Non-lysosomal glucosylceramidase inhibitors and uses thereof
By designing GBA2 inhibitor compounds with specific structures, the problems of non-selective and insufficient brain permeability of inhibitors in the prior art are solved, selective inhibition of GBA2 and increased brain drug concentration, and the therapeutic effect on neurological diseases and liver diseases is enhanced.
CN115803025BActive Publication Date: 2025-09-02ALECTOS THERAPEUTICS INC
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Patent Information
- Application Number
- CN202180046373.0
- Authority / Receiving Office
- CN · China
- Patent Type
- Patents(China)
- Current Assignee / Owner
- Priority Date
- 2020-11-06
- Filing Date
- 2021-05-06
- Publication Date
- 2025-09-02
- Estimated Expiration
- 2041-05-06
AI Technical Summary
Technical Problem
Existing GBA2 inhibitors have non-selective inhibitory effects on other enzymes, affecting the normal function of other enzymes, and lack specificity and brain permeability to GBA2, resulting in limited therapeutic effects.
Method used
A range of structurally specific compounds, including compounds of formula (I) to formula (Ik) and their pharmaceutically acceptable salts, were developed, designed to selectively inhibit the non-lysosomal glucoceramidease GBA2 and improve its permeability in the brain.
Benefits of technology
Selective inhibition of GBA2 is achieved, drug concentration in the brain is increased, therapeutic effect on neurological diseases and liver diseases is enhanced, and interference with other enzymes is reduced.
✦ Generated by Eureka AI based on patent content.
Abstract
The present invention provides a compound for inhibiting glucosylceramidase, a prodrug of the compound, and a pharmaceutical composition comprising the compound or the prodrug of the compound.
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