GenSight Biologics Secures Funding to Advance AAV-Based Gene Therapies for Blinding Eye Diseases

Paris, France – July 17, 2025 – GenSight Biologics, a biopharmaceutical company specializing in gene therapies for retinal neurodegenerative and central nervous system disorders, has raised approximately EUR 500K in new funding. This capital injection aims to ensure operational continuity, finalize manufacturing transfer, and support preparations for anticipated compassionate use access in France, as well as the initiation of the Phase III RECOVER study.

Led by CEO Laurence Rodriguez, GenSight Biologics leverages two core technology platforms – the Mitochondrial Targeting Sequence (MTS) and optogenetics – with its AAV-based gene therapy approach to preserve or restore vision in patients with blinding retinal diseases. Their product candidates are designed for single intravitreal injections to offer long-lasting visual recovery.

GenSight’s lead product candidate, GS010 (lenadogene nolparvovec), is in Phase III development for Leber Hereditary Optic Neuropathy (LHON). LHON is a rare mitochondrial disease that causes irreversible blindness in teens and young adults. GS010 is specifically an AAV2-based gene therapy (rAAV2/2 vector) that delivers a functional copy of the ND4 gene, aiming to restore mitochondrial function.