Gene therapy FT-001 shows promise in treating RPE65-related retinal disease

FT-001, a gene therapy targeting RPE65-related inherited retinal disease, showed promising safety and efficacy, with significant improvements in visual function and retinal sensitivity across all tested doses, according to a poster presented at AAO 2024 on a Phase 1/2 clinical trial.

FT-001 utilizes a rAAV2 vector to deliver a codon-optimized version of the human RPE65 gene to restore visual function.

The open-label study tested 3 dosage levels (1.5E10, 7.5E10, and 15E10 vg/eye) across 2 phases. Nine patients were treated in Phase 1 and monitored for up to 1 year. Phase 2 involved 5 patients, whose previously untreated eyes received the therapy, with follow-ups extending to Day 84.

Results showed significant improvements in visual function and retinal sensitivity starting from Day 28 and lasting throughout the observation period. In addition, the therapy demonstrated favorable safety across all dose groups, with no dose-limiting toxicities reported. In Phase 2, patients experienced enhanced mobility test scores and improved responses in the full-field stimulus test.

Reference
Sui R, et al. Safety and Efficacy Results of an Open-Label, Dose-Escalation and Dose-Expansion Phase 1/2 Study of FT-001 in Subjects With RPE65-IRD. Poster presented at: American Academy of Ophthalmology Annual Meeting; October 2024; Chicago.