TWI897882B - 右心室心律失常性心肌病之基因療法組合物及治療 - Google Patents

右心室心律失常性心肌病之基因療法組合物及治療

Info

Publication number
TWI897882B
TWI897882B TW109132629A TW109132629A TWI897882B TW I897882 B TWI897882 B TW I897882B TW 109132629 A TW109132629 A TW 109132629A TW 109132629 A TW109132629 A TW 109132629A TW I897882 B TWI897882 B TW I897882B
Authority
TW
Taiwan
Prior art keywords
pkp2
gene therapy
vector
nucleic acid
acid sequence
Prior art date
Application number
TW109132629A
Other languages
English (en)
Chinese (zh)
Other versions
TW202126674A (zh
Inventor
湯瑪士 維伊特
裘莉 迪蒙索
派利 艾略特
維吉尼 馬里奧特
Original Assignee
英商Ｕｃｌ商業有限責任公司
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 英商Ｕｃｌ商業有限責任公司 filed Critical 英商Ｕｃｌ商業有限責任公司
Publication of TW202126674A publication Critical patent/TW202126674A/zh
Application granted granted Critical
Publication of TWI897882B publication Critical patent/TWI897882B/zh

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
    • 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
    • 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
    • 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)
TW109132629A 2019-09-20 2020-09-21 右心室心律失常性心肌病之基因療法組合物及治療 TWI897882B (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962903103P 2019-09-20 2019-09-20
US62/903,103 2019-09-20

Publications (2)

Publication Number Publication Date
TW202126674A TW202126674A (zh) 2021-07-16
TWI897882B true TWI897882B (zh) 2025-09-21

Family

ID=72615856

Family Applications (1)

Application Number Title Priority Date Filing Date
TW109132629A TWI897882B (zh) 2019-09-20 2020-09-21 右心室心律失常性心肌病之基因療法組合物及治療

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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019060619A1 (en) * 2017-09-20 2019-03-28 The Regents Of The University Of California STRATEGY OF GENE THERAPY FOR RESTORING ELECTRICAL AND CARDIAC FUNCTION AND CARDIAC STRUCTURE IN ARYTHMOGENIC RIGHT VENTRICULAR CARDIOMYOPATHY

Family Cites Families (7)

* 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
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019060619A1 (en) * 2017-09-20 2019-03-28 The Regents Of The University Of California STRATEGY OF GENE THERAPY FOR RESTORING ELECTRICAL AND CARDIAC FUNCTION AND CARDIAC STRUCTURE IN ARYTHMOGENIC RIGHT VENTRICULAR CARDIOMYOPATHY

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
期刊 Francisco M. Cruz et al. Exercise Triggers ARVC Phenotype in Mice Expressing a Disease-Causing Mutated Version of Human Plakophilin-2. Journal of the American College of Cardiology Vol.65,No.14 Journal of the American College of Cardiology 2015/04/14 P.1438-1450.;期刊 Kyle Chamberlain et al. Cardiac gene therapy with adeno-associated virus-based vectors. Current Opinion in Cardiology 32(3) Current Opinion in Cardiology 2017/05 p.275-282. *
期刊 Kyle Chamberlain et al. Cardiac gene therapy with adeno-associated virus-based vectors. Current Opinion in Cardiology 32(3) Current Opinion in Cardiology 2017/05 p.275-282.

Also Published As

Publication number Publication date
WO2021053222A1 (en) 2021-03-25
TW202126674A (zh) 2021-07-16
KR20220066914A (ko) 2022-05-24
IL291488A (en) 2022-05-01
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

Similar Documents

Publication Publication Date Title
TWI897882B (zh) 右心室心律失常性心肌病之基因療法組合物及治療
US20240042059A1 (en) Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy
ES2741502T3 (es) Cápside de AAV8 modificada para la transferencia génica para terapias de retina
JP7011580B2 (ja) 網膜色素変性症の治療
JP2024517843A (ja) ステレオシリン二重ベクター系を使用して感音性難聴を治療するための組成物及び方法
JP2025163022A (ja) 停止していない鼓動している心臓への薬物灌流
US20230047424A1 (en) Gene therapy composition and treatment for myh7-linked cardiomyopathy
US20250099620A1 (en) Gene therapy composition and treatment for dystrophin-related cardiomyopathy
CN110699367B (zh) 编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
WO2020010491A1 (zh) 编码人nadh脱氢酶亚单位4蛋白的核酸及其应用
Bowles et al. Prospects for gene therapy for inherited cardiomyopathies
HK40112779A (en) Drug perfusion into an unarrested beating heart
HK40077296B (en) Drug perfusion into an unarrested beating heart
HK40077296A (en) Drug perfusion into an unarrested beating heart