AAVantgarde Completes Enrollment in Retinal Gene Therapy Trial for Usher Syndrome Type 1B
Research News
Milestone brings hope to patients with rare inherited retinal disease
AAVantgarde Bio has completed enrollment in LUCE-1, a Phase 1/2 clinical trial testing AAVB-081, an investigational gene therapy for retinitis pigmentosa associated with Usher syndrome type 1B (USH1B). The trial enrolled 15 adult participants, aged 18 to 60. The LUCE-1 study is evaluating the safety, tolerability, and preliminary effectiveness of a single subretinal injection of AAVB-081 across three dose levels.
This marks an important step forward in developing treatments for USH1B, a severe inherited condition that is caused by mutations in the MYO7A gene. Children with USH1B are born deaf, experience balance problems, and then begin losing their vision during their first decade of life. While surgical interventions can help with hearing loss, progressive blindness has remained untreatable.
According to AAVantgarde Bio, approximately 20,000 patients in the United States and European Union are affected by USH1B, making the development of effective therapies critically important for these families.
AAVB-081 uses AAVantgarde's dual-AAV gene therapy platform to overcome a major technical challenge: the MYO7A gene is too large to fit into a standard gene therapy vector. The innovative solution uses two vectors that work together at the cellular level to deliver the full therapeutic gene. LUCE-1 is the first clinical trial to test a dual-AAV approach in the retina.
"Completing enrollment in the LUCE-1 study represents an important milestone in the clinical development of AAVB-081," said Dr. Natalia Misciattelli, CEO of AAVantgarde. "We are grateful to the patients, investigators, and clinical site staff whose commitment has enabled the rapid advancement of this program."
AAVantgarde is also actively recruiting for the CELESTE trial, testing a similar dual-vector gene therapy (AAVB-039) for Stargardt disease, another inherited retinal disease with no approved treatments. The company expects to share updates on both programs throughout 2026.
