-10-25
EP1226264B1 (de) 2005-11-23
US6365394B1 (en) 2002-04-02
DE60024302D1 (de) 2005-12-29
US20020090717A1 (en) 2002-07-11
AU1251801A (en) 2001-04-30
JP2003510089A (ja) 2003-03-18
CY1105440T1 (el) 2010-04-28
EP1226264A2 (de) 2002-07-31

Similar Documents

Publication Publication Date Title
DE60024302D1 (de) Zelllinien und konstrukte für die herstellung von e1-deletierten adenoviren in abwesenheit von replikationskompetentem adenovirus
DE69435108D1 (de) Defekte adenovirus-vektoren und deren verwendung in der gentherapie
ATE325885T1 (de) Methode für die produktion von adenoviralen vektoren, die nicht vom serotyp c abgeleitet sind
CA2391591A1 (en) Permanent amniocyte cell line, the production thereof and its use for producing gene transfer vectors
WO1998013499A3 (en) Packaging cell lines for use in facilitating the development of high-capacity adenoviral vectors
NO20000265D0 (no) Kontrollert cytolyse av målceller, midler og sammensetninger som forårsaker cytolyse og forbindelser som kan anvendes for å fremstille midlene
ATE317011T1 (de) Expressionsvektoren und anwendungsmethoden
WO1998027204A3 (en) AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
NO992704D0 (no) Serum-fritt celledyrkningsmedium
ATE397075T1 (de) Chimärisches , adenovirales faserprotein und verfahren zu dessen verwendung
NZ334034A (en) Use of a melanoma differentiation associated gene (mda-7) for reversing a cancerous phenotype
ATE286139T1 (de) Replikationskompetente anti-tumor vektoren
NO910982D0 (no) Uratoksydase-aktivt protein, rekombinant gen som koder for det, ekspresjonsvektor, mikroorganismer og transformerteceller.
ES2194237T3 (es) Preparaciones cosmeticas.
TR199901386T2 (en) �midazopiridazinler.
EP1144666A3 (de) Replikation -defektive baculoviren expressionssystem
ES2184059T3 (es) Vector retrovirico y su utilizacion en terapia genica.
FR2799472B1 (fr) Preparation d'adenovirus recombinants et de banques adenovirales
HUP0102413A1 (hu) Vektorok nukleinsavak bevitelére, ezeket tartalmazó készítmények és alkalmazásuk
AU2350997A (en) Novel adenoviral vector for transferring human genes in vivo
EP0843006A3 (de) Rekombinante prostataspezifische Antigen-Vektoren und diese verwendende Assays
AU2000265672A1 (en) Somatic cloning gene transfer for the production of recombinant proteins, cells and organs
Berger Damn the facts--roll the presses.
ITBO960469A0 (it) Metodo per la produzione di formaggi, in particolare del tipo di mozzarelle e simili
Holden German science adviser gets the boot.

Legal Events

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

Ref document number: 1226264

Country of ref document: EP

REN Ceased due to non-payment of the annual fee