KR20240118855A - 바이러스 벡터로 bag-3 관련 심근병증을 치료하는 방법 및 조성물 - Google Patents

바이러스 벡터로 bag-3 관련 심근병증을 치료하는 방법 및 조성물 Download PDF

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KR20240118855A
KR20240118855A KR1020247023092A KR20247023092A KR20240118855A KR 20240118855 A KR20240118855 A KR 20240118855A KR 1020247023092 A KR1020247023092 A KR 1020247023092A KR 20247023092 A KR20247023092 A KR 20247023092A KR 20240118855 A KR20240118855 A KR 20240118855A
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sequence
nucleic acid
raav
promoter
expression construct
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배리 존 번
페드로 크루즈
아이린 졸로투킨
와이들러 케이시
마누엘라 코르티
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유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드
애반티바이오, 인크.
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    • C07K14/4702—Regulators; Modulating activity
    • A—HUMAN NECESSITIES
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cardiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020247023092A 2021-12-10 2022-12-09 바이러스 벡터로 bag-3 관련 심근병증을 치료하는 방법 및 조성물 Pending KR20240118855A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163288520P 2021-12-10 2021-12-10
US63/288,520 2021-12-10
PCT/US2022/081323 WO2023108159A1 (en) 2021-12-10 2022-12-09 Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector

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KR20240118855A true KR20240118855A (ko) 2024-08-05

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KR1020247023092A Pending KR20240118855A (ko) 2021-12-10 2022-12-09 바이러스 벡터로 bag-3 관련 심근병증을 치료하는 방법 및 조성물

Country Status (8)

Country Link
US (1) US20250041453A1 (https=)
EP (1) EP4444364A1 (https=)
JP (1) JP2024545183A (https=)
KR (1) KR20240118855A (https=)
AU (1) AU2022407655A1 (https=)
CA (1) CA3242351A1 (https=)
IL (1) IL313427A (https=)
WO (1) WO2023108159A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3254775A1 (en) * 2022-03-18 2023-09-21 University Of Florida Research Foundation, Incorporated METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
EP4665749A1 (en) * 2023-02-13 2025-12-24 Astrazeneca Ireland Limited Nucleic acids encoding bcl2-associated athanogene 3 (bag3) for gene therapy
WO2024215655A1 (en) * 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
AU2024265705A1 (en) * 2023-05-02 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3099333B8 (en) * 2014-01-31 2020-12-30 Temple University of the Commonwealth System of Higher Education Bag3 as a target for therapy of heart failure
WO2015153889A2 (en) * 2014-04-02 2015-10-08 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
EP3452101A2 (en) * 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
US20190328836A1 (en) * 2017-01-09 2019-10-31 Duke University Methods for treating ischemic injury
KR20210112339A (ko) * 2019-01-04 2021-09-14 울트라제닉스 파마수티컬 인코포레이티드 윌슨병을 치료하기 위한 유전자 요법 구축물
WO2021154923A2 (en) * 2020-01-29 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing aav particles

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Publication number Publication date
WO2023108159A1 (en) 2023-06-15
CA3242351A1 (en) 2023-06-15
US20250041453A1 (en) 2025-02-06
IL313427A (en) 2024-08-01
EP4444364A1 (en) 2024-10-16
JP2024545183A (ja) 2024-12-05
AU2022407655A1 (en) 2024-06-20

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