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.
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.
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a single eye injection restore sight in genetic blindness?
- A sharper look at the retina: new imaging technology may spot eye disease earlier
- Stem cell eye transplant aims to halt blindness from rare genetic disease
- Gene Therapy's lasting effects on vision tracked in Long-Term study
- Could stem cells restore sight in damaged eyes?
- Gene therapy injection aims to restore sight in rare blindness