Gene therapy OCU400 shows promise for improving vision in retinitis pigmentosa and Leber congenital amaurosis

OCU400, a gene modifier therapy, demonstrates safety and potential clinical benefits in improving vision for patients with retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) associated with specific genetic mutations, according to updated results from a Phase 1/2 trial presented at AAO 2024.

The study found that 75% of patients with RP experienced visual improvements or stabilization after treatment, supporting OCU400’s effectiveness and its gene-agnostic mechanism of action.

This multicenter, open-label trial involved 18 adult participants with specific biallelic mutations, who received a unilateral subretinal injection of OCU400 (AAV5-hNR2E3) at varying doses in their more affected eye.

The study assessed safety and efficacy over a 52-week period. Results indicated that OCU400 was generally safe and well tolerated, with serious adverse effects reported in 2 participants from the high-dose group that were not attributed to the therapy. The presentation noted that 75% of patients with RP experienced improvements in visual acuity and mobility test scores, with many showing either stabilization or enhancement of vision in the treated eye.

Reference
Lam BL, et al. Safety and Efficacy of OCU400 Gene Modifier Therapy for Retinitis Pigmentosa: Phase 1/2 Study Updates. Poster presented at: American Academy of Ophthalmology Annual Meeting; October 2024; Chicago.