ECSP17074016A - Producción de vectores adeno-asociados de gran tamaño - Google Patents

Producción de vectores adeno-asociados de gran tamaño

Info

Publication number
ECSP17074016A
ECSP17074016A ECIEPI201774016A ECPI201774016A ECSP17074016A EC SP17074016 A ECSP17074016 A EC SP17074016A EC IEPI201774016 A ECIEPI201774016 A EC IEPI201774016A EC PI201774016 A ECPI201774016 A EC PI201774016A EC SP17074016 A ECSP17074016 A EC SP17074016A
Authority
EC
Ecuador
Prior art keywords
aav
production
adeno
associated vectors
genomes
Prior art date
Application number
ECIEPI201774016A
Other languages
English (en)
Inventor
David Souza
Karen Vincent
Sirkka Kyostio-Moore
Original Assignee
Genzyme Corp
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=56943906&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ECSP17074016(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of ECSP17074016A publication Critical patent/ECSP17074016A/es

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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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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
    • C12N2750/14152—Methods 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)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Epidemiology (AREA)
  • Pulmonology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Se proporcionan en el presente documento métodos para producir una partícula de virus adenoasociado (AAV) que contiene un genoma de AAV recombinante de gran tamaño (por ejemplo, mayor de 4,7 kb). En algunos aspectos, la invención proporciona partículas AAV y vectores de AAV que comprenden genomas de rAAV de gran tamaño. También se proporcionan líneas celulares productoras para producir partículas AAV que comprenden genomas de gran tamaño.
ECIEPI201774016A 2015-04-08 2017-11-08 Producción de vectores adeno-asociados de gran tamaño ECSP17074016A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562144862P 2015-04-08 2015-04-08
US201562220067P 2015-09-17 2015-09-17

Publications (1)

Publication Number Publication Date
ECSP17074016A true ECSP17074016A (es) 2018-02-28

Family

ID=56943906

Family Applications (1)

Application Number Title Priority Date Filing Date
ECIEPI201774016A ECSP17074016A (es) 2015-04-08 2017-11-08 Producción de vectores adeno-asociados de gran tamaño

Country Status (25)

Country Link
US (1) US10815497B2 (es)
EP (1) EP3280799A2 (es)
JP (3) JP6878299B2 (es)
KR (1) KR20170133500A (es)
CN (1) CN107864657A (es)
AU (3) AU2016245806A1 (es)
BR (1) BR112017021505A2 (es)
CA (1) CA2982123A1 (es)
CL (1) CL2017002537A1 (es)
CO (1) CO2017011344A2 (es)
CR (1) CR20170505A (es)
EA (1) EA201792236A1 (es)
EC (1) ECSP17074016A (es)
GT (1) GT201700214A (es)
HK (1) HK1246339A1 (es)
IL (3) IL294965A (es)
MX (1) MX2017012935A (es)
NZ (1) NZ737009A (es)
PE (1) PE20171800A1 (es)
PH (1) PH12017501836A1 (es)
SG (3) SG10201912912QA (es)
TN (1) TN2017000431A1 (es)
TW (1) TWI707951B (es)
UY (1) UY36611A (es)
WO (1) WO2016164609A2 (es)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HRP20231183T1 (hr) 2013-02-15 2024-01-05 Bioverativ Therapeutics Inc. Optimizirani gen faktora viii
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2016073693A2 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
BR112018006074A2 (pt) 2015-09-24 2018-10-09 Biomarin Pharm Inc vetores de fator viii de vírus adeno-associado, partículas virais associadas e formulações terapêuticas compreendendo as mesmas
HRP20221089T1 (hr) 2016-02-01 2022-11-25 Bioverativ Therapeutics Inc. Optimizirani geni faktora viii
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
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
EP3458589A4 (en) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING HUNTINGTON'S DISEASE
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
US11518991B2 (en) 2017-02-24 2022-12-06 Georg-August-Universitaet Goettingen Stiftung Oeffentlichen Rechts, Universitaetsmedizin Method for re-expression of different hypermethylated genes involved in fibrosis, like hypermethylated RASAL1 and use thereof in treatment of fibrosis as well as kit of parts for re-expression of hypermethylated genes including RASAL1 in a subject
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
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
MA49153A (fr) * 2017-05-19 2021-03-24 Encoded Therapeutics Inc Éléments régulateurs à haute activité
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2020019002A1 (en) * 2018-07-20 2020-01-23 University Of Utah Research Foundation Gene therapy for macular degeneration
EP3863662A1 (en) * 2018-10-12 2021-08-18 Genzyme Corporation Generation of improved human pah for treatment of severe pku by liver-directed gene replacement therapy
EP4022097A4 (en) * 2019-08-29 2022-12-14 Siemens Healthcare Diagnostics Inc. REAGENTS AND METHODS FOR DETECTING ADENO-ASSOCIATED VIRUS SHEDDING
EP4038182A1 (en) 2019-09-30 2022-08-10 Bioverativ Therapeutics Inc. Lentiviral vector formulations
CN111218446B (zh) * 2019-12-25 2023-03-28 劲帆生物医药科技（武汉）有限公司 一种肝脏特异性启动子及其应用
CN115552037A (zh) * 2020-03-16 2022-12-30 北卡罗来纳大学教堂山分校 用于选择性检测肿瘤源性病毒dna的组合物和方法
CA3183557A1 (en) 2020-06-24 2021-12-30 Bioverativ Therapeutics Inc. Methods for the removal of free factor viii from preparations of lentiviral vectors modified to express said protein
CA3189673A1 (en) 2020-08-23 2022-03-03 Ajay MAGHODIA Modified baculovirus system for improved production of closed-ended dna (cedna)
CA3229323A1 (en) 2021-08-23 2023-03-02 Ajay MAGHODIA Optimized factor viii genes
US20230091932A1 (en) 2021-08-23 2023-03-23 Bioverativ Therapeutics Inc. Closed-end dna production with inverted terminal repeat sequences
WO2023028440A2 (en) 2021-08-23 2023-03-02 Bioverativ Therapeutics Inc. Baculovirus expression system
AU2022355088A1 (en) 2021-09-30 2024-05-16 Bioverativ Therapeutics Inc. Nucleic acids encoding factor viii polypeptides with reduced immunogenicity

