MX2023008153A - Metodos y compuestos para tratar la ataxia de friedreich. - Google Patents
Metodos y compuestos para tratar la ataxia de friedreich.Info
- Publication number
- MX2023008153A MX2023008153A MX2023008153A MX2023008153A MX2023008153A MX 2023008153 A MX2023008153 A MX 2023008153A MX 2023008153 A MX2023008153 A MX 2023008153A MX 2023008153 A MX2023008153 A MX 2023008153A MX 2023008153 A MX2023008153 A MX 2023008153A
- Authority
- MX
- Mexico
- Prior art keywords
- terminus
- compounds
- methods
- binding
- ataxia
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 208000024412 Friedreich ataxia Diseases 0.000 title 1
- 230000027455 binding Effects 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 230000004568 DNA-binding Effects 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
- 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
- C07D519/00—Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00
-
- 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
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- 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
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D495/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms
- C07D495/12—Heterocyclic compounds containing in the condensed system at least one hetero ring having sulfur atoms as the only ring hetero atoms in which the condensed system contains three hetero rings
- C07D495/14—Ortho-condensed systems
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
- C07D498/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
- C07D498/04—Ortho-condensed systems
Landscapes
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Life Sciences & Earth Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Psychology (AREA)
- Epidemiology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Indole Compounds (AREA)
- Plural Heterocyclic Compounds (AREA)
- Photoreceptors In Electrophotography (AREA)
- Medicinal Preparation (AREA)
- Polyethers (AREA)
Abstract
La presente divulgación se refiere a compuestos y métodos para modular la expresión de fxn, y tratar enfermedades y afecciones en las que fxn desempeña un papel activo. El compuesto puede ser una molécula moduladora de la transcripción que tiene un primer extremo, un segundo extremo y una estructura oligomérica, en donde: a) el primer extremo comprende un resto de unión a ADN capaz de unirse de forma no covalente a una secuencia de repetición de trinucleótidos GAA; b) el segundo extremo comprende un resto de unión a proteína que se une a una molécula reguladora que modula la expresión de un gen que comprende la secuencia de repetición de nucleótidos GAA; y c) la estructura oligomérica comprende un enlazador entre la primera extremo y la segunda extremo.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202163135427P | 2021-01-08 | 2021-01-08 | |
PCT/US2022/011560 WO2022150555A2 (en) | 2021-01-08 | 2022-01-07 | Methods and compounds for treating friedreich's ataxia |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2023008153A true MX2023008153A (es) | 2023-09-21 |
Family
ID=82358799
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2023008153A MX2023008153A (es) | 2021-01-08 | 2022-01-07 | Metodos y compuestos para tratar la ataxia de friedreich. |
Country Status (11)
Country | Link |
---|---|
US (1) | US20240124491A1 (es) |
EP (1) | EP4274583A2 (es) |
JP (1) | JP2024502469A (es) |
CN (1) | CN117241805A (es) |
AR (1) | AR124589A1 (es) |
AU (1) | AU2022206424A1 (es) |
CA (1) | CA3204523A1 (es) |
IL (1) | IL304322A (es) |
MX (1) | MX2023008153A (es) |
TW (1) | TW202241892A (es) |
WO (1) | WO2022150555A2 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
MX2024008474A (es) * | 2022-01-06 | 2024-07-12 | Design Therapeutics Inc | Compuestos y metodos para tratar la ataxia de friedreich. |
Family Cites Families (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3019342A1 (en) * | 2016-03-30 | 2017-10-05 | Wisconsin Alumni Research Foundation | Methods and compositions for modulating frataxin expression |
US20210283265A1 (en) * | 2018-04-20 | 2021-09-16 | Design Therapeutics, Inc. | Methods and compounds for the treatment of genetic disease |
-
2022
- 2022-01-07 AR ARP220100032A patent/AR124589A1/es unknown
- 2022-01-07 TW TW111100817A patent/TW202241892A/zh unknown
- 2022-01-07 MX MX2023008153A patent/MX2023008153A/es unknown
- 2022-01-07 CN CN202280018641.2A patent/CN117241805A/zh active Pending
- 2022-01-07 WO PCT/US2022/011560 patent/WO2022150555A2/en active Application Filing
- 2022-01-07 JP JP2023541510A patent/JP2024502469A/ja active Pending
- 2022-01-07 US US18/260,549 patent/US20240124491A1/en active Pending
- 2022-01-07 EP EP22737158.0A patent/EP4274583A2/en active Pending
- 2022-01-07 AU AU2022206424A patent/AU2022206424A1/en active Pending
- 2022-01-07 CA CA3204523A patent/CA3204523A1/en active Pending
-
2023
- 2023-07-09 IL IL304322A patent/IL304322A/en unknown
Also Published As
Publication number | Publication date |
---|---|
WO2022150555A2 (en) | 2022-07-14 |
WO2022150555A3 (en) | 2022-12-22 |
EP4274583A2 (en) | 2023-11-15 |
US20240124491A1 (en) | 2024-04-18 |
AR124589A1 (es) | 2023-04-12 |
CA3204523A1 (en) | 2022-07-14 |
TW202241892A (zh) | 2022-11-01 |
CN117241805A (zh) | 2023-12-15 |
IL304322A (en) | 2023-09-01 |
AU2022206424A1 (en) | 2023-08-24 |
AU2022206424A9 (en) | 2024-09-19 |
JP2024502469A (ja) | 2024-01-19 |
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