GS010 (Lenadogene Nolparvovec / Lumevoq)
Leber Hereditary Optic Neuropathy (LHON) due to ND4 mutation
Phase 3Clinical Development Completing
Key Facts
Indication
Leber Hereditary Optic Neuropathy (LHON) due to ND4 mutation
Phase
Phase 3
Status
Clinical Development Completing
Company
About GenSight Biologics
GenSight Biologics is a Paris-based gene therapy company focused on creating transformative treatments for severe inherited retinal and neurodegenerative diseases. Its mission is to translate advanced science into long-lasting, single-administration cures, with its most advanced candidate, Lumevoq (lenadogene nolparvovec), having completed Phase III trials for LHON. The company's strategy leverages two proprietary technology platforms—Mitochondrial Targeting Sequence (MTS) and an optogenetics system—to address unmet medical needs in ophthalmology and beyond, operating with a lean, experienced team to advance its pipeline.
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