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP2369002A1 (en) 1999-08-09 2011-09-28 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
WO2001027303A1 (en) * 1999-10-12 2001-04-19 The University Of North Carolina At Chapel Hill Adeno-associated virus vectors encoding factor viii and methods of using the same
AU2001269723B9 (en) 2000-06-01 2006-11-16 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
CA2406745C (en) 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
US20040092008A1 (en) * 2002-11-12 2004-05-13 Snyder Richard O. Recombinant AAV vectors for gene therapy of hemophilia A
WO2004075861A2 (en) 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
DK2277996T3 (da) 2003-05-21 2014-10-20 Genzyme Corp Fremgangsmåder til fremstilling af præparationer af rekombinante aav-virioner, der i hovedsagen er fri for tomme capsider
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
CA3077531C (en) 2009-06-16 2022-09-20 Genzyme Corporation Improved methods for purification of recombinant aav vectors
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
WO2012112832A1 (en) 2011-02-17 2012-08-23 The Trustees Of The University Of Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
CA2888931C (en) * 2012-10-26 2023-09-05 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
CN112538501A (zh) * 2013-09-12 2021-03-23 生物马林药物股份有限公司 腺相关病毒因子viii载体
CN114231563A (zh) 2014-05-02 2022-03-25 建新公司 用于视网膜及cns基因疗法的aav载体
MX2017010370A (es) 2015-02-10 2017-12-14 Genzyme Corp Suministro mejorado de particulas virales al cuerpo estriado y al cortex.

Also Published As

Publication number Publication date
TW201710498A (zh) 2017-03-16
GT201700214A (es) 2018-10-24
JP2018510648A (ja) 2018-04-19
TWI707951B (zh) 2020-10-21
CN107864657A (zh) 2018-03-30
US20190048362A1 (en) 2019-02-14
EA201792236A1 (ru) 2018-03-30
AU2024201619A1 (en) 2024-03-28
TN2017000431A1 (en) 2019-04-12
IL294965A (en) 2022-09-01
JP6878299B2 (ja) 2021-05-26
AU2016245806A1 (en) 2017-11-30
SG11201708203UA (en) 2017-11-29
HK1246339A1 (zh) 2018-09-07
BR112017021505A2 (pt) 2018-07-03
CR20170505A (es) 2018-01-26
EP3280799A2 (en) 2018-02-14
AU2022203942A1 (en) 2022-06-23
NZ737009A (en) 2024-01-26
SG10201909381WA (en) 2019-11-28
MX2017012935A (es) 2018-02-12
IL283291A (en) 2021-07-29
CL2017002537A1 (es) 2018-05-11
KR20170133500A (ko) 2017-12-05
WO2016164609A2 (en) 2016-10-13
PH12017501836A1 (en) 2018-04-23
WO2016164609A3 (en) 2016-12-15
UY36611A (es) 2016-11-30
SG10201912912QA (en) 2020-02-27
IL254933B (en) 2021-06-30
JP2021118724A (ja) 2021-08-12
AU2022203942B2 (en) 2023-12-14
JP2023116678A (ja) 2023-08-22
IL254933A0 (en) 2017-12-31
CA2982123A1 (en) 2016-10-13
PE20171800A1 (es) 2017-12-28
IL283291B (en) 2022-08-01
US10815497B2 (en) 2020-10-27
CO2017011344A2 (es) 2018-03-28

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