ES2984259T3 - Capside de aav modificada con peptidos - Google Patents

Capside de aav modificada con peptidos Download PDF

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Publication number
ES2984259T3
ES2984259T3 ES21777271T ES21777271T ES2984259T3 ES 2984259 T3 ES2984259 T3 ES 2984259T3 ES 21777271 T ES21777271 T ES 21777271T ES 21777271 T ES21777271 T ES 21777271T ES 2984259 T3 ES2984259 T3 ES 2984259T3
Authority
ES
Spain
Prior art keywords
sequence
gene
aav
muscle
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.)
Active
Application number
ES21777271T
Other languages
English (en)
Spanish (es)
Inventor
Isabelle Richard
Natalia Dominguez
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.)
Institut Nat de la Sante Et De Larecherche Medicale Inserm
Genethon
Université d'Évry Val-d'Essonne
Original Assignee
Institut Nat de la Sante Et De Larecherche Medicale Inserm
Genethon
Université d'Évry Val-d'Essonne
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 Institut Nat de la Sante Et De Larecherche Medicale Inserm, Genethon, Université d'Évry Val-d'Essonne filed Critical Institut Nat de la Sante Et De Larecherche Medicale Inserm
Application granted granted Critical
Publication of ES2984259T3 publication Critical patent/ES2984259T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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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)
ES21777271T 2020-09-10 2021-09-10 Capside de aav modificada con peptidos Active ES2984259T3 (es)

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
ES2984259T3 true ES2984259T3 (es) 2024-10-29

Family

ID=72717827

Family Applications (1)

Application Number Title Priority Date Filing Date
ES21777271T Active ES2984259T3 (es) 2020-09-10 2021-09-10 Capside de aav modificada con peptidos

Country Status (11)

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

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20230004719A (ko) 2020-04-20 2023-01-06 테나야 테라퓨틱스, 인코포레이티드 조작된 캡시드를 갖는 아데노-연관 바이러스
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
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
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
CA3252764A1 (en) 2022-06-09 2023-12-14 Institut National de la Santé et de la Recherche Médicale GDE truncated at the N-terminal level for the treatment of type III glycogen storage disease
PL4536686T3 (pl) 2022-06-10 2026-03-23 Genethon Kapsyd aav zmodyfikowany peptydem o zwiększonej skuteczności transdukcji mięśniowej
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
CN120035601A (zh) * 2022-10-12 2025-05-23 Cpc株式会社 基因导入法、基因治疗法及组织再生法
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
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
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
CN121941776A (zh) * 2023-08-25 2026-04-28 新加坡科技研究局 用于鉴定向心肌和/或骨骼肌纤维进行差异化基因递送的新型aav变体的组合物和方法
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
CN119751595B (zh) * 2024-07-16 2025-09-09 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
WO2026064442A2 (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
AU2014287005C1 (en) * 2013-07-12 2021-06-03 The Children's Hospital Of Philadelphia AAV vector and assay for anti-AAV (adeno-associated virus) neutralizing antibodies
AU2017301600B2 (en) * 2016-07-26 2022-08-04 Biomarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins
CN112004925B (zh) 2018-04-05 2024-09-27 吉尼松公司 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
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
US20240209030A1 (en) 2024-06-27
JP2023544683A (ja) 2023-10-25
EP4421179A3 (en) 2024-12-18
BR112023004176A2 (pt) 2023-04-11
FI4211231T3 (fi) 2024-07-17
WO2022053630A1 (en) 2022-03-17
PT4211231T (pt) 2024-07-24
EP4211231B1 (en) 2024-06-26
DK4211231T3 (da) 2024-07-08
PL4211231T3 (pl) 2024-11-04
EP4211231A1 (en) 2023-07-19
CA3193128A1 (en) 2022-03-17
EP4421179A2 (en) 2024-08-28
JP7834725B2 (ja) 2026-03-24
CN116096431A (zh) 2023-05-09

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