Biopharmaceutical company GenSight Biologics (Euronext Paris:SIGHT) on Thursday announced regulatory authorisations for the use of its investigational gene therapy, GS010/LUMEVOQ, under Individual Patient Expanded Access in the United States.

The US Food and Drug Administration (FDA) and the Institutional Review Board (IRB) of the University of Pittsburgh School of Medicine approved treatment for one patient scheduled for November 2025.

This marks the first regulatory authorisation for GS010/LUMEVOQ since GenSight's withdrawal of its European marketing application in 2023. The therapy targets Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that causes rapid and irreversible vision loss. It remains in Phase III development without marketing approval in any country.

GenSight continues to engage with the French medicines agency ANSM, which is reviewing an early access dossier and a proposed dose-ranging study related to a potential early access programme in France.

The company is also finalising a technology transfer to its new manufacturing partner, Catalent, expected to be completed by the end of the year, enabling production of new batches in 2026. These efforts align with GenSight's broader strategy to ensure clinical supply, advance regulatory discussions, and expand patient access to GS010/LUMEVOQ globally.