PE20241730A1 - Compuestos y metodos para tratar la ataxia de friedreich - Google Patents
Compuestos y metodos para tratar la ataxia de friedreichInfo
- Publication number
- PE20241730A1 PE20241730A1 PE2024001532A PE2024001532A PE20241730A1 PE 20241730 A1 PE20241730 A1 PE 20241730A1 PE 2024001532 A PE2024001532 A PE 2024001532A PE 2024001532 A PE2024001532 A PE 2024001532A PE 20241730 A1 PE20241730 A1 PE 20241730A1
- Authority
- PE
- Peru
- Prior art keywords
- terminus
- ataxia
- compounds
- methods
- fxn
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 3
- 208000024412 Friedreich ataxia Diseases 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 230000027455 binding Effects 0.000 abstract 2
- 230000014509 gene expression Effects 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 230000004568 DNA-binding Effects 0.000 abstract 1
- 102000003869 Frataxin Human genes 0.000 abstract 1
- 108090000217 Frataxin Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
- 238000013518 transcription Methods 0.000 abstract 1
- 230000035897 transcription Effects 0.000 abstract 1
Classifications
-
- 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
-
- 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
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
- A61K31/437—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
- A61K47/595—Polyamides, e.g. nylon
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
- A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
- A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic Table
- C07F9/02—Phosphorus compounds
- C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
- C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Biochemistry (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263297090P | 2022-01-06 | 2022-01-06 | |
| US202263382854P | 2022-11-08 | 2022-11-08 | |
| PCT/US2023/010331 WO2023133284A2 (en) | 2022-01-06 | 2023-01-06 | Compounds and methods for treating friedreich's ataxia |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20241730A1 true PE20241730A1 (es) | 2024-08-19 |
Family
ID=87074158
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2024001532A PE20241730A1 (es) | 2022-01-06 | 2023-01-06 | Compuestos y metodos para tratar la ataxia de friedreich |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20250109132A1 (https=) |
| EP (1) | EP4460302A4 (https=) |
| JP (1) | JP2025502064A (https=) |
| KR (1) | KR20240138079A (https=) |
| AU (1) | AU2023205889A1 (https=) |
| CA (1) | CA3246468A1 (https=) |
| CL (1) | CL2024002029A1 (https=) |
| CO (1) | CO2024010651A2 (https=) |
| CR (1) | CR20240321A (https=) |
| IL (1) | IL314006A (https=) |
| MX (1) | MX2024008474A (https=) |
| PE (1) | PE20241730A1 (https=) |
| TW (1) | TW202334148A (https=) |
| WO (1) | WO2023133284A2 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2025076181A1 (en) * | 2023-10-03 | 2025-04-10 | Design Therapeutics, Inc. | Methods and compounds for modulating huntington's disease |
| WO2025076219A1 (en) * | 2023-10-03 | 2025-04-10 | Design Therapeutics, Inc. | Methods and compounds for modulating huntington's disease |
| WO2025076236A1 (en) * | 2023-10-03 | 2025-04-10 | Design Therapeutics, Inc. | Methods and compounds for modulating huntington's disease |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2013097052A1 (en) * | 2011-12-30 | 2013-07-04 | Abbott Laboratories | Bromodomain inhibitors |
| CN106068266A (zh) * | 2013-10-11 | 2016-11-02 | 千叶县 | 以癌症驱动基因的基因突变为靶点进行烷基化的新型烷基化剂 |
| US11759533B2 (en) * | 2017-03-29 | 2023-09-19 | Wisconsin Alumni Research Foundation | Methods and compositions for modulating gene expression |
| WO2020253711A1 (zh) * | 2019-06-17 | 2020-12-24 | 中国科学院上海药物研究所 | 一种吡咯并吡啶酮类化合物、其制备方法、其组合物和用途 |
| KR102677015B1 (ko) * | 2019-07-02 | 2024-06-19 | 누베이션 바이오 인크. | Bet 억제제로서의 헤테로시클릭 화합물 |
| 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 |
-
2023
- 2023-01-06 JP JP2024540893A patent/JP2025502064A/ja active Pending
- 2023-01-06 US US18/726,567 patent/US20250109132A1/en active Pending
- 2023-01-06 TW TW112100613A patent/TW202334148A/zh unknown
- 2023-01-06 MX MX2024008474A patent/MX2024008474A/es unknown
- 2023-01-06 IL IL314006A patent/IL314006A/en unknown
- 2023-01-06 PE PE2024001532A patent/PE20241730A1/es unknown
- 2023-01-06 WO PCT/US2023/010331 patent/WO2023133284A2/en not_active Ceased
- 2023-01-06 AU AU2023205889A patent/AU2023205889A1/en active Pending
- 2023-01-06 KR KR1020247025794A patent/KR20240138079A/ko active Pending
- 2023-01-06 CA CA3246468A patent/CA3246468A1/en active Pending
- 2023-01-06 EP EP23737637.1A patent/EP4460302A4/en active Pending
- 2023-01-06 CR CR20240321A patent/CR20240321A/es unknown
-
2024
- 2024-07-03 CL CL2024002029A patent/CL2024002029A1/es unknown
- 2024-08-02 CO CONC2024/0010651A patent/CO2024010651A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2023133284A3 (en) | 2023-08-24 |
| CO2024010651A2 (es) | 2024-08-08 |
| CA3246468A1 (en) | 2023-07-13 |
| AU2023205889A1 (en) | 2024-08-15 |
| CR20240321A (es) | 2024-10-16 |
| EP4460302A2 (en) | 2024-11-13 |
| CL2024002029A1 (es) | 2024-11-04 |
| JP2025502064A (ja) | 2025-01-24 |
| KR20240138079A (ko) | 2024-09-20 |
| MX2024008474A (es) | 2024-07-12 |
| TW202334148A (zh) | 2023-09-01 |
| US20250109132A1 (en) | 2025-04-03 |
| IL314006A (en) | 2024-08-01 |
| EP4460302A4 (en) | 2025-12-24 |
| WO2023133284A2 (en) | 2023-07-13 |
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