Experimental eye injection aims to restore sight in rare blindness conditions

NCT ID NCT05282953

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study tested an experimental drug called KIO-301, given as an injection into the eye, in 32 adults with retinitis pigmentosa or choroideremia—two inherited conditions that cause progressive vision loss. The main goal was to check safety and tolerability at increasing doses. Researchers also looked for signs that the drug might improve vision.

What this could mean

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

Active substance

KIO-301 (an experimental drug injected into the eye)

What this could lead to

If it works, this could point toward a treatment that restores some vision in people with inherited retinal diseases.

What could go wrong

This is an early-phase trial with only 32 participants, so results may not apply to everyone. The drug is injected directly into the eye, which carries risks like inflammation or infection.

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.

choroideremia inherited retinal dystrophy retinitis pigmentosa

As listed by the trial registrant

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

Contacts and locations

Locations

  • Harley Eye Clinic

    North Adelaide, South Australia, 5006, Australia

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Save Sight Institute

    Sydney, New South Wales, 2001, Australia