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[HEALTH] · United States, France, Switzerland, United Kingdom, Germany · 13 sources

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Optogenetic therapy restores rudimentary vision in clinical trial

An international clinical trial has demonstrated that optogenetic therapy can restore rudimentary vision in patients with advanced retinitis pigmentosa. The study, involving ten participants from France, the United States, the United Kingdom, and Switzerland, was published in The New England Journal of Medicine.

The treatment involves a single injection of a viral vector carrying the ChrimsonR light-sensitive protein into the eye. This process modifies surviving retinal ganglion cells, enabling them to respond to light. To facilitate vision, patients wear specialized goggles equipped with cameras that convert visual scenes into pulses of amber light, which then stimulate the modified cells.

Results showed that seven of the ten participants became more sensitive to light, with six achieving clinically meaningful improvements. These patients were able to perform tasks such as detecting, locating, or reaching toward objects, and some could follow lines or find doorways. However, the therapy did not restore normal sight, and no participants were able to read or recognize faces.

One severe adverse event was reported immediately following the injection, but it resolved within minutes. The research, which aligns with the 2026 Nobel Prize in Physiology or Medicine awarded for breakthroughs in optogenetics, provides a foundation for developing more precise visual restoration therapies.

Entities

Botond Roska · GenSight Biologics · Institute of Molecular and Clinical Ophthalmology Basel · José-Alain Sahel · Life Biosciences · New England Journal of Medicine · Retinitis Pigmentosa · University Hospital Schleswig-Holstein · University of Basel · University of Pittsburgh

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