Oral mHTT-Lowering Compounds for Huntington's Disease Progression
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Solution Overview
Problem
There are no approved disease-modifying treatments for Huntington's disease, leaving a high unmet need for medications that can slow disease progression.
Innovation Solution
Development of compounds represented by Formula (I′) or their pharmaceutically acceptable salts, which can lower mutant huntingtin (mHTT) protein levels in subjects, thereby treating or ameliorating Huntington's disease.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Quantity of substance
If RNA interference therapies (viral vectors or ASO infusions) are used to lower mHTT levels, then mHTT protein levels are reduced, but the treatment requires complex surgical delivery or repeated lumbar puncture procedures
Solution Approach 1:
The patent replaces complex mechanical delivery systems (viral vectors requiring surgical injection, ASO infusions requiring lumbar puncture) with a simple oral small molecule compound that can be administered through the gastrointestinal tract, eliminating the need for invasive procedures while achieving the same mHTT reduction effect
Solution Approach 2:
The small molecule compound is designed to be orally administered and automatically absorbed through the gastrointestinal tract, utilizing the body's natural absorption mechanisms rather than requiring external mechanical delivery systems or invasive procedures
2Reliability
If no disease-modifying treatments are available, then current HD management is limited to symptomatic treatment, but this leaves high unmet need and does not slow disease progression
Solution Approach 1:
The patent changes the fundamental parameter of disease management by transitioning from symptomatic treatment to disease-modifying treatment through the small molecule compound that lowers mHTT levels, thereby slowing disease progression and expanding therapeutic options for HD patients
Data Source
AI summary
The present disclosure provides a compound of Formula (I′), or a pharmaceutically acceptable salt thereof and its use in, e.g. treating a condition, disease, or disorder in which lowering mutant huntingtin protein (“mHTT”) in a subject is of therapeutic benefit, specifically in treating Huntington disease (“HD”). This disclosure also features a composition containing the same as well as methods of using and making the same.