CN115516100A - 靶向肌肉疾病中肌肉的优化基因疗法 - Google Patents

靶向肌肉疾病中肌肉的优化基因疗法 Download PDF

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Publication number
CN115516100A
CN115516100A CN202080097838.0A CN202080097838A CN115516100A CN 115516100 A CN115516100 A CN 115516100A CN 202080097838 A CN202080097838 A CN 202080097838A CN 115516100 A CN115516100 A CN 115516100A
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gne
raav
muscle
promoter
ires
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P·T·马丁
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Nationwide Childrens Hospital Inc
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Nationwide Childrens Hospital Inc
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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
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202080097838.0A 2019-12-20 2020-12-21 靶向肌肉疾病中肌肉的优化基因疗法 Pending CN115516100A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962951564P 2019-12-20 2019-12-20
US62/951564 2019-12-20
PCT/US2020/066477 WO2021127655A1 (en) 2019-12-20 2020-12-21 Optimized gene therapy for targeting muscle in muscle diseases

Publications (1)

Publication Number Publication Date
CN115516100A true CN115516100A (zh) 2022-12-23

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CN202080097838.0A Pending CN115516100A (zh) 2019-12-20 2020-12-21 靶向肌肉疾病中肌肉的优化基因疗法

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US (1) US20230227515A1 (https=)
EP (1) EP4077687A1 (https=)
JP (2) JP2023507794A (https=)
CN (1) CN115516100A (https=)
AU (1) AU2020408225A1 (https=)
CA (1) CA3165469A1 (https=)
IL (1) IL294072A (https=)
WO (1) WO2021127655A1 (https=)

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CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
IL301675A (en) 2020-10-09 2023-05-01 Tenaya Therapeutics Inc Methods and preparations for placophilin-2 gene therapy
CA3236188A1 (en) * 2021-10-27 2023-05-04 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i
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
EP4698651A1 (en) * 2023-04-19 2026-02-25 Gradalis, Inc. Compositions and methods for modulating sialic acid production and treating hereditary inclusion body myopathy (hibm)
WO2025157262A1 (en) * 2024-01-25 2025-07-31 Sichuan Real & Best Biotech Co., Ltd. Nucleic acids encoding follistatin and uses thereof
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
WO2025188993A2 (en) 2024-03-07 2025-09-12 Research Institute At Nationwide Children's Hospital Gene therapy for treating gne-related disorders
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

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WO2007059303A2 (en) * 2005-11-15 2007-05-24 The Board Of Trustees Of The Leland Stanford Junior University Compositions for coordinated vegf and pdgf expression, and methods of use
WO2013102904A1 (en) * 2012-01-05 2013-07-11 Hadasit Medical Research Services & Development Ltd. Methods and compositions for gene delivery
WO2016016119A1 (en) * 2014-07-26 2016-02-04 Consiglio Nazionale Delle Ricerche Compositions and methods for treatment of muscular dystrophy
WO2017120470A1 (en) * 2016-01-06 2017-07-13 Wilson D Travis Methods and compositions for the prevention and treatment of duchenne muscular dystrophy

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PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
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WO2002002148A2 (en) * 2000-06-30 2002-01-10 Collateral Therapeutics, Inc. Dual recombinant gene therapy compositions and methods of use
WO2007059303A2 (en) * 2005-11-15 2007-05-24 The Board Of Trustees Of The Leland Stanford Junior University Compositions for coordinated vegf and pdgf expression, and methods of use
WO2013102904A1 (en) * 2012-01-05 2013-07-11 Hadasit Medical Research Services & Development Ltd. Methods and compositions for gene delivery
WO2016016119A1 (en) * 2014-07-26 2016-02-04 Consiglio Nazionale Delle Ricerche Compositions and methods for treatment of muscular dystrophy
WO2017120470A1 (en) * 2016-01-06 2017-07-13 Wilson D Travis Methods and compositions for the prevention and treatment of duchenne muscular dystrophy

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AU2020408225A1 (en) 2022-07-14
JP2023507794A (ja) 2023-02-27
WO2021127655A1 (en) 2021-06-24
JP2026009430A (ja) 2026-01-20
EP4077687A1 (en) 2022-10-26
CA3165469A1 (en) 2021-06-24
IL294072A (en) 2022-08-01
US20230227515A1 (en) 2023-07-20

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