Drug discovery for gastrointestinal motility disorders using hPSC-derived enteric ganglioids
Grant Award Details
Grant Type:
Grant Number:
DISC2-15119
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Cell Line Generation:
Award Value:
$1,954,367
Status:
Active
Grant Application Details
Application Title:
Drug discovery for gastrointestinal motility disorders using hPSC-derived enteric ganglioids
Public Abstract:
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
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
Statement of Benefit to California:
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.