Gene therapy shows promise in Long-Term study for rare blindness

NCT ID NCT03406104

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 62 people with Leber Hereditary Optic Neuropathy (LHON), a rare inherited eye disease that causes vision loss, for up to 5 years after they received a single gene therapy treatment called GS010. The goal was to see if the treatment remained safe and if any vision improvements lasted over time. Researchers tracked eye-related side effects and measured changes in visual sharpness.

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Conditions

The condition(s) this trial relates to.

Eye Diseases, Hereditary eye disorder hereditary disease 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.

LEBER HEREDITARY OPTIC NEUROPATHY

Contacts and locations

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Locations

CHNO Les Quinze Vingts

Paris, 75012, France
Doheny Eye Center UCLA

Pasadena, California, 91105, United States
Emory University Hospital

Atlanta, Georgia, 30322, United States
LMU Klinikum der Universität München / Friedrich-Baur-Institut

Munich, 80336, Germany
Moorfields Eye Hospital

London, Greater London, EC1V 2PD, United Kingdom
Ospedale Bellaria

Bologna, 40139, Italy
Wills Eye Institute

Philadelphia, Pennsylvania, 19107, United States

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