Cystinosis clinical trials at University of California Health
1 in progress, 0 open to eligible people
Sorry, in progress, not accepting new patients
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)
Our lead scientists for Cystinosis research studies include Stephanie Cherqui, Ph.D..