Can stem cells restore vision in stargardt disease? a First-in-Human trial aims to find out

NCT ID NCT07734064

First seen Jul 29, 2026 · Last updated Jul 30, 2026 · Updated 1 time

Summary

This early-phase trial tests a single injection of ASP2020, stem cells turned into retinal cells, placed under the retina in people with Stargardt disease or similar inherited macular dystrophies. The main goal is to check safety and tolerability, while also looking for signs that the cells replace damaged tissue and improve central vision. The study includes adults, teenagers, and children, with doses tested in stages.

What this could mean

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

Active substance
ASP2020, human stem cells turned into retinal cells, given as a single injection under the retina
What this could lead to
If safe and effective, this approach could restore central vision in people with Stargardt disease and similar inherited macular dystrophies.
What could go wrong
This is a very early, small trial testing safety first. Risks include immune reactions, abnormal cell growth, or no vision improvement. Surgery under general anesthesia carries its own risks.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

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