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Isabelle SCARABIN

BUSINESS DEVELOPMENT DIRECTOR

Gene Therapy, retinal degenerative diseases, prevent sight loss, restore vision, blindness, ophthalmology, rare diseases, orphan market, AAV, LHON, Retinitis Pigmentosa

GenSight Biologics is a clinical-stage biopharma company focused on developing gene therapies for retinal neurodegenerative diseases and central nervous system disorders. Our 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.

Our lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Positive results at 72W of REVERSE Phase III trial show clinically meaningful improvement of +15 ETDRS letters in visual acuity of treated eyes.
Our second product, GS030, combines gene therapy with optogenetics and is in Phase I/II clinical trial PIONE.