CN116406297A - 治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法 - Google Patents

治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法 Download PDF

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CN116406297A
CN116406297A CN202180068345.9A CN202180068345A CN116406297A CN 116406297 A CN116406297 A CN 116406297A CN 202180068345 A CN202180068345 A CN 202180068345A CN 116406297 A CN116406297 A CN 116406297A
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heart
cardiomyopathy
seq
heart failure
syndrome
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Chinese (zh)
Inventor
迈克尔·W·奥卡拉汉
费尔津·赛斯纳
罗杰·哈尔
安娜·特里亚科娃
迈克尔·L·罗伯茨
胡安·曼努埃尔·伊格莱西亚斯·冈萨雷斯
安东尼亚·埃夫里皮奥蒂
辛克莱·库珀
豪尔赫·奥马尔·亚内斯库纳
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AskBio Inc
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Asklepios Biopharmaceutical Inc
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Publication of CN116406297A publication Critical patent/CN116406297A/zh
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    • 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
    • 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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • 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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Cardiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202180068345.9A 2020-08-05 2021-08-05 治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法 Pending CN116406297A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063061342P 2020-08-05 2020-08-05
US63/061,342 2020-08-05
US202163214119P 2021-06-23 2021-06-23
US63/214,119 2021-06-23
PCT/US2021/044650 WO2022031914A2 (en) 2020-08-05 2021-08-05 Methods of treating cardiac disorders and congestive heart failure and administering aav vectors

Publications (1)

Publication Number Publication Date
CN116406297A true CN116406297A (zh) 2023-07-07

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CN202180068345.9A Pending CN116406297A (zh) 2020-08-05 2021-08-05 治疗心脏疾患和充血性心力衰竭以及施用aav载体的方法

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US (1) US20230340528A1 (https=)
EP (1) EP4192580A4 (https=)
JP (1) JP2023536921A (https=)
CN (1) CN116406297A (https=)
AU (1) AU2021320244A1 (https=)
CA (1) CA3188398A1 (https=)
IL (1) IL300367A (https=)
WO (1) WO2022031914A2 (https=)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3947700A4 (en) 2019-04-01 2023-01-04 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
BR112023006305A2 (pt) 2020-10-09 2023-05-09 Tenaya Therapeutics Inc Métodos e composições de terapia gênica com placofilina-2
AU2022297795A1 (en) * 2021-06-23 2024-01-04 AskBio Inc. Regulatory nucleic acid sequences
WO2023178338A2 (en) * 2022-03-18 2023-09-21 University Of Florida Research Foundation, Incorporated Methods and compositions for treating tmem43 related cardiomyopathy with a viral vector
JP2025512405A (ja) * 2022-04-11 2025-04-17 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の処置の方法
CN116256523B (zh) * 2023-02-02 2023-09-12 香港大学深圳医院 生物标志物在制备用于糖尿病患者HFpEF检测试剂中的应用
IL322735A (en) * 2023-02-13 2025-10-01 Astrazeneca Ireland Ltd Nucleic acids encoding BCL2-associated athenogen 3 (BAG3) for gene therapy
CN116644347B (zh) * 2023-05-23 2025-11-28 珠海市康利莱医疗器械有限公司 一种用于风湿性瓣膜病的血清差异多肽检测系统
EP4719440A1 (en) * 2023-06-01 2026-04-08 Alkahest, Inc. Compositions of blood plasma fractions and blood plasma subfractions and their use in treatment of disease
TW202525313A (zh) * 2023-09-06 2025-07-01 瑞士商迪納柯公司 用ａａｖ基因治療載體及治療調配物治療肥厚性心肌病之方法
WO2025259575A1 (en) * 2024-06-10 2025-12-18 Asklepios Biopharmaceutical, Inc. Methods and compositions for treatment of duchenne muscular dystrophy

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101056539B (zh) * 2004-09-09 2012-12-12 综合医院公司 调节心脏细胞中的磷酸酶活性
BRPI0912559A2 (pt) * 2008-05-16 2015-10-13 Corthera Inc método de tratar insuficiência cardíaca crônica
EP4081643A1 (en) * 2019-12-24 2022-11-02 Asklepios Biopharmaceutical, Inc. Regulatory nucleic acid sequences

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Publication number Publication date
AU2021320244A1 (en) 2023-03-23
IL300367A (en) 2023-04-01
JP2023536921A (ja) 2023-08-30
WO2022031914A2 (en) 2022-02-10
CA3188398A1 (en) 2022-02-10
WO2022031914A3 (en) 2022-03-31
EP4192580A2 (en) 2023-06-14
EP4192580A4 (en) 2025-06-18
US20230340528A1 (en) 2023-10-26

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