Our Story

In 2012, a group of scientists founded GenSight Biologics with the ambition of turning advances in gene therapy into transformative treatments for patients with inherited blinding diseases.

Since then, GenSight Biologics has reached major milestones. Our lead product candidates have progressed from scientific concept to clinical development, with proof of concept in humans, bringing us closer to achieving our founding ambition.

Our Goal

The eye is one of the most promising targets for gene therapy, and home to rare inherited diseases for which therapeutic solutions remain limited. Today we are advancing candidate treatments for neurodegenerative ophthalmic diseases such as Leber Hereditary Optic Neuropathy (LHON) and Retinitis Pigmentosa (RP).

Beyond ophthalmology, our technology platforms have the potential to address other disorders of the central nervous system.

The GenSight Biologics Journey

The story of GenSight Biologics is marked by scientific, clinical and financial milestones, each one bringing us closer to delivering treatments for patients facing high unmet needs.

April 2012

Founding of GenSight Biologics

GenSight Biologics is founded in Paris by a group of scientists, driven by a combination of scientific curiosity and a desire to realize the therapeutic potential of gene therapy in blinding diseases.

February 2014

First in-Human Trial for GS010

The first of 15 patients is enrolled in REVEAL, the first-in-human Phase 1/2 trial of GS010 for ND4-LHON, which evaluates the safety and efficacy of a single gene therapy injection.

February 2016

First Phase 3 Trials for GS010

The first patients are enrolled in RESCUE (39 patients) and REVERSE (37 patients), two Phase 3 trials of GS010 conducted in parallel to test the efficacy of a one-time unilateral injection.

July 2016

IPO

GenSight Biologics lists on Euronext Paris, raising approximately €40 million to fund late-stage development of GS010.

March 2018

Third Phase 3 Trial for GS010

The first of 98 patients is enrolled in REFLECT, a third Phase 3 trial for GS010, evaluating the efficacy and safety of bilateral injections and, together with RESCUE and REVERSE, bringing enrollment in the Phase 3 studies to 174 treated patients.

October 2018

First in-Human Trial for GS030

The first patient is enrolled in PIONEER, the first-in-human Phase 1/2 trial of GS030, a novel optogenetic treatment combining a gene therapy with a wearable light-stimulation headset for blind patients in late-stage RP.

December 2019

First Early Access Patient

The French medicines agency ANSM grants the first named-patient Temporary Authorization for Use (ATU) for GS010, allowing a patient to be treated outside a clinical trial. Early access treatment later expands to patients in Italy, the U.K. and the U.S.

April 2023

Withdrawal of EMA Application for LUMEVOQ®

GenSight Biologics voluntarily withdraws its European Marketing Authorization Application for GS010/LUMEVOQ®, following an unexpected contralateral effect in the Phase 3 trials that complicated the demonstration of efficacy. After further discussions with the EMA and FDA, the Company agrees to conduct a new Phase 3 trial, RECOVER, informed by the learnings from the earlier trials.

December 2023

New Leadership

Laurence Rodriguez is appointed Chief Executive Officer. With more than 30 years of experience in life sciences and rare diseases, she is tasked with leading the Company as it enters the next phase of its development. Jan Eryk Umiastowski joins as Chief Financial Officer in 2024.

December 2025

REVISE Study and Early Access Programs

The French medicines agency ANSM approves REVISE, a Phase 2 dose-ranging study evaluating GS010 in France. At the same time, the agency authorizes a named patient early access program (AAC) for GS010, following earlier early access authorizations in the U.S. and Israel.

What's Ahead

What’s Ahead – Writing the Next Chapter

GenSight Biologics is preparing to initiate the pivotal global Phase 3 RECOVER trial of GS010 while continuing to explore options for early access treatments in various countries to respond to unmet therapeutic needs.

Who We Are

Meet the team shaping GenSight Biologics’ next chapter.

Management

Our management team sets the strategy, oversees operations and carries the scientific vision of GenSight Biologics. Together, we are advancing gene therapy approaches for patients with rare and serious inherited diseases.

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