Optogénétique : restauration de la vision avec MCO‑010

In March 2025, Nanoscope Therapeutics published results in Molecular Therapy from the first clinical trials using a synthetic opsin (MCO‑010) delivered via an AAV2 vector in four blind patients with ABCA4-related RP. After 52 weeks, participants showed significant improvements in visual acuity, shape discrimination, and mobility without major adverse effects. The program has received Fast Track and Orphan Drug Designation from the FDA and is in the final stages before a BLA submission (RESTORE trial, NCT04945772).