Key Takeaways

  • First patients in France have received the experimental gene therapy GS010 (Lumevoq) through an early access program, offering a potential option for individuals with ND4-related LHON 

  • Alongside early access, the REVISE clinical trial is progressing, with multiple patients enrolled, providing two pathways in France for patients to access the investigational treatment.

The 15-20 National Hospital in Paris and GenSight Biologics announced that the first patients have been treated under France’s Named Patient Early Access Program (Autorisation d’Accès Compassionnel, AAC) for the investigational gene therapy GS010, also known as Lumevoq.

The initial treatments were administered on March 19 at the Paris-based hospital. Additional patients are expected to receive the therapy in the coming weeks as part of the program, which was recently authorized by the French medicines agency (ANSM).

GS010 is a gene therapy currently in clinical development for Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that causes sudden and often irreversible vision loss. The therapy specifically targets patients with a mutation in the ND4 mitochondrial gene—the most common and severe form of LHON.

“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 hospital. “Our clinical teams are fully mobilized to care for these patients, whether through the REVISE study or the AAC program.”

The AAC program enables patients with serious or rare diseases to access investigational treatments outside of clinical trials when no approved therapies are available. In this case, it offers a potential option for patients facing progressive vision loss with limited alternatives.

In parallel with the early access rollout, GenSight is advancing its REVISE clinical study, a dose-ranging trial evaluating GS010. The study, authorized in December 2025 by both the ANSM and an ethics committee, treated its first patient in February 2026. Since then, three additional patients have been enrolled.

“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.”