GenSight Biologics has unveiled promising findings from recent real-world data gathered through early access programs (EAP). These data reinforce the efficacy and safety of LUMEVOQ® in patients with Leber Hereditary Optic Neuropathy (LHON) stemming from the ND4 mutation (ND4-LHON), corroborating outcomes observed in clinical trials. Notably, patients who received bilateral injections exhibited a remarkable average gain of +23 ETDRS letters in best-corrected visual acuity (BCVA) from nadir, surpassing the gains seen in patients with unilateral injections.

These results, presented at the 2024 annual meeting of the North American Neuro-Ophthalmology Society (NANOS), indicate significant visual improvement among a majority of treated patients. Dr. Chiara La Morgia, MD, PhD, emphasized the consistency and robustness of these findings, underlining their relevance for patients grappling with severe vision impairment. With a more comprehensive dataset compared to earlier releases, these analyses offer a definitive perspective on the sustained upward trajectory of mean visual acuity post-treatment, further supporting the viability of lenadogene nolparvovec as a therapeutic option.

Additionally, safety assessments aligned with previous clinical studies, affirming the favorable safety profile of LUMEVOQ® with comparable rates of intraocular inflammation events. These promising outcomes herald a new chapter in the treatment landscape for LHON patients, promising hope and tangible benefits for those afflicted with this debilitating condition.