Gene therapy injection shows promise for rare eye disease
NCT ID NCT02652767
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Centre National Hospitalier d'Ophtalmologie des Quinze-VingtCentre National Hospitalier d'Ophtalmologie des Quinze-Vingt
Paris, Paris, France
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Department of Neurology, University of Munich, Friedrich-Baur-Institute
Munich, 80336, Germany
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Department of Ophthalmology, Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Doheny Eye Center, University of California, Los Angeles
Los Angeles, California, 91105, United States
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IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria
Bologna, Bologna, Italy
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Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD, United Kingdom
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Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States