DK3684422T3 - Connexin-43 gene therapy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy - Google Patents

Connexin-43 gene therapy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy Download PDF

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Publication number
DK3684422T3
DK3684422T3 DK18858620.0T DK18858620T DK3684422T3 DK 3684422 T3 DK3684422 T3 DK 3684422T3 DK 18858620 T DK18858620 T DK 18858620T DK 3684422 T3 DK3684422 T3 DK 3684422T3
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cardiac
connexin
gene therapy
right ventricular
arrhythmogenic right
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DK18858620.0T
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English (en)
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DK3684422T5 (en
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Farah Sheikh
Jing Zhang
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Univ California
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    • 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
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • A61P9/06—Antiarrhythmics
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0375—Animal model for cardiovascular diseases
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cardiology (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
DK18858620.0T 2017-09-20 2018-09-20 Connexin-43 gene therapy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy DK3684422T5 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762560989P 2017-09-20 2017-09-20
PCT/US2018/052057 WO2019060619A1 (en) 2017-09-20 2018-09-20 STRATEGY OF GENE THERAPY FOR RESTORING ELECTRICAL AND CARDIAC FUNCTION AND CARDIAC STRUCTURE IN ARYTHMOGENIC RIGHT VENTRICULAR CARDIOMYOPATHY

Publications (2)

Publication Number Publication Date
DK3684422T3 true DK3684422T3 (en) 2024-03-25
DK3684422T5 DK3684422T5 (en) 2024-08-05

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
DK18858620.0T DK3684422T5 (en) 2017-09-20 2018-09-20 Connexin-43 gene therapy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy

Country Status (11)

Country Link
US (1) US20200215155A1 (da)
EP (2) EP3684422B1 (da)
JP (2) JP7418826B2 (da)
CN (1) CN111107880A (da)
AU (1) AU2018335401A1 (da)
CA (1) CA3076227A1 (da)
DK (1) DK3684422T5 (da)
ES (1) ES2976559T3 (da)
IL (1) IL273448A (da)
PT (1) PT3684422T (da)
WO (1) WO2019060619A1 (da)

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EP3729381B1 (en) 2017-12-22 2023-01-18 Magic Leap, Inc. Viewpoint dependent brick selection for fast volumetric reconstruction
SG11202110607WA (en) 2019-04-01 2021-10-28 Tenaya Therapeutics Inc Adeno-associated virus with engineered capsid
US20220389452A1 (en) * 2019-09-20 2022-12-08 Ucl Business Ltd Gene therapy composition and treatment of right ventricular arrythmogenic cardiomyopathy
WO2021187380A1 (ja) * 2020-03-16 2021-09-23 国立大学法人大阪大学 不整脈源性心筋症患者由来の多能性幹細胞およびその利用ならびに不整脈源性心筋症治療用医薬
KR20230043869A (ko) * 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
KR20230084542A (ko) * 2020-10-09 2023-06-13 테나야 테라퓨틱스, 인코포레이티드 플라코필린 2 유전자 치료 방법 및 조성물
EP4308172A2 (en) * 2021-03-19 2024-01-24 UCL Business Ltd Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy
WO2023159190A1 (en) * 2022-02-18 2023-08-24 Ginkgo Bioworks, Inc. Gene therapy for arrhythmogenic cardiomyopathy
WO2023219655A1 (en) * 2022-05-13 2023-11-16 University Of Utah Research Foundation Gja1-20k to limited cardiac arrhythmias
WO2024050464A2 (en) * 2022-08-31 2024-03-07 University Of Utah Research Foundation Gja1-20k compositions and methods for mitigating ischemia-reperfusion injury

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2324540T3 (es) 1998-05-27 2009-08-10 Genzyme Corporation Vectores aav para la fabricacion de medicamentos para la administracion potenciada por conveccion.
EP1453382A4 (en) * 2001-11-08 2005-05-18 Univ California METHODS AND COMPOSITIONS FOR CORRECTION OF CARDIAC CONDUCTION DISORDERS
CN1703257A (zh) * 2002-11-16 2005-11-30 加利福尼亚大学董事会 心脏刺激系统和方法
WO2005085423A1 (en) * 2004-02-27 2005-09-15 Michigan State University Oct-4 and gjic expression as markers for adult human stem cells and cancer cell precursors
US7729761B2 (en) * 2004-07-14 2010-06-01 Cardiac Pacemakers, Inc. Method and apparatus for controlled gene or protein delivery
AU2005295720A1 (en) * 2004-10-13 2006-04-27 Medtronic, Inc. Cellular and genetic intervention to treat ventricular tachycardia
KR20080031154A (ko) * 2005-02-03 2008-04-08 코다 테라퓨틱스 (엔지) 리미티드 항-코넥신 화합물 및 그의 용도
WO2009111755A2 (en) * 2008-03-07 2009-09-11 The Trustees Of Columbia University In The City Of New York Compensating for atrioventricular block using a nucleic acid encoding a sodium channel or gap junction protein
WO2009120082A2 (en) * 2008-03-27 2009-10-01 Academisch Medisch Centrum Bij De Universiteit Van Amsterdam Means and methods for influencing electrical activity of cells
EP2792742A1 (en) * 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
CN106596972B (zh) * 2016-12-16 2018-08-10 青岛科技大学 一种检测力竭运动小鼠心肌连接蛋白43含量的方法

Also Published As

Publication number Publication date
CA3076227A1 (en) 2019-03-28
EP4382116A1 (en) 2024-06-12
AU2018335401A1 (en) 2020-04-02
WO2019060619A1 (en) 2019-03-28
EP3684422A1 (en) 2020-07-29
JP7418826B2 (ja) 2024-01-22
EP3684422A4 (en) 2021-06-23
CN111107880A (zh) 2020-05-05
PT3684422T (pt) 2024-03-27
JP2024050553A (ja) 2024-04-10
IL273448A (en) 2020-05-31
JP2020534314A (ja) 2020-11-26
US20200215155A1 (en) 2020-07-09
DK3684422T5 (en) 2024-08-05
ES2976559T3 (es) 2024-08-05
EP3684422B1 (en) 2023-12-20

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