This study focuses on patients with ABCA4 Mutation-Associated Retinal Dystrophy, which is an inherited eye disease that causes progressive vision loss due to damage to the light-sensing cells in the retina. The study will test a new experimental treatment called VG801, which is given as a single injection underneath the retina (through subretinal injection).
The purpose of this research is to evaluate the safety of VG801 and to see if it might help improve vision in people with this genetic eye condition. The treatment being studied is a sterile solution that contains two active components: VG801-1 and VG801-2. During the study, participants will receive the treatment in one eye and will be monitored through various eye examinations and tests.
Throughout the study, doctors will perform regular eye examinations using special imaging techniques such as optical coherence tomography to look at the retina in detail. They will also test vision using standard eye charts and measure how well participants can see in different lighting conditions. Additionally, participants will complete a special test using virtual reality to assess their ability to move around in their environment.
Germany 
