FI4211231T3 - Peptidimodifioitu aav-kapsidi - Google Patents

Peptidimodifioitu aav-kapsidi Download PDF

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Publication number
FI4211231T3
FI4211231T3 FIEP21777271.4T FI21777271T FI4211231T3 FI 4211231 T3 FI4211231 T3 FI 4211231T3 FI 21777271 T FI21777271 T FI 21777271T FI 4211231 T3 FI4211231 T3 FI 4211231T3
Authority
FI
Finland
Prior art keywords
sequence
capsid protein
aav capsid
amino acids
modified
Prior art date
Application number
FIEP21777271.4T
Other languages
English (en)
Finnish (fi)
Inventor
Isabelle Richard
Natalia Dominguez
Original Assignee
Genethon
Inst Nat Sante Rech Med
Univ Devry Val Dessonne
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 Genethon, Inst Nat Sante Rech Med, Univ Devry Val Dessonne filed Critical Genethon
Application granted granted Critical
Publication of FI4211231T3 publication Critical patent/FI4211231T3/fi

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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)
FIEP21777271.4T 2020-09-10 2021-09-10 Peptidimodifioitu aav-kapsidi FI4211231T3 (fi)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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
FI4211231T3 true FI4211231T3 (fi) 2024-07-17

Family

ID=72717827

Family Applications (1)

Application Number Title Priority Date Filing Date
FIEP21777271.4T FI4211231T3 (fi) 2020-09-10 2021-09-10 Peptidimodifioitu aav-kapsidi

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)

* Cited by examiner, † Cited by third party
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

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015006743A1 (en) * 2013-07-12 2015-01-15 The Children's Hospital Of Philadelphia Aav vector and assay for anti-aav (adeno-associated virus) neutralizing antibodies
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
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
JP7374119B2 (ja) * 2018-04-05 2023-11-06 ジェネトン 減少した肝臓向性を有するAAV9とAAVrh74とのハイブリッド組換えアデノ随伴ウイルス血清型
CA3097375A1 (en) 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases

Also Published As

Publication number Publication date
CN116096431A (zh) 2023-05-09
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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