Experimental gene injection targets dry AMD

NCT ID NCT03846193

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tested a gene therapy called GT005 for dry age-related macular degeneration (AMD), a leading cause of vision loss. The therapy delivers a working gene to the retina via a single injection. The study enrolled 56 people and focused on safety and dosing, but was terminated early. If it works, it could offer a one-time treatment to slow vision loss.

What this could mean

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

Active substance

GT005 (a gene therapy that delivers a working copy of the complement factor I gene via a single injection under the retina)

What this could lead to

If successful, this could lead to a one-time gene therapy that slows or stops vision loss in people with dry age-related macular degeneration.

What could go wrong

This was a very early (Phase 1/2) trial with only 56 participants, and it was terminated early. The therapy involves a surgical injection into the eye, which carries risks like infection or retinal damage.

Disclaimer Read more

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.

age-related macular degeneration dry age related macular degeneration eye disorder Geographic Atrophy macular degeneration primary anetoderma retinal degeneration retinal disorder

As listed by the trial registrant

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

Contacts and locations

Locations

  • Bristol Eye Hospital

    Bristol, United Kingdom

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242, United States

  • Manchester Eye Hospital

    Manchester, United Kingdom

  • Mid-Atlantic Retina

    Philadelphia, Pennsylvania, 19107, United States

  • Midwest Eye Institute

    Indianapolis, Indiana, 46290, United States

  • Moorfields Eye Hospital

    London, United Kingdom

  • Ophthalamic Consultants of Boston (OCB)

    Boston, Massachusetts, 02114, United States

  • Oxford University Hospital

    Oxford, United Kingdom

  • Pepose Vision Institute

    Chesterfield, Missouri, 63017, United States

  • Retina Clinic London

    London, W1G 7LA, United Kingdom

  • Sierra Eye Associates

    Reno, Nevada, 89502, United States

  • Sunderland Eye Infirmary

    Sunderland, United Kingdom

  • Wolfe Eye Clinic

    West Des Moines, Iowa, 50266, United States