CA3176909A1 - Compositions and methods for the prevention and/or treatment of mitochondrial disease, including friedreich's ataxia - Google Patents
Compositions and methods for the prevention and/or treatment of mitochondrial disease, including friedreich's ataxiaInfo
- Publication number
- CA3176909A1 CA3176909A1 CA3176909A CA3176909A CA3176909A1 CA 3176909 A1 CA3176909 A1 CA 3176909A1 CA 3176909 A CA3176909 A CA 3176909A CA 3176909 A CA3176909 A CA 3176909A CA 3176909 A1 CA3176909 A1 CA 3176909A1
- Authority
- CA
- Canada
- Prior art keywords
- compound
- ch2ch3
- independently
- formula
- group
- 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
- C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
- C07F7/00—Compounds containing elements of Groups 4 or 14 of the Periodic Table
- C07F7/02—Silicon compounds
- C07F7/08—Compounds having one or more C—Si linkages
- C07F7/0803—Compounds with Si-C or Si-Si linkages
- C07F7/081—Compounds with Si-C or Si-Si linkages comprising at least one atom selected from the elements N, O, halogen, S, Se or Te
-
- 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
- A61K31/045—Hydroxy compounds, e.g. alcohols; Salts thereof, e.g. alcoholates
- A61K31/05—Phenols
-
- 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
- A61K31/12—Ketones
- A61K31/122—Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
-
- 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
- A61K31/695—Silicon compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
- A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
-
- 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
- A61P27/02—Ophthalmic agents
-
- 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
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C43/00—Ethers; Compounds having groups, groups or groups
- C07C43/02—Ethers
- C07C43/20—Ethers having an ether-oxygen atom bound to a carbon atom of a six-membered aromatic ring
- C07C43/23—Ethers having an ether-oxygen atom bound to a carbon atom of a six-membered aromatic ring containing hydroxy or O-metal groups
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C50/00—Quinones
- C07C50/02—Quinones with monocyclic quinoid structure
- C07C50/06—Quinones with monocyclic quinoid structure with unsaturation outside the quinoid structure
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C50/00—Quinones
- C07C50/26—Quinones containing groups having oxygen atoms singly bound to carbon atoms
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C50/00—Quinones
- C07C50/26—Quinones containing groups having oxygen atoms singly bound to carbon atoms
- C07C50/28—Quinones containing groups having oxygen atoms singly bound to carbon atoms with monocyclic quinoid structure
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D317/00—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms
- C07D317/08—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3
- C07D317/44—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 ortho- or peri-condensed with carbocyclic rings or ring systems
- C07D317/46—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 ortho- or peri-condensed with carbocyclic rings or ring systems condensed with one six-membered ring
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D319/00—Heterocyclic compounds containing six-membered rings having two oxygen atoms as the only ring hetero atoms
- C07D319/10—1,4-Dioxanes; Hydrogenated 1,4-dioxanes
- C07D319/14—1,4-Dioxanes; Hydrogenated 1,4-dioxanes condensed with carbocyclic rings or ring systems
- C07D319/16—1,4-Dioxanes; Hydrogenated 1,4-dioxanes condensed with carbocyclic rings or ring systems condensed with one six-membered ring
- C07D319/18—Ethylenedioxybenzenes, not substituted on the hetero ring
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C2601/00—Systems containing only non-condensed rings
- C07C2601/12—Systems containing only non-condensed rings with a six-membered ring
- C07C2601/16—Systems containing only non-condensed rings with a six-membered ring the ring being unsaturated
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Life Sciences & Earth Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Engineering & Computer Science (AREA)
- Physical Education & Sports Medicine (AREA)
- Epidemiology (AREA)
- Ophthalmology & Optometry (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Diabetes (AREA)
