Gene therapy hope for kids and adults with rare Blindness-Causing mutation

NCT ID NCT07063030

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

Summary

This early-phase study tests a gene therapy called LX107 for people with a rare inherited eye disease caused by mutations in the AIPL1 gene. The treatment is given as an injection under the retina to try to improve vision and slow disease progression. The study will include 13 adults and children aged 4 and older, and will focus on safety and whether the therapy helps with mobility in different lighting conditions.

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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.

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Conditions

The condition(s) this trial relates to.

AIPL1-related retinopathy inherited retinal dystrophy

As listed by the trial registrant

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

Contacts and locations

Locations

  • Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

    RECRUITING

    Shanghai, China

    Contact Phone: •••-•••-•••• Email: •••••@•••••