50
Participants
Start Date
November 14, 2022
Primary Completion Date
November 30, 2036
Study Completion Date
November 30, 2036
Safety and Efficacy Assessments
Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.
University of California San Diego, La Jolla
Collaborators (2)
California Institute for Regenerative Medicine (CIRM)
OTHER
Cystinosis Research Foundation
OTHER
Novartis Pharmaceuticals
INDUSTRY
Stephanie Cherqui
OTHER