First Patient Treated in REVISE Gene Therapy Study of GS010/LUMEVOQ® for ND4-LHON
The 15-20 National Hospital in Paris and GenSight Biologics have treated the first patient in the REVISE dose-ranging study of GS010/LUMEVOQ®, a gene therapy candidate for ND4-associated Leber Hereditary Optic Neuropathy (LHON). The ANSM-approved study will enroll 14 patients in France and further strengthens the long-standing clinical partnership between the two institutions in advancing treatments for rare inherited optic neuropathies.
The 15-20 National Hospital (l’Hôpital national des 15-20) in Paris and GenSight Biologics announced the treatment of the first patient enrolled in the REVISE dose-ranging study. The open-label, single center study aims to enroll 14 patients in France. With REVISE now underway, the hospital is currently the only institution in Europe with a clinical study involving GS010/LUMEVOQ®, GenSight Biologics’ candidate gene therapy being developed as a treatment for Leber Hereditary Optic Neuropathy (LHON) caused by a mutated ND4 mitochondrial gene. The hospital is also the sole institution in Europe authorized to perform named patient early access (AAC) treatments with GS010.
“As a leading hospital specialized in the treatment of vision disorders, 15-20 National Hospital is committed to supporting the most promising scientific advances rigorously and in the service of patients affected by rare diseases,” said Nicolas Péju, Chief Executive Officer of the 15-20 National Hospital. “The start of the REVISE study testifies to this drive and to the trust given to our clinical and research teams.”
The REVISE study will investigate two doses of GS010 for the treatment of ND4-LHON. The study was requested by the French medicines agency ANSM (Agence nationale de sécurité du medicaments et des produits de santé) during the review of the application for a named patient early access program (AAC) for GS010 and was authorized in December 2025.
Reviewed in parallel with the REVISE study, the AAC program received the authorization from the ANSM later in December 2025 and is potentially open to patients from outside of France depending on regulatory requirements in their home country. In accordance with regulations, the first named patient requests for AAC were submitted to the ANSM last week. The named patient requests, each of which resulted from a multidisciplinary consultation, will be individually evaluated by the agency. Enrollment into REVISE is prioritized for patients eligible for both programs.
“We at GenSight are gratified that two avenues are now available for patients to be treated with GS010 in France,” said Dr. Magali Taiel, Chief Medical Officer of GenSight Biologics. “The unmet medical need among ND4-LHON patients and the urgency to treat the condition continue to animate our efforts to advance the clinical development of the gene therapy, including our push to begin a new Phase III clinical study by the end of this year.”
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.
GS010/LUMEVOQ® has not received marketing authorization in any jurisdiction and is not commercially available.
GenSight Biologics is a clinical-stage biopharmaceutical company focused on the development of innovative gene and optogenetic therapies for retinal neurodegenerative diseases and disorders of the central nervous system. Prof. Botond Roska and Prof. José-Alain Sahel are scientific co-founders of GenSight Biologics. Prof. Roska serves as Chairman of the Scientific Advisory Board, contributing his pioneering expertise in retinal neuroscience and optogenetics to the company’s scientific strategy. Prof. Sahel, Vice-Chairman of the Scientific Advisory Board and a member of the Board of Directors, brings extensive clinical and translational experience in retinal diseases and plays a key role in guiding the company’s research and development efforts.
Quelle: GenSight Biologics