IL313427A - Methods and preparations for treating BAG-3-related cardiomyopathy with a viral vector - Google Patents

Methods and preparations for treating BAG-3-related cardiomyopathy with a viral vector

Info

Publication number
IL313427A
IL313427A IL313427A IL31342724A IL313427A IL 313427 A IL313427 A IL 313427A IL 313427 A IL313427 A IL 313427A IL 31342724 A IL31342724 A IL 31342724A IL 313427 A IL313427 A IL 313427A
Authority
IL
Israel
Prior art keywords
compositions
methods
viral vector
related cardiomyopathy
treating bag
Prior art date
Application number
IL313427A
Other languages
English (en)
Hebrew (he)
Original Assignee
Aavantibio Inc
Univ Florida
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 Aavantibio Inc, Univ Florida filed Critical Aavantibio Inc
Publication of IL313427A publication Critical patent/IL313427A/en

Links

Classifications

    • 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
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N2750/14141—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
    • C12N2800/22—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • 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)
IL313427A 2021-12-10 2022-12-09 Methods and preparations for treating BAG-3-related cardiomyopathy with a viral vector IL313427A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163288520P 2021-12-10 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

Publications (1)

Publication Number Publication Date
IL313427A true IL313427A (en) 2024-08-01

Family

ID=86731334

Family Applications (1)

Application Number Title Priority Date Filing Date
IL313427A IL313427A (en) 2021-12-10 2022-12-09 Methods and preparations for treating BAG-3-related cardiomyopathy with a viral vector

Country Status (8)

Country Link
US (1) US20250041453A1 (enExample)
EP (1) EP4444364A4 (enExample)
JP (1) JP2024545183A (enExample)
KR (1) KR20240118855A (enExample)
AU (1) AU2022407655A1 (enExample)
CA (1) CA3242351A1 (enExample)
IL (1) IL313427A (enExample)
WO (1) WO2023108159A1 (enExample)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250195694A1 (en) * 2022-03-18 2025-06-19 University Of Florida Research Foundation, Incorporated Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector
KR20250169529A (ko) * 2023-02-13 2025-12-03 아스트라제네카 아일랜드 리미티드 유전자 요법을 위한 Bcl2-관련 아타노진 3(BAG3)을 인코딩하는 핵산
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 (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015117010A2 (en) * 2014-01-31 2015-08-06 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
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
US20190328836A1 (en) * 2017-01-09 2019-10-31 Duke University Methods for treating ischemic injury
CN113015742A (zh) * 2018-06-08 2021-06-22 联邦高等教育系统天普大学 优化bag3基因治疗
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

Also Published As

Publication number Publication date
US20250041453A1 (en) 2025-02-06
EP4444364A4 (en) 2026-07-01
KR20240118855A (ko) 2024-08-05
EP4444364A1 (en) 2024-10-16
AU2022407655A1 (en) 2024-06-20
JP2024545183A (ja) 2024-12-05
CA3242351A1 (en) 2023-06-15
WO2023108159A1 (en) 2023-06-15

Similar Documents

Publication Publication Date Title
IL315697A (en) Methods and compositions for treating TNNT2-related cardiomyopathy with a viral vector
IL313426A (en) Methods and preparations for treating MYBPC3-associated hypertrophic cardiomyopathy with a viral vector
IL315695A (en) Methods and compositions for treating RBM20-associated cardiomyopathy with a viral vector
IL280947A (en) Methods and preparations for genome modulation
IL276341A (en) Methods and compositions for bypassing virus antibody vectors
ES3066018T3 (en) Carton comprising stress-relief panels
IL288419A (en) Recombinant viral herpes vector
GB202010009D0 (en) Vector
ZA202202370B (en) Compositions and methods for treating viral infections
GB202019108D0 (en) Vector
GB2590774B (en) Vector
IL287262A (en) Methods and preparations for transgene expression
IL286476A (en) Vector and method for treating angelman syndrome
GB201907413D0 (en) Viral vector
HK40118454A (en) Methods and compositions for treating bag-3 related cardiomyopathy with a viral vector
IL277773A (en) Preparations and methods for the treatment of ischemia and cardiomyopathy
HK40124087A (en) Methods and compositions for treating rbm20 related cardiomyopathy with a viral vector
GB202219321D0 (en) Closure
GB202205606D0 (en) Lentiviral vector
GB202312083D0 (en) Viral vector
IL316314A (en) Compositions and methods for viral vectors
HK40122589A (en) Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector
IL293194B1 (en) Intermodal calling branch instruction
IL317172A (en) Lentiviral vector maintenance vehicles and uses thereof
IL316172A (en) Lentiviral vector