IL296544A - Plasmid system - Google Patents

Plasmid system

Info

Publication number
IL296544A
IL296544A IL296544A IL29654422A IL296544A IL 296544 A IL296544 A IL 296544A IL 296544 A IL296544 A IL 296544A IL 29654422 A IL29654422 A IL 29654422A IL 296544 A IL296544 A IL 296544A
Authority
IL
Israel
Prior art keywords
plasmid
nucleotides
rep
gene
helper
Prior art date
Application number
IL296544A
Other languages
English (en)
Hebrew (he)
Inventor
Horer Markus
Sonntag Florian
Kober Renee
Original Assignee
Freeline Therapeutics Ltd
Horer Markus
Sonntag Florian
Kober Renee
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
Priority claimed from GBGB1905263.8A external-priority patent/GB201905263D0/en
Priority claimed from EP19169121.1A external-priority patent/EP3722434B1/en
Application filed by Freeline Therapeutics Ltd, Horer Markus, Sonntag Florian, Kober Renee filed Critical Freeline Therapeutics Ltd
Publication of IL296544A publication Critical patent/IL296544A/en

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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (AREA)
IL296544A 2019-04-12 2020-04-14 Plasmid system IL296544A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
GBGB1905263.8A GB201905263D0 (en) 2019-04-12 2019-04-12 Plasmid system
EP19169121.1A EP3722434B1 (en) 2019-04-12 2019-04-12 Plasmid system
PCT/GB2020/050947 WO2020208379A1 (en) 2019-04-12 2020-04-14 Plasmid system

Publications (1)

Publication Number Publication Date
IL296544A true IL296544A (en) 2022-11-01

Family

ID=70295566

Family Applications (2)

Application Number Title Priority Date Filing Date
IL296544A IL296544A (en) 2019-04-12 2020-04-14 Plasmid system
IL287100A IL287100A (en) 2019-04-12 2021-10-07 Plasmid system

Family Applications After (1)

Application Number Title Priority Date Filing Date
IL287100A IL287100A (en) 2019-04-12 2021-10-07 Plasmid system

Country Status (11)

