Promising ROd-cone DYstrophy Gene therapY

Summary

This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a mutation in the RHO, PDE6A, or PDE6B gene (Step 1), or in any RCD-causative gene (Step 2).

Timeline

Start

2023-04-12

Primary completion

2027-02

Completion

2031-02

Publications

• Clerin E, Yang Y, Pagan D, Achiedo S, Blond F, Millet-Puel G, Ait-Ali N, Harichane I, Degardin J, Cesar Q, Simonutti M, Bennett J, Zeitz C, Audo I, Cronin T, Leveillard T, Sahel JA. Combined Expression of hRdCVF and hRdCVFL Through AAV-Mediated Delivery for the Treatment of Retinitis Pigmentosa. Invest Ophthalmol Vis Sci. 2026 Mar 2;67(3):2. doi: 10.1167/iovs.67.3.2.
• Marie M, Churet L, Gautron AS, Farjo R, Mizuyoshi K, Stevenson V, Khabou H, Leveillard T, Sahel JA, Lorget F. Preclinical safety and biodistribution of SPVN06, a novel gene- and mutation-independent gene therapy for rod-cone dystrophies. Gene Ther. 2025 Aug 4. doi: 10.1038/s41434-025-00556-3. Online ahead of print.

Drugs

Indications