GS010 (lenadogene nolparvovec)
Leber Hereditary Optic Neuropathy (LHON) due to ND4 mutation
Phase 3Clinical development completing; Long-term follow-up ongoing
Key Facts
Indication
Leber Hereditary Optic Neuropathy (LHON) due to ND4 mutation
Phase
Phase 3
Status
Clinical development completing; Long-term follow-up ongoing
Company
About GenSight Biologics
GenSight Biologics is a Paris-based gene therapy company founded in 2012, focused on developing and commercializing novel therapies for retinal degenerative diseases and central nervous system disorders. Its core technology platforms combine gene therapy with a proprietary Mitochondrial Targeting Sequence (MTS) and optogenetics. The company has two product candidates in clinical trials, has received prestigious scientific recognition, and is preparing for the potential commercialization of its lead asset for LHON.
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