GenSight Biologics announced that it has received regulatory authorization in the United States to provide its investigational gene therapy, GS010 (LUMEVOQ), to individual patients under expanded access protocols, according to the company. The authorizations were granted by the US Food and Drug Administration (FDA) and enable the treatment of patients affected by Leber Hereditary Optic Neuropathy (LHON), a rare genetic disease that causes vision loss.
According to the company, the expanded access authorization represents a key step in making LUMEVOQ available to patients outside of clinical trials. The treatment uses an adeno-associated viral vector to deliver a functional copy of the ND4 gene to retinal cells, aiming to restore or preserve vision in individuals with LHON caused by ND4 mutations.
GenSight Biologics said that LUMEVOQ has already been administered to several patients in Europe under compassionate use programs while awaiting formal regulatory approval. The company claims that the US authorization reflects growing recognition of the therapy’s potential benefits and supports continued efforts to make the treatment more widely accessible.
According to the company, GenSight continues to engage with regulatory authorities in both the United States and Europe to advance the approval process for LUMEVOQ and ensure that eligible patients can receive treatment through appropriate clinical and expanded access pathways.
