Gene therapy injection shows promise for rare eye disease

NCT ID NCT02652767

Completed Disease control Sponsor: GenSight Biologics Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tested a single injection of GS010 gene therapy in 39 people with Leber Hereditary Optic Neuropathy (LHON) who had vision loss for 6 months or less. The treatment aims to improve vision by delivering a working copy of the ND4 gene to the eye. Results measured changes in visual acuity over 48 weeks.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: GS010 (Lenadogene Nolparvovec) - a gene therapy injected into the eye
What this could lead to: If it works, this could point toward a treatment that slows or partially reverses vision loss in people with LHON.
What could go wrong: This is a small, early-phase trial with only 39 participants. The treatment may not improve vision for everyone, and there are risks from the injection itself, such as eye inflammation or infection.

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Conditions

The condition(s) this trial relates to.

Eye Diseases, Hereditary eye disorder hereditary disease hereditary optic atrophy inborn mitochondrial metabolism disorder Leber hereditary optic neuropathy nervous system disorder neurodegenerative disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

OPTIC, ATROPHY, HEREDITARY, LEBER

Contacts and locations

Locations

Centre National Hospitalier d'Ophtalmologie des Quinze-VingtCentre National Hospitalier d'Ophtalmologie des Quinze-Vingt

Paris, Paris, France
Department of Neurology, University of Munich, Friedrich-Baur-Institute

Munich, 80336, Germany
Department of Ophthalmology, Emory University School of Medicine

Atlanta, Georgia, 30322, United States
Doheny Eye Center, University of California, Los Angeles

Los Angeles, California, 91105, United States
IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria

Bologna, Bologna, Italy
Moorfields Eye Hospital NHS Foundation Trust

London, EC1V 2PD, United Kingdom
Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

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Gene therapy shows promise for rare eye disease causing blindness