Research Objective

Our goal is to use stem cell models to identify a small molecule drug candidate for GI motility disorders.

Impact

The candidate will be used for severe gastrointestinal motility disorders including chronic constipation, achalasia and gastroparesis.

Major Proposed Activities

• Assessment of candidate compounds on stem cell derived models from diverse genetic backgrounds
• Evaluation of effectiveness on mouse tissue
• Understanding the underlying therapeutic effect of the drug candidates
• Identification of the best performing drug candidate for follow-up assessment in animal models
• Evaluation of safety and dosing regimen in animal models
• Testing the effectiveness of drug candidate in animal models

Gastrointestinal motility disorders are highly common and affect a large patient population in California. Currently available treatment options are limited and ineffective leading to reduced quality of life and increased burden of care.