Gene therapy to restore vision in children with Stargardt disease

Plain-English translation of NCT07063251 on ClinicalTrials.gov ↗ · Source last updated August 2025 · Translation generated June 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This trial is testing HG005, a new gene therapy designed to treat Stargardt disease type 1 (STGD1), a rare inherited eye condition that causes progressive vision loss. HG005 works by delivering a healthy copy of the ABCA4 gene to the retina, which helps restore the eye's natural ability to clear toxic waste buildup that damages vision. The goal is to slow or stop vision loss in children and teenagers with this condition.

Stargardt disease has no approved treatment options, and it often leads to significant vision loss or blindness during childhood or adolescence. This study exists to determine whether the medication is safe and whether it can help protect remaining vision by restoring the eye's natural cleanup process.

Do you qualify?

You likely qualify if…

you are between 6 and 17 years old
you have been diagnosed with Stargardt disease type 1 caused by mutations in the ABCA4 gene (biallelic mutations — one from each parent)
both of your eyes have clear signs of macular damage consistent with Stargardt disease
your vision falls within specific criteria measured by a standard eye chart
you are willing to use contraception during the study if applicable

You likely don't qualify if…

you have active inflammation or infection inside or around either eye
you have had an eye infection in the past 2 weeks
you have a history of HIV or hepatitis A, B, or C
you have received any previous gene therapy or cell therapy
you have had eye surgery in the study eye within 3 months before enrollment or are taking medications that might interfere with eye healing

What participation looks like

If you're enrolled, you will receive the treatment through a procedure that delivers the gene therapy to your retina. You'll be part of a small group (up to 6 participants) divided into different dose levels so doctors can carefully monitor safety and tolerability. You'll have multiple follow-up visits and eye examinations to track how your vision and eye health respond to the treatment over time.

AI-generated summary from trial data · Jun 3, 2026 · Not medical advice

Gene therapy to restore vision in children with Stargardt disease

What is this trial?

Do you qualify?

What participation looks like

Trial locations