An international team led by José-Alain Sahel (University of Pittsburgh School of Medicine, Chair of the IOB Scientific Advisory Board) and Botond Roska at IOB has reported new results from the first clinical cohort of blind patients with advanced retinitis pigmentosa treated with optogenetic therapy.

The therapy combines gene therapy with light stimulating goggles to make surviving retinal ganglion cells responsive to light. 

Importantly, the treatment was safe in all 10 participants, which was the study’s primary endpoint. Six patients showed a clinically meaningful increase in light sensitivity, and several improved in tasks such as detecting or locating objects, finding doorways and following lines while wearing light-stimulating goggles. Brain recordings also detected visual signals in treated patients, and those who trained more with the goggles tended to perform better in object-detection tests.

While the treatment did not restore normal sight, the findings show that a degree of visual function can be recovered even at very advanced stages of retinal degeneration. The study builds on the team’s 2021 single-patient proof of concept and provides a foundation for further improving optogenetic therapies.

Scientific contact:

Botond Roska, Head of the Human Retinal and Central Visual Circuits Group

E-Mail: headoffice_roska@iob.ch

Illustrations:

Feature and Landing Page © IOB, V. Juvin, 2021, all rights reserved 

Header © IOB, S. Futterknecht, 2026 (image generated with AI)

Original Publication

Optogenetic Therapy for Restoring Aspects of Visual Function

José-Alain Sahel, Stefan Futterknecht, Isabelle Audo, Joseph N Martel, Shouyu Ling, Chloé Pagot, Angelo Arleo, Elise Boulanger-Scemama, Antonin Duret, Jean-Baptiste de Saint Aubert, Alexandre Delaux, Simona Degli Esposti, Cécilia Coen, Caroline de Montleau, Dana Aravich, William Smith, Cecile Hayem, Magali Taiel, Marlene Behrmann, Botond Roska

New England Journal of Medicine. 2026 October 8. 

doi: 10.1056/NEJMoa2602215

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