GenSight Biologics Announces Regulatory Authorization for Early Access Treatment with GS010/LUMEVOQ® in Israel

Paris, France, December 22, 2025, 6:00 pm CET – 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 Israel’s Ministry of Health Pharmaceutical Division has authorized the use of the candidate gene therapy GS010/LUMEVOQ^®[1] for the early access treatment of individual patients in the country.

Early access treatment in Israel is potentially available to patients with a life‑threatening or seriously debilitating disease who cannot be properly treated with a registered and approved medicinal product in Israel or abroad and who cannot be included in an appropriate clinical trial. As in many countries, the decision to treat must be initiated by a patient’s physician, after which the treating hospital’s ethics committee (Helsinki Committee) and Ministry of Health must both approve treatment. The application for early access treatment includes the scientific rationale for the treatment and evidence from non-clinical and clinical studies as well as professional literature that support the benefit-risk assessment for the patient. The Company is working with SK-Pharma, its partner of choice in Israel, to ensure access to GS010/LUMEVOQ for patients with unmet medical needs in the country.

“We are very pleased that we are able to provide limited quantities of GS010/LUMEVOQ for early access treatment worldwide, after authorization by relevant competent authorities,” commented Laurence Rodriguez, Chief Executive Officer of GenSight Biologics. “At the same time, we are gratified that the evidence that we could make available, regarding the clinical data on GS010, could support the compassionate treatment application.”

“For now, the company is giving the highest priority to ensuring sufficient supply for the REVISE dose-ranging study and named patient Early Access Program (AAC) recently authorized in France,” explained Ms. Rodriguez. “These programs represent essential steps towards our ultimate goal of maximizing patient access to GS010/LUMEVOQ through full market authorizations in key markets of the world.”

Business Update

The Company is currently completing the technology transfer to its new manufacturing partner, Catalent, which is expected to be finalized by year-end 2025. The transition will enable the production of new batches in 2026 to fully address the projected clinical needs.

In parallel, the Company is preparing to resume discussions with global regulatory agencies to pave the way for the launch of the pivotal GS010/LUMEVOQ^® Phase III study RECOVER in H2 2026 and in anticipation of a submission in the UK.

The Company is also pursuing opportunities to out-license GS010 in markets outside the USA and Europe, while exploring paid Early Access Programs worldwide.

Financial Calendar 2026

Q4 2025 Cash position                                                                              January 8, 2026

EGM to renew financial authorization for future potential fundraising       January 28, 2026

FY 2025 Financial statement                                                                     March 26, 2026

Q1 2026 Cash position                                                                              April 7, 2026

AGM                                                                                                           May 19, 2026

Q2 2026 Cash position                                                                              July 8, 2026

First half year 2026 Financial Statement                                                   September 29, 2026

Q3 2026 Cash position                                                                              October 8, 2026

About Leber Hereditary Optic Neuropathy (LHON)

LHON is a rare, maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells, which results in precipitous and usually irreversible vision loss and typically leads to legal blindness. The ND4 mitochondrial mutation is the most common of the mutations that cause LHON and is associated with the worst prognosis among the leading mutations.

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