Pharmacological correctors of rhodopsin and uses thereof
Small molecule correctors, specifically compounds of various formulas, address the lack of treatment for RHO-adRP by preventing Rhodopsin misfolding and restoring its trafficking, effectively preserving vision in patients with this progressive blindness disease.
WO2025101780A1 Publication Date: 2025-05-15OCTANT INC
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Patent Information
- Application Number
- PCT/US2024/054963
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-10-28
- Filing Date
- 2024-11-07
- Publication Date
- 2025-05-15
AI Technical Summary
Technical Problem
There is currently no approved pharmacologic treatment for rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP), a progressive blindness disease caused by misfolding mutations of Rhodopsin.
Method used
Development of small molecule correctors, specifically compounds of Formula (I), (Ia), (II), (IIa), (III), (IIIa), (IV), and (IVa), or their pharmaceutically acceptable salts or solvates, which prevent or mitigate RHO misfolding, thereby restoring Rhodopsin trafficking and preventing photoreceptor cell death.
Benefits of technology
The proposed compounds effectively prevent RHO misfolding, restore proper trafficking of Rhodopsin, and thereby preserve vision by preventing photoreceptor cell death in patients with RHO-adRP.
✦ Generated by Eureka AI based on patent content.
Abstract
Described herein are pharmacologic correctors of rhodopsin, methods of making such compounds, pharmaceutical compositions and medicaments comprising such compounds, and methods of using such compounds in treating disease.
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