AAVantgarde Bio Presents New Clinical Data from LUCE-1 Gene Therapy Trial in Usher Syndrome Type 1B

AAVantgarde Bio has presented updated clinical results from the ongoing Phase 1/2 LUCE-1 study evaluating AAVB-081, a dual AAV vector gene therapy for Usher syndrome type 1B-associated retinitis pigmentosa. The data were shared at the 25th Annual Congress of the European Society of Retina Specialists (EURETINA) in Paris.

About AAVB-081 and the LUCE-1 Study

AAVB-081 is a dual AAV vector gene therapy designed to address the genetic root cause of Usher syndrome type 1B, which is associated with progressive retinitis pigmentosa. The LUCE-1 trial (NCT06591793) is a Phase 1/2, multicenter, open-label, dose-escalation study evaluating the safety, tolerability, and preliminary efficacy of three dose levels of dual AAV8.MYO7A (AAVB-081) administered via subretinal injection.

Patient Cohorts and Dosing

The analysis presented by Prof. Francesca Simonelli of the University Hospital of Campania “Luigi Vanvitelli” (Naples) included 11 participants across three dose groups:

       • Low-dose cohort (n=5)

       • Medium-dose cohort (n=5)

       • High-dose cohort (n=1)

Safety and Tolerability Outcomes

Among the first four participants with at least 180 days of follow-up, including one patient followed out to 12 month, AAVB-081 was found to be well tolerated. Key safety outcomes included:

       • No drug-related serious adverse events

       • No dose-limiting toxicities reported

       • Ocular inflammation was infrequent and reversible with steroid treatment

Early Signals of Visual Function Benefit

Visual function outcomes among the first four patients demonstrated encouraging preliminary results:

       • All four participants experienced more than one line of improvement in best-corrected visual acuity (BCVA)

       • The first two participants demonstrated over three lines of improvement in low-luminance visual acuity (LLVA)

       • Fixation stability on microperimetry improved in three out of four participants

Expert Commentary

“The data we presented at EURETINA 2025 are very encouraging, showing that treatment with AAVB-081 has been well tolerated and is beginning to demonstrate signals of clinical benefit,” said Prof. Francesca Simonelli, LUCE-1 Principal Investigator. “For patients living with Usher syndrome type 1B, who currently face inevitable vision loss without any therapeutic options, these findings represent an important step forward.”