CN116096431A - 肽修饰的aav衣壳 - Google Patents

肽修饰的aav衣壳 Download PDF

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Publication number
CN116096431A
CN116096431A CN202180055064.XA CN202180055064A CN116096431A CN 116096431 A CN116096431 A CN 116096431A CN 202180055064 A CN202180055064 A CN 202180055064A CN 116096431 A CN116096431 A CN 116096431A
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CN
China
Prior art keywords
aav
sequence
muscle
modified
aav9
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202180055064.XA
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English (en)
Chinese (zh)
Inventor
I·里夏尔
N·多明格斯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Evry Wald Esson University
Institut National de la Sante et de la Recherche Medicale INSERM
Genethon
Original Assignee
Evry Wald Esson University
Institut National de la Sante et de la Recherche Medicale INSERM
Genethon
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 Evry Wald Esson University, Institut National de la Sante et de la Recherche Medicale INSERM, Genethon filed Critical Evry Wald Esson University
Publication of CN116096431A publication Critical patent/CN116096431A/zh
Pending legal-status Critical Current

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    • 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
    • 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
    • C12N2750/14122—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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for viral vectors

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202180055064.XA 2020-09-10 2021-09-10 肽修饰的aav衣壳 Pending CN116096431A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20306005 2020-09-10
EP20306005.8 2020-09-10
PCT/EP2021/074964 WO2022053630A1 (en) 2020-09-10 2021-09-10 Peptide-modified aav capsid

Publications (1)

Publication Number Publication Date
CN116096431A true CN116096431A (zh) 2023-05-09

Family

ID=72717827

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180055064.XA Pending CN116096431A (zh) 2020-09-10 2021-09-10 肽修饰的aav衣壳

Country Status (11)

Country Link
US (1) US20240209030A1 (https=)
EP (2) EP4211231B1 (https=)
JP (1) JP7834725B2 (https=)
CN (1) CN116096431A (https=)
CA (1) CA3193128A1 (https=)
DK (1) DK4211231T3 (https=)
ES (1) ES2984259T3 (https=)
FI (1) FI4211231T3 (https=)
PL (1) PL4211231T3 (https=)
PT (1) PT4211231T (https=)
WO (1) WO2022053630A1 (https=)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL297391A (en) 2020-04-20 2022-12-01 Tenaya Therapeutics Inc An adeno-associated virus with an engineered capsid
EP4314295A1 (en) 2021-03-26 2024-02-07 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
CN117897492A (zh) 2021-08-04 2024-04-16 吉尼松公司 用于肌肉和cns中的基因表达的杂合启动子
US20250249052A1 (en) * 2022-04-07 2025-08-07 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
CN116925192B (zh) * 2022-04-12 2025-04-29 合肥星眸生物科技有限公司 一种融合型腺相关病毒及其应用
JP2025524363A (ja) 2022-06-09 2025-07-30 ジェネトン 糖原病ｉｉｉの処置のためのｎ末端短縮型ｇｄｅ
EP4707399A2 (en) 2022-06-10 2026-03-11 Généthon Peptide-modified aav capsid with enhanced muscle transduction efficiency
US20260053923A1 (en) 2022-10-07 2026-02-26 Genethon Immunotherapy of Skeletal Myopathies Using ANTI-FAP CAR-T Cells
EP4609882A1 (en) * 2022-10-12 2025-09-03 CPC Corporation Gene introduction method, gene therapy method, and tissue regeneration method
TW202502803A (zh) 2023-03-10 2025-01-16 美商戴諾治療公司 衣殼多肽及其使用方法
EP4677073A1 (en) 2023-03-10 2026-01-14 Genethon Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter
CN121420060A (zh) 2023-06-29 2026-01-27 吉尼松公司 用于治疗糖原贮积病iii的n-端截短的糖原脱支酶
WO2025048709A1 (en) * 2023-08-25 2025-03-06 Agency For Science, Technology And Research Composition and methods for identification of novel aav variants for differentiated gene delivery to cardiomyoctes and/or skeletal myofibers
WO2025133247A1 (en) 2023-12-21 2025-06-26 Genethon Methods for aav vector re-administration
KR102933125B1 (ko) * 2024-02-01 2026-03-04 주식회사 글루진테라퓨틱스 평활근 세포 특이적 유전자 전달이 가능한 아데노연관바이러스 벡터
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
WO2026017727A1 (en) 2024-07-16 2026-01-22 Genethon Alternative barcoding method to assess transduction efficiency of aav vectors
WO2026064428A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2026064444A1 (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 (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1222673A1 (zh) * 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
PE20190401A1 (es) * 2016-07-26 2019-03-13 Biomarin Pharm Inc Novedosas proteinas de la capside del virus adenoasociado
EP3775173A1 (en) 2018-04-05 2021-02-17 Genethon Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
AU2019258830B2 (en) 2018-04-27 2025-12-18 Universität Heidelberg Modified AAV capsid polypeptides for treatment of muscular diseases

Also Published As

Publication number Publication date
EP4211231B1 (en) 2024-06-26
JP2023544683A (ja) 2023-10-25
ES2984259T3 (es) 2024-10-29
DK4211231T3 (da) 2024-07-08
EP4421179A2 (en) 2024-08-28
US20240209030A1 (en) 2024-06-27
EP4211231A1 (en) 2023-07-19
FI4211231T3 (fi) 2024-07-17
WO2022053630A1 (en) 2022-03-17
EP4421179A3 (en) 2024-12-18
BR112023004176A2 (pt) 2023-04-11
JP7834725B2 (ja) 2026-03-24
PT4211231T (pt) 2024-07-24
PL4211231T3 (pl) 2024-11-04
CA3193128A1 (en) 2022-03-17

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