Gene therapy aims to restore sight in rare blindness condition
NCT ID NCT07406854
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tests a gene therapy called NR082 for Leber's hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. About 95 people aged 12 to 75 with a specific ND4 mutation will receive a single injection of the therapy or a sham procedure. The goal is to see if the treatment can improve eyesight over 52 weeks.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
NR082 (rAAV2-ND4) gene therapy
What this could lead to
If successful, this gene therapy could restore some vision in people with LHON caused by ND4 mutations, offering a targeted treatment for this rare inherited blindness.
What could go wrong
This is an early-phase trial with only 95 participants, so results may not apply to everyone. Gene therapy carries risks like eye inflammation or no improvement, and long-term effects are unknown.
Disclaimer
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the original study
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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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
Contacts and locations
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Locations
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Beijing Tongren Hospital, Capital Medical University
Beijing, Beijing Municipality, China