IL291488A - Combination of gene therapy and treatment of right ventricular arrhythmogenic cardiomyopathy - Google Patents

Combination of gene therapy and treatment of right ventricular arrhythmogenic cardiomyopathy

Info

Publication number
IL291488A
IL291488A IL291488A IL29148822A IL291488A IL 291488 A IL291488 A IL 291488A IL 291488 A IL291488 A IL 291488A IL 29148822 A IL29148822 A IL 29148822A IL 291488 A IL291488 A IL 291488A
Authority
IL
Israel
Prior art keywords
arrythmogenic
cardiomyopathy
treatment
gene therapy
right ventricular
Prior art date
Application number
IL291488A
Other languages
English (en)
Hebrew (he)
Inventor
Thomas Voit
Julie Dumonceaux
Perry Elliot
Virginie Mariot
Original Assignee
Ucl Business Ltd
Thomas Voit
Julie Dumonceaux
Perry Elliot
Virginie Mariot
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 Ucl Business Ltd, Thomas Voit, Julie Dumonceaux, Perry Elliot, Virginie Mariot filed Critical Ucl Business Ltd
Publication of IL291488A publication Critical patent/IL291488A/en

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for 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
    • 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)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cardiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
IL291488A 2019-09-20 2022-03-18 Combination of gene therapy and treatment of right ventricular arrhythmogenic cardiomyopathy IL291488A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962903103P 2019-09-20 2019-09-20
PCT/EP2020/076290 WO2021053222A1 (en) 2019-09-20 2020-09-21 Gene therapy composition and treatment of right ventricular arrythmogenic cardiomyopathy

Publications (1)

Publication Number Publication Date
IL291488A true IL291488A (en) 2022-05-01

Family

ID=72615856

Family Applications (1)

Application Number Title Priority Date Filing Date
IL291488A IL291488A (en) 2019-09-20 2022-03-18 Combination of gene therapy and treatment of right ventricular arrhythmogenic cardiomyopathy

Country Status (9)

Country Link
US (3) US20220389452A1 (https=)
EP (1) EP4031672A1 (https=)
JP (1) JP2022549140A (https=)
KR (1) KR20220066914A (https=)
AU (1) AU2020350140A1 (https=)
CA (1) CA3151036A1 (https=)
IL (1) IL291488A (https=)
TW (1) TWI897882B (https=)
WO (1) WO2021053222A1 (https=)

Families Citing this family (15)

* 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
EP4087489B1 (en) * 2020-01-08 2025-05-14 Vitruvia Holdings Inc. Methods and computing system for processing ultrasound image to determine health of subdermal tissue
EP4103214B1 (en) 2020-02-13 2026-04-29 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
KR20230043869A (ko) * 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
BR112023006305A2 (pt) * 2020-10-09 2023-05-09 Tenaya Therapeutics Inc Métodos e composições de terapia gênica com placofilina-2
US11781156B2 (en) * 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
AU2022239846A1 (en) * 2021-03-19 2023-10-26 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy
CN117178057A (zh) * 2021-04-12 2023-12-05 加利福尼亚大学董事会 用于致心律失常性右心室心肌病的基因治疗
JP2025508743A (ja) * 2022-02-18 2025-04-10 ギンコ バイオワークス インコーポレイテッド 不整脈原性心筋症のための遺伝子治療
US20250205365A1 (en) * 2022-03-18 2025-06-26 University Of Florida Research Foundation, Incorporated Methods and compositions for treating rbm20 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
WO2024064796A2 (en) * 2022-09-21 2024-03-28 Rejuvenate Bio Gene therapy methods for arrhythmogenic cardiomyopathy
TW202421788A (zh) * 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用ａａｖ基因療法載體治療致心律不整性心肌病
CN116064612A (zh) * 2022-11-17 2023-05-05 百世诺（北京）医疗科技有限公司 肥厚型心肌病变异基因alpk3及其应用
WO2025010312A1 (en) * 2023-07-06 2025-01-09 The Regents Of The University Of California Gene replacement therapy for alpk3 cardiomyopathy

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2792742A1 (en) * 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
SG11201803934YA (en) * 2015-11-11 2018-06-28 Intrexon Corp Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
WO2018208998A1 (en) * 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
RU2770922C2 (ru) * 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
ES2976559T3 (es) * 2017-09-20 2024-08-05 Univ California Terapia génica con conexina 43 para restaurar la función eléctrica y cardíaca y la estructura cardíaca, en la miocardiopatía arritmogénica del ventrículo derecho
US20210363193A1 (en) * 2018-04-27 2021-11-25 Universität Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
CN109004092A (zh) 2018-06-29 2018-12-14 云谷（固安）科技有限公司 有机电致发光器件和有机电致发光装置
KR20230043869A (ko) * 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법

Also Published As

Publication number Publication date
WO2021053222A1 (en) 2021-03-25
TW202126674A (zh) 2021-07-16
KR20220066914A (ko) 2022-05-24
US20250163470A1 (en) 2025-05-22
JP2022549140A (ja) 2022-11-24
US20240076696A1 (en) 2024-03-07
CA3151036A1 (en) 2021-03-25
US20220389452A1 (en) 2022-12-08
EP4031672A1 (en) 2022-07-27
AU2020350140A1 (en) 2022-03-31
TWI897882B (zh) 2025-09-21

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