JPWO2023178339A5 - - Google Patents

Info

Publication number
JPWO2023178339A5
JPWO2023178339A5 JP2024555341A JP2024555341A JPWO2023178339A5 JP WO2023178339 A5 JPWO2023178339 A5 JP WO2023178339A5 JP 2024555341 A JP2024555341 A JP 2024555341A JP 2024555341 A JP2024555341 A JP 2024555341A JP WO2023178339 A5 JPWO2023178339 A5 JP WO2023178339A5
Authority
JP
Japan
Prior art keywords
nucleic acid
sequence
promoter
expression
raav
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
JP2024555341A
Other languages
English (en)
Japanese (ja)
Other versions
JP2025509828A5 (https=
JP2025509828A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/US2023/064673 external-priority patent/WO2023178339A2/en
Publication of JP2025509828A publication Critical patent/JP2025509828A/ja
Publication of JPWO2023178339A5 publication Critical patent/JPWO2023178339A5/ja
Publication of JP2025509828A5 publication Critical patent/JP2025509828A5/ja
Pending legal-status Critical Current

Links

JP2024555341A 2022-03-18 2023-03-18 ウイルスベクターによるtnnt2関連心筋症を処置するための方法および組成物 Pending JP2025509828A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202263321264P 2022-03-18 2022-03-18
US63/321,264 2022-03-18
PCT/US2023/064673 WO2023178339A2 (en) 2022-03-18 2023-03-18 Methods and compositions for treating tnnt2 related cardiomyopathy with a viral vector

Publications (3)

Publication Number Publication Date
JP2025509828A JP2025509828A (ja) 2025-04-11
JPWO2023178339A5 true JPWO2023178339A5 (https=) 2026-03-27
JP2025509828A5 JP2025509828A5 (https=) 2026-03-27

Family

ID=88024553

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024555341A Pending JP2025509828A (ja) 2022-03-18 2023-03-18 ウイルスベクターによるtnnt2関連心筋症を処置するための方法および組成物

Country Status (8)

Country Link
US (1) US20250195694A1 (https=)
EP (1) EP4493702A4 (https=)
JP (1) JP2025509828A (https=)
KR (1) KR20250007509A (https=)
AU (1) AU2023234604A1 (https=)
CA (1) CA3254775A1 (https=)
IL (1) IL315697A (https=)
WO (1) WO2023178339A2 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025214477A1 (en) * 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors
WO2025226841A1 (en) * 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2
WO2025235807A1 (en) * 2024-05-10 2025-11-13 Solid Biosciences, Inc. Compositions and methods to improve graft survival

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP5553547B2 (ja) * 2009-07-14 2014-07-16 俊一 梶岡 トロポニン含有医薬組成物
US9783805B2 (en) * 2013-02-28 2017-10-10 City Of Hope Replication capable rAAV vectors encoding inhibitory siRNA and methods of their use
EP2792742A1 (en) * 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
AU2019258830B2 (en) * 2018-04-27 2025-12-18 Universität Heidelberg Modified AAV capsid polypeptides for treatment of muscular diseases
CN114207134B (zh) * 2019-03-25 2024-11-15 俄亥俄州国家创新基金会 工程化的mRNA序列及其用途
CN114450411A (zh) * 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
AU2020310201A1 (en) * 2019-07-10 2022-01-27 Locanabio, Inc. RNA-targeting knockdown and replacement compositions and methods for use
WO2021154923A2 (en) * 2020-01-29 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
WO2021163357A2 (en) * 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
KR20240118855A (ko) * 2021-12-10 2024-08-05 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 바이러스 벡터로 bag-3 관련 심근병증을 치료하는 방법 및 조성물

Similar Documents

Publication Publication Date Title
US20220331409A1 (en) Factor ix gene therapy
AU2017250791A1 (en) Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
JP6573991B2 (ja) 血管性浮腫の治療としてのc1eiのアデノ随伴ウイルス介在性送達
JPWO2022053630A5 (https=)
CN114276419B (zh) 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
US11077208B2 (en) Wilson's disease gene therapy
CN113121651B (zh) 低中和抗体腺相关病毒衣壳蛋白
JPWO2023178339A5 (https=)
CA3178591A1 (en) Immunosuppressive agents and viral delivery re-dosing methods for gene therapy
WO2023165477A1 (zh) 白介素1受体拮抗蛋白的表达盒以及基于aav的基因递送系统
CN113121654B (zh) 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体
US12415850B2 (en) Compositions and methods for regulating production of an antibody like protein and ribonucleic acid
WO2021169167A1 (en) Method for treating coronavirus infections
JPWO2022031756A5 (https=)
JPWO2023108159A5 (https=)
JPWO2023108157A5 (https=)
WO2023124741A1 (zh) 用于治疗肌营养不良症的转基因表达盒
CN121759520A (zh) 一种lamp2b的过表达载体及其应用
CN109306011B (zh) 靶向RyR2蛋白的胞内纳米抗体在制备抗心力衰竭药物中的用途
JP2022514271A5 (https=)
CN112961220A (zh) 新型腺相关病毒衣壳蛋白及包含其的新型腺相关病毒载体
WO2026046394A1 (en) Nucleic acids and uses thereof for plakophilin 2 (pkp2) gene therapy
JPWO2020038473A5 (https=)
RU2021105703A (ru) Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок фактора свёртывания крови IX, и ее применение
JPWO2023108029A5 (https=)