JP2022549140A5 - - Google Patents

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Publication number
JP2022549140A5
JP2022549140A5 JP2022517343A JP2022517343A JP2022549140A5 JP 2022549140 A5 JP2022549140 A5 JP 2022549140A5 JP 2022517343 A JP2022517343 A JP 2022517343A JP 2022517343 A JP2022517343 A JP 2022517343A JP 2022549140 A5 JP2022549140 A5 JP 2022549140A5
Authority
JP
Japan
Prior art keywords
pkp2
gene therapy
therapy vector
therapeutic formulation
isoform
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
JP2022517343A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022549140A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2020/076290 external-priority patent/WO2021053222A1/en
Publication of JP2022549140A publication Critical patent/JP2022549140A/ja
Publication of JP2022549140A5 publication Critical patent/JP2022549140A5/ja
Pending legal-status Critical Current

Links

JP2022517343A 2019-09-20 2020-09-21 遺伝子療法組成物および不整脈源性右心室心筋症の治療 Pending JP2022549140A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962903103P 2019-09-20 2019-09-20
US62/903,103 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 (2)

Publication Number Publication Date
JP2022549140A JP2022549140A (ja) 2022-11-24
JP2022549140A5 true JP2022549140A5 (https=) 2023-09-22

Family

ID=72615856

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022517343A Pending JP2022549140A (ja) 2019-09-20 2020-09-21 遺伝子療法組成物および不整脈源性右心室心筋症の治療

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

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

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