- Neurology (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Rheumatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Hydrogenated Pyridines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063004639P | 2020-04-03 | 2020-04-03 | |
| US63/004,639 | 2020-04-03 | ||
| PCT/US2021/025558 WO2021202986A1 (en) | 2020-04-03 | 2021-04-02 | Compositions and methods for the prevention and/or treatment of mitochondrial disease, including friedreich's ataxia |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3176909A1 true CA3176909A1 (en) | 2021-10-07 |
Family
ID=75728888
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3176909A Pending CA3176909A1 (en) | 2020-04-03 | 2021-04-02 | Compositions and methods for the prevention and/or treatment of mitochondrial disease, including friedreich's ataxia |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US12037350B2 (https=) |
| EP (1) | EP4126836A1 (https=) |
| JP (1) | JP2023520462A (https=) |
| KR (1) | KR20230005855A (https=) |
| CN (4) | CN115996916B (https=) |
| AU (1) | AU2021246142A1 (https=) |
| CA (1) | CA3176909A1 (https=) |
| IL (1) | IL296985A (https=) |
| WO (1) | WO2021202986A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3222132A1 (en) | 2021-06-10 | 2022-12-15 | Stealth Biotherapeutics Inc. | Compounds, compositions and methods for the prevention and/or treatment of various mitochondrial diseases or disorders, including friedreich's ataxia |
| US11541116B1 (en) | 2022-01-07 | 2023-01-03 | Kojin Therapeutics, Inc. | Methods and compositions for inducing ferroptosis in vivo |
| WO2024263742A1 (en) * | 2023-06-22 | 2024-12-26 | Stealth Biotherapeutics Inc. | Combination therapy for the treatment of friedreich's ataxia |
| WO2025024247A1 (en) * | 2023-07-21 | 2025-01-30 | Stealth Biotherapeutics Inc. | Methods, compositions and uses related to the treatment and prevention of leigh's syndrome |
| WO2025101748A1 (en) | 2023-11-09 | 2025-05-15 | Stealth Biotherapeutics Inc. | Cyclo-hexa-2,5-diene-1,4-diones for use in the treatment of cardiomyopathy |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4522811A (en) | 1982-07-08 | 1985-06-11 | Syntex (U.S.A.) Inc. | Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides |
| JP3507486B2 (ja) | 1991-03-15 | 2004-03-15 | アムジエン・インコーポレーテツド | 顆粒球コロニー刺激因子の肺内投与 |
| US5858784A (en) | 1991-12-17 | 1999-01-12 | The Regents Of The University Of California | Expression of cloned genes in the lung by aerosol- and liposome-based delivery |
| US5716644A (en) | 1992-06-11 | 1998-02-10 | Alkermes, Inc. | Composition for sustained release of non-aggregated erythropoietin |
| US5674534A (en) | 1992-06-11 | 1997-10-07 | Alkermes, Inc. | Composition for sustained release of non-aggregated erythropoietin |
| US5451569A (en) | 1994-04-19 | 1995-09-19 | Hong Kong University Of Science And Technology R & D Corporation Limited | Pulmonary drug delivery system |
| US5989463A (en) | 1997-09-24 | 1999-11-23 | Alkermes Controlled Therapeutics, Inc. | Methods for fabricating polymer-based controlled release devices |
| CA2272971C (en) * | 1998-10-29 | 2008-01-15 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Quinone derivatives for treating or preventing diseases associated with iron overload |
| US6245740B1 (en) | 1998-12-23 | 2001-06-12 | Amgen Inc. | Polyol:oil suspensions for the sustained release of proteins |
| WO2005033093A1 (en) | 2003-09-19 | 2005-04-14 | Galileo Pharmaceuticals, Inc. | 7,8-bicycloalkyl-chroman derivatives |
| ES2619303T3 (es) | 2005-06-01 | 2017-06-26 | Edison Pharmaceuticals, Inc. | Productos terapéuticos redox activos para el tratamiento de enfermedades mitocondriales y otras afecciones y modulación de biomarcadores energéticos |
| SI1933821T1 (sl) | 2005-09-15 | 2020-11-30 | Ptc Therapeutics, Inc. | Repne variante redoks-aktivnih terapevtikov za zdravljenje mitohondrijskih bolezni in drugih bolezni ter modulacijo energijskih biooznačevalcev |
| EP2332534B1 (en) | 2006-02-22 | 2018-11-14 | Bioelectron Technology Corporation | Phenol and benzoquinone derivatives for use in the treatment of mitochondrial diseases and modulation of energy biomarkers |
| WO2007100652A2 (en) | 2006-02-22 | 2007-09-07 | Edison Pharmaceuticals, Inc. | Side chain variants of redox-active therapeutics for treatment of mitochondrial diseases and other conditions and modulation of energy biomarkers |
| WO2009111576A2 (en) | 2008-03-05 | 2009-09-11 | Edison Pharmaceuticals, Inc. | 2-SUBSTITUTED-p-QUINONE DERIVATIVES FOR TREATMENT OF OXIDATIVE STRESS DISEASES |
