Experimental gene injection aims to save sight in rare retinal disorder

NCT ID NCT07656753

First seen Jun 22, 2026 · Last updated Jun 22, 2026

Summary

This early-phase trial tests a gene therapy called PUMCH-E111 for a rare inherited retinal disease caused by RLBP1 mutations. Six adults aged 18-55 will receive a single injection into the eye at a low or high dose. The main goal is to check safety, but researchers will also measure changes in vision and light sensitivity.

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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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Contacts and locations

Study contacts

  • Contact

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

Locations

  • Peking Union Medical College Hospital

    RECRUITING

    Beijing, Beijing Municipality, 100730, China

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

    Contact

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

PUMCH-E111 gene therapy injection

What this could lead to

If successful, this could point toward a treatment that slows or partially reverses vision loss in people with RLBP1-related retinal dystrophy.

What could go wrong

This is a very early, small trial (6 people) focused on safety, not proof of effectiveness. The therapy may not improve vision and could cause side effects like eye inflammation or infection.

As listed by the trial registrant

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