Country Link
US (1) US20220162642A1 (https=)
EP (1) EP3953482A1 (https=)
JP (1) JP7562558B2 (https=)
KR (1) KR20220019669A (https=)
CN (1) CN113891942A (https=)
AU (1) AU2020272557A1 (https=)
BR (1) BR112021020401A2 (https=)
CA (1) CA3136545A1 (https=)
IL (2) IL296544A (https=)
SG (1) SG11202111149VA (https=)
WO (1) WO2020208379A1 (https=)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW202545984A (zh) 2017-08-09 2025-12-01 美商生物化學醫療公司 核酸分子及其用途
WO2020033863A1 (en) 2018-08-09 2020-02-13 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof for non-viral gene therapy
EP3722434B1 (en) 2019-04-12 2022-07-27 Freeline Therapeutics Limited Plasmid system
EP4051704A2 (en) 2019-11-01 2022-09-07 Freeline Therapeutics Limited Factor viii construct
JP2024504365A (ja) * 2021-01-22 2024-01-31 バイオジェン・エムエイ・インコーポレイテッド 組換えウイルスベクター力価を増加させる微小管不安定化剤添加剤
AU2022227005B2 (en) * 2021-02-26 2025-09-11 Logicbio Therapeutics, Inc. Manufacturing and use of recombinant aav vectors
EP4410971A4 (en) * 2021-09-30 2025-06-04 FUJIFILM Corporation METHOD FOR PRODUCING AN ADENO-ASSOCIATED VIRUS, CELLS AND EXPRESSION VECTOR
GB202214328D0 (en) 2022-09-29 2022-11-16 Freeline Therapeutics Ltd Method
WO2024160232A1 (zh) * 2023-02-01 2024-08-08 纽福斯(苏州)生物科技有限公司 质粒包装系统及其应用
CN118633572A (zh) * 2023-03-06 2024-09-13 北京锦篮基因科技有限公司 神经元核内包涵体病小鼠模型构建方法及应用
WO2024223931A1 (en) * 2023-04-28 2024-10-31 AstraZeneca Ireland Limited Manufacturing and use of recombinant self-complementary aav vectors
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)
WO2025083617A1 (en) * 2023-10-18 2025-04-24 Nanocell Therapeutics Holdings B.V. Methods and compositions for nucleic construct delivery
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
TW202548023A (zh) 2024-03-14 2025-12-16 美商雅菲納治療公司 用於生產重組腺相關病毒的質體系統
EP4628576A1 (en) 2024-04-07 2025-10-08 Ascend Advanced Therapies GmbH Microrna molecules for use in methods for improving recombinant aav production
WO2025238067A1 (en) 2024-05-15 2025-11-20 Ascend Advanced Therapies Gmbh Novel vector system for aav production
WO2026064444A1 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10044384A1 (de) 2000-09-08 2002-04-18 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
EP1419245A4 (en) * 2001-07-13 2006-04-05 Univ Iowa Res Found ADENO-ASSOCIATED PSEUDOTYPE VIRUSES AND USES THEREOF
US20050080027A1 (en) 2001-11-30 2005-04-14 Markus Horer Optimized production of viral vectors derived from paroviruses in packaging and production cells by hsv infection or treatment with dna methylation inhibitors
ES2330826B1 (es) * 2008-06-04 2010-07-26 Proyecto De Biomedicina Cima, S.L. Sistema para empaquetamiento de adenovirus de alta capacidad.
PL2403867T3 (pl) * 2009-03-04 2019-12-31 Deutsches Krebsforschungszentrum Białko aktywujące składanie (aap) i jego zastosowanie do wytwarzania cząstek parwowirusa zasadniczo składających się z vp3
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
JP6348064B2 (ja) 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
CN108048483B (zh) * 2018-01-30 2021-02-02 中国疾病预防控制中心病毒病预防控制所 复制型重组腺病毒HAdV-5载体系统及其应用

Also Published As

Publication number Publication date
KR20220019669A (ko) 2022-02-17
US20220162642A1 (en) 2022-05-26
JP2022530192A (ja) 2022-06-28
BR112021020401A2 (pt) 2021-12-07
EP3953482A1 (en) 2022-02-16
IL287100A (en) 2021-12-01
CN113891942A (zh) 2022-01-04
SG11202111149VA (en) 2021-11-29
CA3136545A1 (en) 2020-10-15
JP7562558B2 (ja) 2024-10-07
WO2020208379A1 (en) 2020-10-15
AU2020272557A1 (en) 2021-12-09

Similar Documents

Publication Publication Date Title
US20220162642A1 (en) Plasmid system
EP3722434B1 (en) Plasmid system
US20230323387A1 (en) Plasmid system
CA3153133A1 (en) Adeno-associated virus (aav) systems for treatment of genetic hearing loss
JP7530431B2 (ja) Ｄｎａ増幅方法
US20250263746A1 (en) Method for producing recombinant aav particles
US20260117255A1 (en) Plasmid system
US20260022401A1 (en) Novel AAV rep ORFs and Rep polypeptides
EP4628576A1 (en) Microrna molecules for use in methods for improving recombinant aav production
WO2025238067A1 (en) Novel vector system for aav production
AU2024239386A1 (en) Method for the production of recombinant aav particle preparations
WO2025168663A1 (en) Method for producing recombinant adeno-associated viral particles
HK40061787A (en) Dna amplification method
WO2026068629A1 (en) Methods and constructs for raav production
HK40117756A (zh) 用於生产重组aav颗粒的方法
CN119569833A (zh) 靶向骨骼肌的aav衣壳蛋白变体及其用途
HK40129257A (zh) 用於产生重组aav颗粒制备物的方法