Use of metformin and analogs thereof to reduce RAN protein levels in the treatment of neurological disorders
US11903910B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Sep 26, 2018 |
| Grant date | Feb 20, 2024 |
| Priority date | — |
| Expiry date | Mar 21, 2039 |
Classification
- Technology area (CPC G)Physics
- CPC primaryG01N2800/28
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present disclosure provides the use of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) in treating a neurological disease associated with repeat expansions and/or RAN protein accumulation, reducing the level of one or more repeat associated non-ATG (RAN) proteins, and reducing the accumulation of RAN proteins in a subject and/or biological sample. Also provided is the use of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) in inhibiting RAN protein translation in a subject and in a biological sample (e.g., cells, tissue). Also provided in the present disclosure are pharmaceutical compositions, Skits, and uses of compounds of Formulae (I), (II), (III), (III-A), and (III-B) (e.g., metformin) for treating diseases associated with repeat expansions. Exemplary diseases associated with repeat expansions include, but are not limited to, C90RFf72 amyotrophic lateral sclerosis (ALS), or C90RFf72 frontotemporal dementia; myotonic dystrophy type 1 (DM1) and myotonic dystrophy type 2 (DM2); spinocerebellar ataxia; Huntington's disease; Fragile X Tremor Ataxia Syndrome (FXTAS); and Fragile XE syndrome (FRAXE).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.