GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a prominent biopharmaceutical company specializing in innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, has disclosed recent prospective real-world data from early access programs (EAP) affirming the efficacy of LUMEVOQ® in patients with Leber Hereditary Optic Neuropathy from the ND4 mutation (ND4-LHON), consistent with clinical trial findings.
One year post-treatment, bilaterally injected patients exhibited an average gain from nadir1 in best-corrected visual acuity (BCVA) of +23 ETDRS letters, surpassing the 18-letter average gain in patients with unilateral injection. Notably, these improvements exceeded the conventional threshold for clinical meaningfulness. Safety profiles of gene therapy remain favorable across both patient groups.
Presented at the 2024 annual meeting of the North American Neuro-Ophthalmology Society (NANOS), these findings will also be showcased at major medical conferences in Europe and the US. Dr. Chiara La Morgia, MD, PhD, highlighted the consistent, significant visual improvement observed with lenadogene nolparvovec, offering hope for patients grappling with LHON.
These analyses, drawing from a more robust data pool than previous releases, provide a clearer picture of the sustained improvement in mean visual acuity post-treatment.
Responder analyses demonstrate that the majority of patients experienced clinically meaningful BCVA improvement, with on-chart vision attained nine months post-injection. Safety results align with clinical studies, with intraocular inflammation events comparable in frequency, intensity, and location.
Laurence Rodriguez, CEO of GenSight, expresses the team’s enthusiasm to restore early access availability for eligible patients by Q3 2024. LUMEVOQ® (GS010; lenadogene nolparvovec) was offered through EAPs in select countries, with plans to resume early access in France under the Autorisation de l’accès compassionnel (AAC) program.
GenSight Biologics S.A. is committed to developing innovative gene therapies leveraging the Mitochondrial Targeting Sequence (MTS) and optogenetics platforms to address retinal neurodegenerative diseases. LHON, a rare mitochondrial genetic disease, remains a key focus for the company, with LUMEVOQ® representing a promising treatment option leveraging gene therapy-based approaches.
