JP2023516886A5 - - Google Patents

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Publication number
JP2023516886A5
JP2023516886A5 JP2022546698A JP2022546698A JP2023516886A5 JP 2023516886 A5 JP2023516886 A5 JP 2023516886A5 JP 2022546698 A JP2022546698 A JP 2022546698A JP 2022546698 A JP2022546698 A JP 2022546698A JP 2023516886 A5 JP2023516886 A5 JP 2023516886A5
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JP
Japan
Prior art keywords
alkyl
hydrogen
substituted
independently
optionally substituted
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
JP2022546698A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023516886A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/016481 external-priority patent/WO2021158707A1/en
Publication of JP2023516886A publication Critical patent/JP2023516886A/ja
Publication of JP2023516886A5 publication Critical patent/JP2023516886A5/ja
Pending legal-status Critical Current

Links

JP2022546698A 2020-02-03 2021-02-03 遺伝性疾患の処置のための方法および化合物 Pending JP2023516886A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062969644P 2020-02-03 2020-02-03
US62/969,644 2020-02-03
US202163135476P 2021-01-08 2021-01-08
US63/135,476 2021-01-08
PCT/US2021/016481 WO2021158707A1 (en) 2020-02-03 2021-02-03 Methods and compounds for the treatment of genetic disease

Publications (2)

Publication Number Publication Date
JP2023516886A JP2023516886A (ja) 2023-04-21
JP2023516886A5 true JP2023516886A5 (https=) 2024-02-21

Family

ID=77200317

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022546698A Pending JP2023516886A (ja) 2020-02-03 2021-02-03 遺伝性疾患の処置のための方法および化合物

Country Status (7)

Country Link
US (2) US20230149550A1 (https=)
EP (1) EP4100404A4 (https=)
JP (1) JP2023516886A (https=)
CN (2) CN121045206A (https=)
CA (1) CA3169810A1 (https=)
TW (1) TW202142261A (https=)
WO (1) WO2021158707A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024502469A (ja) * 2021-01-08 2024-01-19 デザイン セラピューティクス，インク． フリードライヒ運動失調症を治療するための方法及び化合物
WO2023014793A1 (en) * 2021-08-04 2023-02-09 Design Therapeutics, Inc. Process for polyamide synthesis
KR20250016096A (ko) * 2022-04-01 2025-02-03 디자인 테라퓨틱스, 인크. 헌팅턴병을 조절하기 위한 방법 및 화합물
US20250295632A1 (en) * 2022-05-09 2025-09-25 Design Therapeutics, Inc. Compounds and methods for treating friedreich's ataxia
WO2025111500A1 (en) * 2023-11-22 2025-05-30 Ionis Pharmaceuticals, Inc. Modulation of gene expression
WO2025122762A1 (en) * 2023-12-08 2025-06-12 Design Therapeutics, Inc. Compounds and methods for treating sickle cell disease

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060270727A1 (en) * 2005-05-03 2006-11-30 Christian Melander Small molecule therapeutics and uses therefor
RU2720237C2 (ru) * 2013-11-18 2020-04-28 Форма Терапеутикс, Инк. Композиции, содержащие бензопиперазин, в качестве ингибиторов бромодоменов вет
GB201504314D0 (en) * 2015-03-13 2015-04-29 Univ Dundee Small molecules
US10517877B2 (en) * 2016-03-30 2019-12-31 Wisconsin Alumni Research Foundation Compounds and methods for modulating frataxin expression
US11759533B2 (en) * 2017-03-29 2023-09-19 Wisconsin Alumni Research Foundation Methods and compositions for modulating gene expression
US20190343873A1 (en) * 2018-05-14 2019-11-14 Kyoto University Sequence-selective gene expression regulators
EP4234550A3 (en) * 2018-05-22 2023-09-06 Design Therapeutics, Inc. Methods and compounds for the treatment of genetic disease
AU2019368245A1 (en) * 2018-10-23 2021-05-20 Wisconsin Alumni Research Foundation Next generation synthetic transcription factors

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