CA3201756A1 - Methods and compounds for modulating myotonic dystrophy 1 - Google Patents
Methods and compounds for modulating myotonic dystrophy 1Info
- Publication number
- CA3201756A1 CA3201756A1 CA3201756A CA3201756A CA3201756A1 CA 3201756 A1 CA3201756 A1 CA 3201756A1 CA 3201756 A CA3201756 A CA 3201756A CA 3201756 A CA3201756 A CA 3201756A CA 3201756 A1 CA3201756 A1 CA 3201756A1
- Authority
- CA
- Canada
- Prior art keywords
- optionally substituted
- solvate
- pharmaceutically acceptable
- acceptable salt
- formula
- 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
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/001—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof by chemical synthesis
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
- C07D403/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
- A61K31/4164—1,3-Diazoles
- A61K31/4178—1,3-Diazoles not condensed 1,3-diazoles and containing further heterocyclic rings, e.g. pilocarpine, nitrofurantoin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D401/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
- C07D401/14—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
- C07D405/14—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
- C07D417/14—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing three or more hetero rings
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D471/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
- C07D471/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
- C07D471/04—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
- C07D487/04—Ortho-condensed systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Gastroenterology & Hepatology (AREA)
- Physical Education & Sports Medicine (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Epidemiology (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Polyesters Or Polycarbonates (AREA)
- Excavating Of Shafts Or Tunnels (AREA)
- Medicinal Preparation (AREA)
- Plural Heterocyclic Compounds (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063124592P | 2020-12-11 | 2020-12-11 | |
| US63/124,592 | 2020-12-11 | ||
| PCT/US2021/062974 WO2022126000A1 (en) | 2020-12-11 | 2021-12-11 | Methods and compounds for modulating myotonic dystrophy 1 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3201756A1 true CA3201756A1 (en) | 2022-06-16 |
Family
ID=81974018
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3201756A Pending CA3201756A1 (en) | 2020-12-11 | 2021-12-11 | Methods and compounds for modulating myotonic dystrophy 1 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240166693A1 (https=) |
| EP (1) | EP4259133A4 (https=) |
| JP (1) | JP2023554317A (https=) |
| CN (1) | CN117279636A (https=) |
| AU (1) | AU2021396454A1 (https=) |
| CA (1) | CA3201756A1 (https=) |
| WO (1) | WO2022126000A1 (https=) |
Families Citing this family (5)
| 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 |
| US20250120928A1 (en) * | 2021-07-14 | 2025-04-17 | University Of Maryland, Baltimore | Suberoylanilide hydroxamic acid (saha) drugs, conjugates, and nanoparticles, and methods of use thereof |
| KR20250016096A (ko) * | 2022-04-01 | 2025-02-03 | 디자인 테라퓨틱스, 인크. | 헌팅턴병을 조절하기 위한 방법 및 화합물 |
| US11976066B1 (en) | 2023-10-23 | 2024-05-07 | King Faisal University | Substituted 7-amino-3-(substituted benzoyl)indolizine-1-carboxylates as anti-tubercular agents |
| GB202401731D0 (en) * | 2024-02-08 | 2024-03-27 | Univ Strathclyde | Polyamides |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN1260006A (zh) * | 1996-02-26 | 2000-07-12 | 加利福尼亚州技术学院 | 双链dna和杂环低聚物之间复合物的形成 |
| US6506906B1 (en) * | 1996-02-26 | 2003-01-14 | California Institute Of Technology | Preparation and use of bifunctional molecules having DNA sequence binding specificity |
| US20060270727A1 (en) * | 2005-05-03 | 2006-11-30 | Christian Melander | Small molecule therapeutics and uses therefor |
| GB0722088D0 (en) * | 2007-11-09 | 2007-12-19 | Spirogen Ltd | Pyrrolobenzodiazepines |
| WO2010001933A1 (ja) * | 2008-07-01 | 2010-01-07 | 学校法人日本大学 | 標的遺伝子特異的ヒストン修飾制御剤 |
| US10517877B2 (en) * | 2016-03-30 | 2019-12-31 | Wisconsin Alumni Research Foundation | Compounds and methods for modulating frataxin expression |
| US20190343873A1 (en) * | 2018-05-14 | 2019-11-14 | Kyoto University | Sequence-selective gene expression regulators |
| EP4234549A1 (en) * | 2018-05-22 | 2023-08-30 | Design Therapeutics, Inc. | Methods and compounds for the treatment of genetic disease |
-
2021
- 2021-12-11 WO PCT/US2021/062974 patent/WO2022126000A1/en not_active Ceased
- 2021-12-11 EP EP21904532.5A patent/EP4259133A4/en active Pending
- 2021-12-11 AU AU2021396454A patent/AU2021396454A1/en active Pending
- 2021-12-11 CA CA3201756A patent/CA3201756A1/en active Pending
- 2021-12-11 JP JP2023535364A patent/JP2023554317A/ja active Pending
- 2021-12-11 US US18/256,864 patent/US20240166693A1/en active Pending
- 2021-12-11 CN CN202180093566.1A patent/CN117279636A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20240166693A1 (en) | 2024-05-23 |
| WO2022126000A1 (en) | 2022-06-16 |
| EP4259133A4 (en) | 2024-11-27 |
| EP4259133A1 (en) | 2023-10-18 |
| JP2023554317A (ja) | 2023-12-27 |
| CN117279636A (zh) | 2023-12-22 |
| AU2021396454A1 (en) | 2023-07-20 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| CA3201756A1 (en) | Methods and compounds for modulating myotonic dystrophy 1 | |
| US9783573B2 (en) | IAP antagonists | |
| EP3019517A1 (en) | Iap antagonists | |
| US20240050576A1 (en) | Methods and compounds for the treatment of genetic disease | |
| CN101677554A (zh) | 化合物 | |
| EP4234553A2 (en) | Methods and compounds for the treatment of genetic disease | |
| CA3218442A1 (en) | Targeted protein degradation using bifunctional compounds that bind ubiquitin ligase and target mcl-1 protein | |
| CA3204523A1 (en) | Methods and compounds for treating friedreich's ataxia | |
| WO2023133284A2 (en) | Compounds and methods for treating friedreich's ataxia | |
| WO2023192642A2 (en) | Methods and compounds for modulating huntington's disease | |
| EP4234549A1 (en) | Methods and compounds for the treatment of genetic disease | |
| EP4234550A2 (en) | Methods and compounds for the treatment of genetic disease | |
| EP4539839A1 (en) | Methods and compounds for modulating inherited genetic diseases | |
| HK40104905A (zh) | 用於调节强直性肌营养不良1型的方法和化合物 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD Year of fee payment: 3 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20241206 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT Effective date: 20241206 Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20241206 |
|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED Effective date: 20251203 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT Effective date: 20251203 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20251203 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD Year of fee payment: 4 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20251205 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20251205 |
|
| D00 | Search and/or examination requested or commenced |
Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT Effective date: 20260202 |
|
| D11 | Substantive examination requested |
Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT Effective date: 20260202 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT Effective date: 20260202 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED Effective date: 20260202 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20260202 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20260203 |