CN116096431A - 肽修饰的aav衣壳 - Google Patents

肽修饰的aav衣壳 Download PDF

Info

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
Authority
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
Other languages
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

Links

Images

Classifications

    • 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

Landscapes

  • 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 (enExample)
EP (2) EP4421179A3 (enExample)
JP (1) JP7834725B2 (enExample)
CN (1) CN116096431A (enExample)
CA (1) CA3193128A1 (enExample)
DK (1) DK4211231T3 (enExample)
ES (1) ES2984259T3 (enExample)
FI (1) FI4211231T3 (enExample)
PL (1) PL4211231T3 (enExample)
PT (1) PT4211231T (enExample)
WO (1) WO2022053630A1 (enExample)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12552838B2 (en) 2020-04-20 2026-02-17 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
US20240165271A1 (en) 2021-03-26 2024-05-23 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
EP4381077A1 (en) 2021-08-04 2024-06-12 Genethon Hybrid promoters for gene expression in muscles and in the cns
EP4504952A4 (en) * 2022-04-07 2026-04-08 Kate Therapeutics Inc COMPOSITIONS AND METHODS FOR MUSCULAR 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
WO2023237731A1 (en) 2022-06-09 2023-12-14 Genethon N-terminal truncated gde for the treatment of glycogen storage disease iii
ES3057171T3 (en) 2022-06-10 2026-02-26 Genethon Peptide-modified aav capsid with enhanced muscle transduction efficiency
CN120112308A (zh) 2022-10-07 2025-06-06 吉尼松公司 使用抗fap car-t细胞免疫治疗骨骼肌病
EP4609882A1 (en) * 2022-10-12 2025-09-03 CPC Corporation Gene introduction method, gene therapy method, and tissue regeneration method
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
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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
WO2025165162A1 (ko) * 2024-02-01 2025-08-07 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
CN119751599B (zh) * 2024-07-16 2025-08-19 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2026064442A2 (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
WO2026064444A1 (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
IL305149A (en) * 2016-07-26 2023-10-01 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
MA51353B1 (fr) 2018-04-05 2022-09-30 Univ Sorbonne Sérotype du virus adéno-associé recombinant hybride entre aav9 et aavrh74 possédant un tropisme hépatique réduit
US20210363193A1 (en) * 2018-04-27 2021-11-25 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases

Also Published As

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

Similar Documents

Publication Publication Date Title
EP4211231B1 (en) Peptide-modified aav capsid
CN112004925B (zh) 具有降低的肝向性的AAV9和AAVrh74的杂合重组腺相关病毒血清型
JP6495273B2 (ja) 変異ａａｖ、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法
US20230242905A1 (en) Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping
WO2021219762A1 (en) Use of a synthetic aav capsid for gene therapy of muscle and central nervous system disorders
EP4536686B1 (en) Peptide-modified aav capsid with enhanced muscle transduction efficiency
WO2020208032A1 (en) Hybrid promoters for muscle expression
BR112021001498A2 (pt) vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva)
HK40091751A (en) Peptide-modified aav capsid
BR112023004176B1 (pt) Proteína de capsídeo de aav, polinucleotídeo, plasmídeo recombinante, partícula de vetor de aav, seu uso para tratamento de doenças musculares e composição farmacêutica
WO2026041758A1 (en) Modified aav capsid with enhanced transduction efficiency
US20240425879A1 (en) Compositions and methods for adeno-associated (aav) virus dnase expression

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination