GenSight Biologics S.A.’s GS010/LUMEVOQ rollout in France and Israel has done more than expand access—it may be redefining how gene therapies are delivered in ophthalmology. As the company prepares for bilateral administration under early access programs for Leber Hereditary Optic Neuropathy (LHON), it is stepping into a growing shift across the field: a move away
GenSight Biologics S.A.’s early access rollout of GS010/LUMEVOQ in France and Israel has drawn new attention to how regulators are quietly repurposing compassionate use frameworks into strategic regulatory sandboxes for advanced therapies. As rare disease gene therapies edge closer to commercial viability, a patchwork of early access programs across key jurisdictions is beginning to influence
GenSight Biologics S.A. has received approval from Israel’s Ministry of Health to provide early access treatment with its investigational gene therapy GS010/LUMEVOQ (lenadogene nolparvovec) for individual patients diagnosed with ND4-related Leber Hereditary Optic Neuropathy (LHON). This authorization permits bilateral intravitreal injections under compassionate use protocols, with treatments expected to begin in the first quarter of
France grants compassionate use access to GenSight Biologics’ LUMEVOQ for LHON. Explore what this decision changes and what risks remain.
Fondazione Telethon has received regulatory approval from the United States Food and Drug Administration for Waskyra (etuvetidigene autotemcel), an ex vivo gene therapy designed to treat Wiskott-Aldrich syndrome, a rare X-linked primary immunodeficiency. This approval makes Waskyra the first gene therapy for this condition cleared for use in the United States and solidifies Fondazione Telethon’s