GenSight Biologics is a clinical-stage biotechnology company focused on discovering and developing therapies for mitochondrial and neurodegenerative eye diseases, with potential future applications in the central nervous system. The company integrates two core technology platforms: a mitochondrial targeting sequence (MTS) and optogenetics to preserve or restore vision. Its lead product candidate, GS010, is a gene therapy targeting Leber’s Hereditary Optic Neuropathy (LHON) that uses a proprietary MTS technology. GS010 has received orphan drug designation in both the United States and the European Union. GenSight completed a Phase I/II trial of GS010 and expected to begin pivotal Phase III trials in LHON in the fourth quarter of 2015. The company announced a Series B financing to advance its product pipeline and support further clinical development of GS010. GenSight Biologics is a Paris, France–based biopharmaceutical company focused on developing ophthalmic therapeutics via gene therapy. The company closed a €32M Series A financing to advance its pipeline. GenSight intends to use the capital to develop a gene replacement therapy for Leber’s hereditary optic neuropathy (LHON) and an optogenetic therapy for retinitis pigmentosa (RP). Its lead product was expected to enter the clinic in 2013 in LHON patients. The company was co-founded by CEO Bernard Gilly alongside academic founders including Professor José-Alain Sahel, Jean Bennett, Connie Cepko, Ernst Bamberg, Luk Vandenberghe and Serge Picaud. The financing and experienced scientific co-founders position the company to progress clinical development in ophthalmic gene therapies.

Total raised

$78M

Funding rounds

2

Latest round

Series B

Latest activity

Jul 2015