Who we are

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

GenSight Biologics’ 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. GenSight Biologics’ 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.

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.

Solid and advanced portfolio in ophthalmic gene therapy

A word from Bernard Gilly

CEO and Co Founder

Dear Madam, dear Sir,

On behalf of the entire team, it is with great pleasure that I thank you, old and new shareholders, for your trust and support, making our IPO on Euronext Paris a success and a new milestone in our development.

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. Our goal is to pursue the development of our product portfolio leveraging two core technology platforms: Mitochondrial Targeting Sequence (MTS), and optogenetics.

Our lead product candidate, GS010, which is at a more advanced stage of development than our other product candidates, based on our MTS technology, targets Leber’s hereditary optic neuropathy (LHON). LHON is a rare mitochondrial genetic disease characterized by the degeneration of retinal ganglion cells that results in rapid, severe and irreversible vision loss that can lead to complete blindness, mainly in adolescents and young adults.

GS010 is currently in Phase III in 7 clinical centers in Europe and the United States, where it has received Orphan Drug designation. Should the results of the ongoing clinical trials meet expectations, we could offer patients with a treatment that should allow them to gain autonomy and benefit from an improved quality of life.

Our second lead product candidate, GS030, based on the optogenetics technology, is currently at the preclinical stage. GS030 is intended to treat all forms of retinitis pigmentosa, and will subsequently target geographic atrophies, resulting from the late-stage form of age-related macular degeneration. GENSIGHT BIOLOGICS is expected to initiate a Phase I/II clinical trial during the second half of 2017.

Today we are delighted to see you involved in backing us in a new stage of our development. The €45.2 million raised will provide us with the necessary resources to finance the clinical development of our GS010 and GS030 candidates, aiming to offer an effective treatment to patients suffering from severe degenerative retinal diseases.

Bernard Gilly,Founder and CEO