AAVantgarde is working on a new gene therapy and has launched a clinical trial for people living with Usher syndrome type 1B. The trial, called LUCE-1, is a Phase 1/2 clinical trial and has completed enrollment of 15 adults between the ages of 18 and 60. This study is focused mainly on checking whether the treatment, called AAVB-081, is safe when given as a one-time injection into the eye. Doctors will also watch for early signs that the therapy may help slow vision loss caused by retinitis pigmentosa (RP).
This gene therapy is designed to deliver a working copy of the MYO7A gene, which does not function properly in people with Usher syndrome type 1B. Because this gene is very large, researchers use a special two-part delivery system to carry it into retinal cells. There are currently no approved treatments for vision loss linked to this condition, making this milestone an important step. Researchers will now follow participants over time to collect safety data and share results as they become available.
What this means for the Usher syndrome community: This milestone shows continued progress toward treatments for Usher syndrome type 1B, a condition with no approved therapies for vision loss. While this trial is focused on safety, the information learned will help guide future studies and move research closer to real treatment options.
