Gene therapy for inherited blindness passes early safety check

NCT ID NCT02064569

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

Summary

This study tested a gene therapy called GS010 in 19 adults with Leber Hereditary Optic Neuropathy (LHON), a rare inherited eye disease that causes rapid vision loss. The main goal was to see if the treatment is safe and tolerable at different doses. The therapy uses a harmless virus to deliver a working gene to the eye, aiming to stop or slow vision loss.

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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 โ€” we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

Leber hereditary optic neuropathy

As listed by the trial registrant

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

Contacts and locations

Locations

  • CIC du CHNO DES QUINZE-VINGTS

    Paris, 75012, France