Gene therapy injection aims to save sight in rare genetic disease

NCT ID NCT07269665

Recruiting now Disease control Sponsor: Axovia Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Aug 05, 2026 · Updated 3 times

Summary

This early-stage trial tests a single injection of AXV-101 gene therapy into one eye of 12 children and teens (ages 4-17) with Bardet-Biedl syndrome type 1, a rare genetic condition that causes progressive vision loss. The main goal is to check safety and find the right dose, while comparing the treated eye to the untreated one over a year. If safe, it could pave the way for larger studies to see if it can slow or stop retinal degeneration.

What this could mean

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

Active substance: AXV-101 (gene therapy injected into the eye)
What this could lead to: If it works, this could slow or stop vision loss in people with BBS1, potentially preserving sight for years.
What could go wrong: This is a very early, small trial (12 people) focused on safety, not proof of effectiveness. Gene therapy in the eye carries risks like inflammation or infection, and it may not improve vision.

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Conditions

The condition(s) this trial relates to.

Bardet-Biedl syndrome 1 retinal degeneration

As listed by the trial registrant

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

BARDET-BIEDL SYNDROME 1 RETINAL DEGENERATION

Contacts and locations

Locations

Moorfields Eye Hospital
RECRUITING

London, EC1V 2PD, United Kingdom

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