Award
Food and Drug Administration 5R01FD008174-02
Gene Therapy for LCA5-Associated Inherited Retinal Degeneration: Extension to Pediatric Cohorts
Recipient
University of Pennsylvania
Award Amount
$897,359.00
Ceiling
$897,359.00
Awarded
July 21, 2025
Identifier
5R01FD008174-02
This award funds a clinical trial and research project to test gene therapy for LCA5-associated inherited retinal degeneration in pediatric patients, focusing on adolescents and very young children, to improve vision and develop new outcome measures.
Description
A Phase I/II dose escalation clinical trial to evaluate gene augmentation therapy for LCA5 in pediatric patients aged 13 to 18 years and develop novel ocular outcome measures for younger children with LCA5. The trial includes doses of 1.0x10^10, 3.0x10^10, and 1.0x10^11 vg/eye with a 3-year follow-up. The project aims to improve vision in pediatric populations with inherited retinal diseases and develop outcome measures for future trials.