| JP5798481B2 (ja) | 2008-06-25 | 2015-10-21 | エジソン ファーマシューティカルズ， インコーポレイテッド | 酸化ストレス疾患を治療するための２−ヘテロシクリルアミノアルキル−（ｐ−キノン）誘導体 |
| MX363223B (es) | 2008-09-10 | 2019-03-15 | Bioelectron Tech Corp | Tratamiento de trastornos generalizados del desarrollo con terapeuticos con actividad redox. |
| ES2553557T3 (es) | 2008-10-28 | 2015-12-10 | Edison Pharmaceuticals, Inc. | Proceso para la producción de alfa-tocotrienol y derivados |
| EP2246048A1 (en) | 2009-04-30 | 2010-11-03 | Santhera Pharmaceuticals (Schweiz) AG | Quinone derivative 2,3-dimethoxy-5-methyl-6-(10-hydroxydecyl)-1,4-benzoquinone for the treatment of primary progressive multiple sclerosis |
| NO2470168T3 (https=) | 2009-08-26 | 2018-06-30 | ||
| SG187744A1 (en) | 2010-08-06 | 2013-03-28 | Edison Pharmaceuticals Inc | Treatment of mitochondrial diseases with naphthoquinones |
| DK2605769T3 (en) | 2010-08-16 | 2016-10-24 | Santhera Pharmaceuticals (Schweiz) Ag | Benzoquinonderivater for the treatment of mitochondrial diseases of the eye |
| EP2606022A2 (en) | 2010-08-16 | 2013-06-26 | Santhera Pharmaceuticals (Schweiz) AG | Novel benzoquinone derivatives and use thereof as modulators of mitochondrial function |
| US20120295985A1 (en) | 2010-11-19 | 2012-11-22 | Miller Guy M | Methods for improving blood glucose control |
| WO2012170773A1 (en) | 2011-06-08 | 2012-12-13 | Edison Pharmaceuticals, Inc. | Adjunctive therapy for the treatment of mitochondrial disorders with quinones and naphthoquinones |
| KR102137450B1 (ko) | 2012-07-12 | 2020-07-27 | 콘드리온 아이피 비.브이. | 미토콘드리아 질환을 치료하기 위한 크로마닐 유도체 |
| CA2883882A1 (en) | 2012-09-07 | 2014-03-13 | Ptc Therapeutics, Inc. | Benzoquinone derivatives for treating oxidative stress disorders |
| CN104870432B (zh) | 2012-11-16 | 2017-05-03 | 美国健康及人类服务部 | 作为溶酶体贮积症的修复剂的生育酚和生育醌衍生物 |
| US10189830B2 (en) | 2013-03-15 | 2019-01-29 | Bioelectron Technology Corporation | Alkyl-heteroaryl substituted quinone derivatives for treatment of oxidative stress disorders |
| WO2015183963A2 (en) | 2014-05-28 | 2015-12-03 | Stealth Peptides International, Inc. | Therapeutic compositions including redox-active parabenzoquinones and uses thereof |
| EP3377179B1 (en) | 2015-11-19 | 2021-04-07 | Concert Pharmaceuticals Inc. | Deuterated epi-743 |
| US10703701B2 (en) | 2015-12-17 | 2020-07-07 | Ptc Therapeutics, Inc. | Fluoroalkyl, fluoroalkoxy, phenoxy, heteroaryloxy, alkoxy, and amine 1,4-benzoquinone derivatives for treatment of oxidative stress disorders |
| CA3011293A1 (en) | 2016-01-12 | 2017-07-20 | Ptc Therapeutics, Inc. | Tocopherol and tocotrienol quinone derivatives for increasing thiosulfate levels or decreasing hydrogen sulfide levels |
| WO2018191732A1 (en) * | 2017-04-14 | 2018-10-18 | Bioelectron Technology Corporation | Methods and compositions for treatment of inflammation and oxidative stress |
| JP7266304B2 (ja) | 2017-04-21 | 2023-04-28 | ユニヴァーシティー オブ タスマニア | 治療化合物および方法 |
| ES2942631T3 (es) | 2017-12-15 | 2023-06-05 | Stealth Biotherapeutics Inc | Péptidos dirigidos a mitocondrias |
-
2021
- 2021-04-02 CN CN202180040296.8A patent/CN115996916B/zh active Active
- 2021-04-02 CA CA3176909A patent/CA3176909A1/en active Pending
- 2021-04-02 EP EP21722328.8A patent/EP4126836A1/en active Pending
- 2021-04-02 CN CN202511246906.6A patent/CN121085756A/zh active Pending
- 2021-04-02 CN CN202511246796.3A patent/CN121085765A/zh active Pending
- 2021-04-02 CN CN202510664780.8A patent/CN120554211A/zh active Pending
- 2021-04-02 IL IL296985A patent/IL296985A/en unknown
- 2021-04-02 AU AU2021246142A patent/AU2021246142A1/en active Pending
- 2021-04-02 KR KR1020227038265A patent/KR20230005855A/ko active Pending
- 2021-04-02 US US17/221,463 patent/US12037350B2/en active Active
- 2021-04-02 WO PCT/US2021/025558 patent/WO2021202986A1/en not_active Ceased
- 2021-04-02 JP JP2022559922A patent/JP2023520462A/ja active Pending
-
2024
- 2024-05-23 US US18/672,819 patent/US20240400588A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023520462A (ja) | 2023-05-17 |
| CN121085765A (zh) | 2025-12-09 |
| CN121085756A (zh) | 2025-12-09 |
| WO2021202986A1 (en) | 2021-10-07 |
| CN120554211A (zh) | 2025-08-29 |
| US20240400588A1 (en) | 2024-12-05 |
| US12037350B2 (en) | 2024-07-16 |
| IL296985A (en) | 2022-12-01 |
| AU2021246142A1 (en) | 2022-10-20 |
| CN115996916B (zh) | 2025-09-23 |
| US20210317146A1 (en) | 2021-10-14 |
| EP4126836A1 (en) | 2023-02-08 |
| KR20230005855A (ko) | 2023-01-10 |
| CN115996916A (zh) | 2023-04-21 |
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