AU2021320244A1 - Methods of treating cardiac disorders and congestive heart failure and administering AAV vectors - Google Patents

Methods of treating cardiac disorders and congestive heart failure and administering AAV vectors Download PDF

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Publication number
AU2021320244A1
AU2021320244A1 AU2021320244A AU2021320244A AU2021320244A1 AU 2021320244 A1 AU2021320244 A1 AU 2021320244A1 AU 2021320244 A AU2021320244 A AU 2021320244A AU 2021320244 A AU2021320244 A AU 2021320244A AU 2021320244 A1 AU2021320244 A1 AU 2021320244A1
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AU
Australia
Prior art keywords
cardiomyopathy
heart
seq
heart failure
syndrome
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.)
Pending
Application number
AU2021320244A
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English (en)
Inventor
Sinclair COOPER
Antonia EVRIPIOTI
Roger Hajjar
Juan Manuel IGLESIAS GONZALEZ
Michael W. O'CALLAGHAN
Michael L. Roberts
Ferzin SETHNA
Anna Tretiakova
Jorge Omar YANEZ-CUNA
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AskBio Inc
Original Assignee
AskBio Inc
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 AskBio Inc filed Critical AskBio Inc
Publication of AU2021320244A1 publication Critical patent/AU2021320244A1/en
Assigned to AskBio Inc. reassignment AskBio Inc. Amend patent request/document other than specification (104) Assignors: ASKLEPIOS BIOPHARMACEUTICAL, INC.
Pending legal-status Critical Current

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Classifications

    • 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
    • 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)
AU2021320244A 2020-08-05 2021-08-05 Methods of treating cardiac disorders and congestive heart failure and administering AAV vectors Pending AU2021320244A1 (en)

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
AU2021320244A1 true AU2021320244A1 (en) 2023-03-23

Family

ID=80120133

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021320244A Pending AU2021320244A1 (en) 2020-08-05 2021-08-05 Methods of treating cardiac disorders and congestive heart failure and administering AAV vectors

Country Status (8)

Country Link
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=)

Families Citing this family (11)

* 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

Family Cites Families (3)

* 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

Also Published As

Publication number Publication date
IL300367A (en) 2023-04-01
JP2023536921A (ja) 2023-08-30
WO2022031914A2 (en) 2022-02-10
CN116406297A (zh) 2023-07-07
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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Owner name: ASKBIO INC.

Free format text: FORMER NAME(S): ASKLEPIOS BIOPHARMACEUTICAL, INC.