GenSight Biologics is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. The company's pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. Its lead product candidate, LUMEVOQ® (GS010; lenadogene nolparvovec), has been submitted for marketing approval in Europe for the treatment of Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease affecting primarily teens and young adults that leads to irreversible blindness. The company's gene therapy-based approach aims to offer patients a sustainable functional visual recovery with a single treatment to each eye by intravitreal injection.

GenSight Biologics  is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics for retinitis pigmentosa, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible low vision and legal blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

Kreos Capital

Sofinnova Partners

3sbio

Heights Capital Management

European Investment Bank

Bpifrance

UPMC Enterprises

Invus

Arix Bioscience

Perceptive Advisors

Versant Ventures

Abingworth

GenSight Biologics

GenSight Biologics S.A., a clinical-stage biotechnology company, engages in the discovery, development, and commercialization of therapies for mitochondrial and neurodegenerative diseases of the eye and central nervous system. The company develops its products through gene therapy-based mitochondrial targeting sequence and optogenetics technology platforms. Its lead product candidates include LUMEVOQ (GS010), a recombinant AAV2-based gene therapy, which is in Phase III clinical trials for the treatment of leber hereditary optic neuropathy caused by a mutated ND4 gene; and GS030, which is in Phase I/II clinical trial for the treatment of retinitis pigmentosa, as well as in preclinical stage to treat dry age-related macular degeneration. The company also develops products that are in preclinical stage targeting ophthalmic and neurodegenerative diseases. GenSight Biologics S.A. was incorporated in 2012 and is headquartered in Paris, France.

GenSight Biologics S.A.

COM:GENSIGHT-BIOLOGICS

GenSight Biologics S.A.

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