The 15-20 National Hospital and GenSight Biologics Announce the First Treatments in the French Named Patient Early Access Program (AAC) for GS010/LUMEVOQ®

Paris, France, March 20, 2026, 7:30 am CET – The 15-20 National Hospital (l’Hôpital national des 15-20) in Paris and GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the first patients have been treated in the French Named Patient Early Access Program (AAC) for GS010/LUMEVOQ^®.[1] The REVISE dose-ranging study, which is being conducted in parallel, has enrolled the next three patients.

First patients treated in the GS010 AAC program

Following the recent authorization of the first named patient requests, an initial group of patients in the GS010 AAC program were treated at the 15-20 National Hospital in Paris on March 19. More treatments are scheduled in the coming weeks.

GS010 is a candidate gene therapy in clinical development for the treatment of Leber Hereditary Optic Neuropathy (LHON) caused by a mutation in the ND4 mitochondrial gene. LHON patients suffer precipitous, usually irreversible vision loss caused by the rapid degeneration of their retinal ganglion cells (RGCs), cells that are essential to vision. Recognizing the urgency to treat the disease in the face of the progressive loss of these cells, the ANSM authorized, under the AAC program, the treatment of patients presenting with vision loss related to ND4-LHON. The ND4 mitochondrial mutation is the most common of the mutations that cause LHON and is associated with the worst visual prognosis among the three most prevalent mutations.

“The 15-20 National Hospital provides LHON patients with access to GS010 within a rigorous regulatory framework,” said Nicolas Péju, Chief Executive Officer of the 15-20 National Hospital. “Our clinical teams are fully mobilized to care for these patients, whether through the REVISE study or the AAC program.”  

REVISE study update

The REVISE dose-ranging study, authorized by the ANSM and the Ethics Committee in December 2025, is proceeding according to plan. The first patient was treated in February 2026, and three additional patients have since been enrolled in the study. French patients eligible for both the REVISE study and the AAC program are prioritized for the clinical study, in accordance with applicable regulations.

“The treatment of the first patients under the AAC program makes access to GS010 a reality for many LHON patients with the ND4 mutation, patients who would otherwise have no appropriate treatment option,” said Dr. Magali Taiel, Chief Medical Officer of GenSight Biologics. “With both the REVISE study and the AAC program running, two avenues have now become available in France for these patients.”

[1] GS010/LUMEVOQ^® has not received marketing authorization in any jurisdiction and is not commercially available.