Cystinosis clinical trials at UCSD
1 research study open to eligible people
Showing trials for
Stem Cell Gene Therapy for Cystinosis
open to eligible people ages 18 years and up
This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)
La Jolla, California
Our lead scientists for Cystinosis research studies include Stephanie Cherqui, Ph.D..