AAVantgarde Presents Interim LUCE-1 Trial Data for Usher Syndrome 1B Gene Therapy at ARVO 2026
Key Insights
AAVantgarde Bio (search) will present preliminary safety and efficacy data from all 15 participants in the LUCE-1 Phase 1/2 clinical trial for AAVB-081 gene therapy targeting Usher syndrome type 1B (search) at ARVO 2026.
The AAVB-081 program utilizes a dual AAV8 vector system to deliver the full-length MYO7A (search) gene, addressing the root cause of vision loss in patients with USH1B (search) who currently have no treatment options.
The company will also participate in key pre-event presentations at Eyecelerator and the Foundation Fighting Blindness's Retinal Therapeutic Innovation Summit on May 1, 2026.
AAVantgarde Bio (search), a clinical-stage biotechnology company developing next-generation gene therapies for inherited retinal diseases (search), will present interim clinical data from its LUCE-1 Phase 1/2 trial at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting 2026. The presentation will feature preliminary safety and efficacy data from all 15 participants receiving AAVB-081 gene therapy for Usher syndrome type 1B (search) (USH1B (search)).
Clinical Trial Results and Presentation Details
The company will present its findings on Sunday, May 3rd, 2026, from 2:00-3:45 p.m. in the Exhibit Hall during the stem cell and gene therapy session. Dr. Francesco Testa will present poster 0636, titled "Interim analysis of the LUCE-1 trial: a Phase I/II Trial investigating AAVB-081 gene therapy in Usher Syndrome Type1B (USH1B (search)) caused by biallelic mutations in the MYO7A (search) gene."
The LUCE-1 trial is a multicenter, open-label, dose escalation study investigating the safety, tolerability, and preliminary efficacy of three dose levels of dual AAV8.MYO7A (search) (AAVB-081) administered subretinally in subjects with retinitis pigmentosa (search) associated with USH1B (search).
Innovative Dual Vector Technology
AAVB-081 represents an intra-retinal AAV8-based dual hybrid product targeting MYO7A (search)-associated Usher syndrome. The therapeutic approach addresses a significant technical challenge in gene therapy: the MYO7A gene is 6.7 kb long, too large to fit inside a standard AAV vector.
AAVantgarde's proprietary dual hybrid platform uses two AAV8 vectors, each containing one half of an expression cassette encoding for the MYO7A (search) gene. The system works at the cell nucleus level, recombining the two halves of the transgene back into a single transgene within the cell. This technology has demonstrated efficient recombination that generates high therapeutically effective protein levels in both small and large animal models.
Addressing Unmet Medical Need
Usher syndrome type 1B (search) affects approximately 9,000 patients in the U.S. and EU5. The condition is caused by mutations in the MYO7A (search) gene and represents a severe inherited disease affecting both the retina and inner ear. Children with USH1B (search) are born deaf, have vestibular dysfunction, and begin to progressively lose vision in their first decade of life.
While surgical treatments are available to address deafness in these patients, no treatments currently exist to treat progressive vision loss and blindness. The AAVB-081 program aims to deliver the full-length protein to the retina, addressing the root cause of the disease.
Additional Conference Participation
Beyond the main ARVO presentation, AAVantgarde will participate in several key pre-event presentations on May 1, 2026. CEO Dr. Natalia Misciattelli will present at Eyecelerator @ ARVO 2026 at 1:30 p.m. in the "Durable Retina Therapeutics – Gene Therapy & Drug Delivery" breakout session.
The company will also participate in the Foundation Fighting Blindness's Retinal Therapeutic Innovation Summit (RTIS) with two presentations. Dr. Misciattelli will deliver a sponsor presentation at 3:25 p.m., while Chief Medical Officer Dr. Jayashree Sahni will present "Design and Early Clinical Insights from LUCE: Dual-Vector MYO7A (search) Gene Therapy in Usher Syndrome Type 1B (search)" at 4:00 p.m. during the retinal gene augmentation clinical session.