Optogenetics Platform

GenSight Biologics’ proprietary optogenetics platform is designed to bypass the loss of photoreceptors by combining gene therapy with a light-stimulation device. This technology, currently being applied to vision loss, may open a new therapeutic pathway for conditions where cellular function is lost.

Programming Surviving Retinal Cells to Respond to Light

The Challenge:

Many blinding diseases are caused by the degeneration and death of photoreceptor cells. These specialized cells detect light and convert it into electrical signals for the brain, enabling sight. Once photoreceptors are lost, the eye loses its normal way of capturing visual information.

The Solution:

GenSight Biologics’ optogenetics platform is designed to compensate for the loss of photoreceptors by conferring light sensitivity on surviving retinal cells. Our gene therapy induces the expression of a light-sensitive protein in retinal ganglion cells, while our optronic device captures the visual environment and projects light at the wavelength and intensity needed to activate these cells and restore a functional visual response.

GS030: Our Lead Optogenetics Program

GS030 is GenSight Biologics’ optogenetics-based product candidate targeting Retinitis Pigmentosa (RP), the world’s most prevalent inherited retinal disease. GS030 aims to restore vision regardless of the 100+ genes that can cause the condition.

GS030 has not been granted marketing authorization in any country and is not commercially available.

The excerpts below are drawn from third-party sources and reflect the views of their authors alone.

“Therapeutic approaches aiming to stimulate these residual cells, independent of the underlying genetic cause, could potentially restore visual function in patients with advanced vision loss, and benefit many more patients than therapies directed at the specific gene implicated in each disorder.”

Potential Applications

Retinal Diseases

Central Nervous System Disorders

Neurodegenerative Disorders

Pipeline

Learn more about the clinical development of GS030,
our optogenetics-based product candidate

A New Chapter

Founded in 2012 to realize the potential of gene therapy, GenSight Biologics has grown into a clinical-stage company advancing multiple programs. In 2024, under a new management team, the company entered a new phase and is now accelerating its clinical and regulatory activities.

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GenSight Biologics S.A.
74 rue du Faubourg Saint-Antoine
75012 Paris
France