“Completing the dosing of the first and only gene therapy trial for cystinosis represents a major milestone for a patient community living with a devastating genetic disease. Unmet medical needs impact the lives of patients and their family members every day,” said Stephanie Cherqui, Ph.D., lead study investigator and associate professor of Pediatrics at UCSD. “To date, the results from the trial show the potential of this investigational gene therapy to stabilize or reduce the impact of cystinosis on different tissues throughout the body with a one-time dose.”
