AR124589A1 - Métodos y compuestos para tratar la ataxia de friedreich - Google Patents
Métodos y compuestos para tratar la ataxia de friedreichInfo
- Publication number
- AR124589A1 AR124589A1 ARP220100032A ARP220100032A AR124589A1 AR 124589 A1 AR124589 A1 AR 124589A1 AR P220100032 A ARP220100032 A AR P220100032A AR P220100032 A ARP220100032 A AR P220100032A AR 124589 A1 AR124589 A1 AR 124589A1
- Authority
- AR
- Argentina
- Prior art keywords
- terminus
- alkyl
- optionally substituted
- hydrogen
- independently
- 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
- 229910052739 hydrogen Inorganic materials 0.000 abstract 5
- 239000001257 hydrogen Substances 0.000 abstract 5
- 230000027455 binding Effects 0.000 abstract 4
- 125000004435 hydrogen atom Chemical class [H]* 0.000 abstract 4
- 108090000623 proteins and genes Proteins 0.000 abstract 4
- 125000004169 (C1-C6) alkyl group Chemical group 0.000 abstract 3
- 239000002773 nucleotide Substances 0.000 abstract 3
- 125000003729 nucleotide group Chemical group 0.000 abstract 3
- 230000004568 DNA-binding Effects 0.000 abstract 2
- 125000004404 heteroalkyl group Chemical group 0.000 abstract 2
- 125000005647 linker group Chemical group 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 230000001105 regulatory effect Effects 0.000 abstract 2
- 238000013518 transcription Methods 0.000 abstract 2
- 230000035897 transcription Effects 0.000 abstract 2
- 125000003837 (C1-C20) alkyl group Chemical group 0.000 abstract 1
- 125000003161 (C1-C6) alkylene group Chemical group 0.000 abstract 1
- 125000003341 7 membered heterocyclic group Chemical group 0.000 abstract 1
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 abstract 1
- 108091008771 Rev-ErbA proteins Proteins 0.000 abstract 1
- 125000000217 alkyl group Chemical group 0.000 abstract 1
- 150000001413 amino acids Chemical class 0.000 abstract 1
- 125000004429 atom Chemical group 0.000 abstract 1
- 125000002993 cycloalkylene group Chemical group 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 125000005549 heteroarylene group Chemical group 0.000 abstract 1
- 150000003839 salts Chemical class 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
- 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
-
- 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
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)
- Medicinal Preparation (AREA)
- Polyethers (AREA)
- Indole Compounds (AREA)
- Plural Heterocyclic Compounds (AREA)
- Photoreceptors In Electrophotography (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 una primera terminal, una segunda terminal y una cadena principal oligomérica, donde a) la primera terminal comprende una porción de unión al DNA capaz de unirse de forma no covalente a una secuencia de repetición de trinucleótidos GAA; b) la segunda terminal comprende una porción de unión a proteínas que se une a una molécula reguladora que modula una expresión de un gen que comprende la secuencia de repetición de nucleótidos GAA; y c) la cadena principal oligomérica comprende un conector entre la primera terminal y la segunda terminal. Reivindicación 1: Una molécula moduladora de la transcripción que tiene un primer terminal, un segundo terminal y una cadena principal oligomérica, caracterizada porque: a) el primer terminal comprende una porción de unión a DNA capaz de unirse de manera no covalente a una secuencia de repetición de nucleótidos GAA, donde el primer terminal tiene la estructura de fórmula (1) o una sal farmacéuticamente aceptable de esta, donde; m₁ es 1 - 4; n₁ es 0 - 2; cada Y¹, Y², Y³ e Y⁴ es independientemente CH o N; cada Z¹, Z², Z³ y Z⁴ es independientemente O, S o NR¹D; cada L³ es alquileno C₁₋₆ opcionalmente sustituido, cicloalquileno C₃₋₇, heterocicleno de 3 a 7 miembros, o heteroarileno de 5 a 6 miembros; cada R³⁰ es hidrógeno o un alquilo C₁₋₆; o cada R³⁰ y L³ se unen con los átomos a los cuales están adheridos para formar un anillo heterocíclico de 4 a 7 miembros; W¹ es hidrógeno, un alquilo C₁₋₆ opcionalmente sustituido, -NR¹E-C(O)-NR¹ER¹F, -C(O)-NR¹ER¹F o (AA)₁₋₁₀; W² es hidrógeno, un alquilo C₁₋₆ opcionalmente sustituido, -C(O)-NR¹ER¹F o (AA)₁₋₁₀; cada R¹D y R¹E es independientemente hidrógeno, alquilo C₁₋₅₀ opcionalmente sustituido, heteroalquilo C₁₋₅₀ o PEG₁₋₅₀; R¹F es hidrógeno, alquilo C₁₋₂₀ opcionalmente sustituido, heteroalquilo C₁₋₂₀, PEG₁₋₂₀ o uno o más AA; y cada AA es independientemente un aminoácido de origen natural; b) el segundo terminal comprende una porción de unión a proteínas que se une a una molécula reguladora que modula la expresión de un gen que comprende la secuencia repetida de nucleótidos GAA; y c) la cadena principal oligomérica que comprende un conector entre el primer terminal y el segundo terminal.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163135427P | 2021-01-08 | 2021-01-08 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR124589A1 true AR124589A1 (es) | 2023-04-12 |
Family
ID=82358799
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220100032A AR124589A1 (es) | 2021-01-08 | 2022-01-07 | Métodos 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 |
|---|---|---|---|---|
| WO2023133284A2 (en) * | 2022-01-06 | 2023-07-13 | Design Therapeutics, Inc. | Compounds and methods for treating friedreich's ataxia |
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 WO PCT/US2022/011560 patent/WO2022150555A2/en active Application Filing
- 2022-01-07 CN CN202280018641.2A patent/CN117241805A/zh active Pending
- 2022-01-07 AU AU2022206424A patent/AU2022206424A1/en active Pending
- 2022-01-07 CA CA3204523A patent/CA3204523A1/en active Pending
- 2022-01-07 AR ARP220100032A patent/AR124589A1/es unknown
- 2022-01-07 US US18/260,549 patent/US20240124491A1/en active Pending
- 2022-01-07 TW TW111100817A patent/TW202241892A/zh unknown
- 2022-01-07 EP EP22737158.0A patent/EP4274583A2/en active Pending
- 2022-01-07 JP JP2023541510A patent/JP2024502469A/ja active Pending
- 2022-01-07 MX MX2023008153A patent/MX2023008153A/es unknown
-
2023
- 2023-07-09 IL IL304322A patent/IL304322A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| WO2022150555A2 (en) | 2022-07-14 |
| MX2023008153A (es) | 2023-09-21 |
| IL304322A (en) | 2023-09-01 |
| CA3204523A1 (en) | 2022-07-14 |
| TW202241892A (zh) | 2022-11-01 |
| US20240124491A1 (en) | 2024-04-18 |
| EP4274583A2 (en) | 2023-11-15 |
| CN117241805A (zh) | 2023-12-15 |
| AU2022206424A1 (en) | 2023-08-24 |
| JP2024502469A (ja) | 2024-01-19 |
| WO2022150555A3 (en) | 2022-12-22 |
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