AAVantgarde Bio Presents Encouraging Clinical Data for Usher Syndrome AAV Gene Therapy

MILAN – September 5, 2025 – AAVantgarde Bio, a clinical-stage biotechnology company, today announced encouraging updated clinical data from its LUCE-1 study at the 25th European Society of Retina Specialists (EURETINA) Annual Congress in Paris. The study is investigating a next-generation gene therapy for inherited retinal diseases.

The data, presented by Prof. Francesca Simonelli, included safety information for the first 11 participants treated across three dose cohorts. The AAV gene therapy, known as AAVB-081, showed a favorable safety profile with no drug-related serious adverse events or dose-limiting toxicities. Ocular inflammation was infrequent and manageable with steroid treatment.

Preliminary efficacy data from four participants with at least 180 days of follow-up demonstrated positive clinical signals. All four showed an improvement of more than one line in BCVA (best-corrected visual acuity), and the first two participants showed a more than three-line improvement in LLVA (low luminance visual acuity). Fixation stability also improved in three of the four participants.

AAVantgarde’s dual AAV vector platform addresses the challenge of delivering large therapeutic genes, such as the 6.7 kb MYO7A gene mutated in Usher syndrome type 1B. This inherited disease affects both the retina and inner ear, leading to deafness and progressive vision loss for which there are currently no approved treatments.

Prof. Simonelli, the trial’s Principal Investigator, described the findings as “very encouraging,” noting that the therapy is well tolerated and shows signals of clinical benefit. AAVantgarde CEO Dr. Natalia Misciattelli added, “The positive safety and efficacy data further validate the potential of our dual AAV vector platform and addresses the high unmet need in Usher syndrome type 1B.”