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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China
Prior art keywords
aav
sequence
muscle
modified
aav9
Prior art date
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Pending
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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 Santé et de la Recherche Médicale
Genethon
Original Assignee
Evry Wald Esson University
Institut National de la Santé et de la Recherche Médicale
Genethon
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Application filed by Evry Wald Esson University, Institut National de la Santé et de la Recherche Médicale, 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
EP20306005.8 2020-09-10
EP20306005 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

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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) EP4211231B1 (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 (22)

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Publication number Priority date Publication date Assignee Title
AU2021259422A1 (en) 2020-04-20 2022-12-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
WO2022204476A1 (en) 2021-03-26 2022-09-29 The Board Of Regents Of The University Of Texas System Nucleotide editing to reframe dmd transcripts by base editing and prime editing
WO2023012313A1 (en) 2021-08-04 2023-02-09 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
CN120590489A (zh) * 2022-04-12 2025-09-05 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
FI4536686T3 (fi) 2022-06-10 2026-01-13 Genethon Lihaksen parannetun transduktiotehokkuuden omaava peptidimodifioitu aav-kapsidi
JP2025532754A (ja) 2022-10-07 2025-10-03 ジェネトン 抗ｆａｐ ｃａｒ－ｔ細胞を使用する骨格筋ミオパチーの免疫療法
JPWO2024080311A1 (enExample) * 2022-10-12 2024-04-18
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス，インコーポレーテッド カプシドポリペプチドおよびその使用方法
WO2024188913A1 (en) 2023-03-10 2024-09-19 Genethon Induction of immune tolerance by aav vector comprising the combination of a liver detargeted capsid and a tandem liver-muscle specific promoter
WO2025003477A1 (en) 2023-06-29 2025-01-02 Genethon N-terminal truncated glycogen debranching enzymes for the treatment of glycogen storage disease iii
EP4766844A1 (en) * 2023-08-25 2026-07-01 Agency for Science, Technology And Research COMPOSITION AND METHODS FOR IDENTIFYING NEW AAV VARIANTS FOR ADMINISTRATING DIFFERENTIATED GENES TO CARDIOMYOCTS 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
CN119751596B (zh) * 2024-07-16 2025-08-01 广州派真生物技术有限公司 腺相关病毒突变体及其应用
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
WO2026122963A1 (en) 2024-12-06 2026-06-11 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105518145A (zh) * 2013-07-12 2016-04-20 费城儿童医院 Aav载体和用于抗aav（腺相关病毒）中和抗体的检测
CN108699565A (zh) * 2015-12-11 2018-10-23 加州理工学院 用于定向腺相关病毒(aav)的靶向肽
WO2019193119A1 (en) * 2018-04-05 2019-10-10 Genethon HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM
WO2019207132A1 (en) * 2018-04-27 2019-10-31 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6994018B2 (ja) * 2016-07-26 2022-01-14 バイオマリン ファーマシューティカル インコーポレイテッド 新規アデノ随伴ウイルスキャプシドタンパク質

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105518145A (zh) * 2013-07-12 2016-04-20 费城儿童医院 Aav载体和用于抗aav（腺相关病毒）中和抗体的检测
CN108699565A (zh) * 2015-12-11 2018-10-23 加州理工学院 用于定向腺相关病毒(aav)的靶向肽
WO2019193119A1 (en) * 2018-04-05 2019-10-10 Genethon HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS SEROTYPE BETWEEN AAV9 AND AAVrh74 WITH REDUCED LIVER TROPISM
US20210107948A1 (en) * 2018-04-05 2021-04-15 Genethon Hybrid Recombinant Adeno-Associated Virus Serotype Between AAV9 and AAVrh74 with Reduced Liver Tropism
WO2019207132A1 (en) * 2018-04-27 2019-10-31 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
CN112040988A (zh) * 2018-04-27 2020-12-04 海德堡大学 修饰的aav衣壳多肽用于治疗肌肉疾病
US20210363193A1 (en) * 2018-04-27 2021-11-25 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
HILDEGARD BÜNING等: "Capsid Modifications for Targeting and Improving the Efficacy of AAV Vectors", 《MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT》, vol. 12, 31 March 2019 (2019-03-31), pages 1 - 18 *
连文昌等: "AAV衣壳蛋白基因工程的修饰研究进展", 《生物技术通报》, no. 12, 26 December 2011 (2011-12-26), pages 22 - 26 *

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

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