AAVantgarde Completes Enrolment in Phase 1/2 Clinical Trial for Usher Syndrome Type B
AAVantgarde Bio has reached an important milestone in its LUCE‑1 clinical trial, completing enrolment for a pioneering gene therapy designed to address vision loss caused by Usher syndrome type 1B. The announcement marks important progress toward a potential treatment option for this currently untreatable inherited retinal condition.
AAVantgarde Bio has announced the completion of enrolment in LUCE-1, their Phase 1/2 clinical trial evaluating AAVB-081 for the treatment of retinitis pigmentosa associated with Usher syndrome type 1B (USH1B).
“Completing enrolment in the LUCE-1 study represents an important milestone in the clinical development of AAVB-081” – Dr. Natalia Misciattelli, CEO of AAVantgarde.
What happens in Ush1B?
Ush1B is an inherited disease that affects the retina and the inner ear caused by mutations in the MYO7A gene. The MYO7A gene is responsible for encoding a protein called myosin VIIA, which is expressed in the retinal pigment epithelium layer and light-sensing photoreceptor cells. Mutations in MYO7A can result in either nonfunctional myosin VIIA protein being produced or a lack of myosin VIIA protein altogether. This causes the loss of critical visual cycle components, the defective transport of key proteins in photoreceptors and gradual photoreceptor degeneration. This results in retinitis pigmentosa, with the gradual loss of peripheral vision and central vision.
What does AAVB-081 do?
Most gene therapies for inherited retinal disease work on the premise of delivering an entire new gene to replace the mutated one, however, the whole MYO7A gene is too large to deliver in one viral vector. Researchers look to overcome this by using dual viral vectors to deliver half of the gene to the cell. Once in the cells, each half of the gene will make half of the desired protein. A technique known as protein splicing will then bond each half the protein together to make it functional. This results in a healthy copy of the MYO7A gene, allowing for the normal production of myosin VIIA protein.
LUCE-1 Trial:
The phase 1/2 trial, known as LUCE-1, is an open-label, dose-escalation and expansion study evaluating the safety, tolerability, and preliminary efficacy of a single subretinal administration of AAVB-081. LUCE-1 is taking place across multiple sites in Italy and the UK. The study has completed the recruitment of 15 adult participants between 18 to 60 years of age.
“Dosing the final patient in the LUCE Phase 1/2 study marks a significant milestone for this programme and for the patients and families affected by this devastating inherited retinal condition. Early-phase clinical research is where scientific innovation begins to translate into real-world impact, and LUCE represents an important step toward the development of a potential disease-modifying therapy. Prof. Michaelides, from Moorfields Eye Hospital
AAVantgarde is making rapid progress on both of its clinical programs and is also recruiting for the CELESTE study for Stargardt disease. Usher 1B and Stargardt Disease are both debilitating inherited retinal conditions with no approved treatments currently available. We eagerly await updates from both programs expected later in 2026.