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

AAVantgarde Bio announced updated clinical results from its ongoing LUCE-1 phase 1/2 study of AAVB-081, presented at the 25th European Society of Retina Specialists (EURETINA) annual congress in Paris.

The LUCE-1 study is evaluating subretinal administration of AAVB-081, a dual AAV vector gene therapy designed to address the genetic cause of Usher syndrome type 1B-associated retinitis pigmentosa.

Data presented by Prof. Francesca Simonelli (University Hospital of Campania “Luigi Vanvitelli,” Naples) included results from the first 11 participants: Low dose cohort (n=5); medium dose cohort (n=5); and high dose cohort (n=1).

Among the first four participants with ≥ 180 days of follow-up (including one out to 12 months):

• No drug-related serious adverse events or dose-limiting toxicities were reported

• Ocular inflammation was infrequent and reversible with steroid treatment

• Visual Function Outcomes

  • All four achieved >1 line improvement in best-corrected visual acuity (BCVA)

  • The first two participants showed >3 lines of improvement in low-luminance visual acuity (LLVA)

  • Microperimetry fixation stability improved in 3 of 4 participants

“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.”

About the LUCE-1 Trial (NCT06591793)

LUCE-1 is a phase 1/2, multicenter, open-label, dose-escalation study investigating the safety, tolerability, and preliminary efficacy of three dose levels of dual AAV8.MYO7A (AAVB-081) in patients with retinitis pigmentosa due to Usher syndrome type 1B. The therapy is administered via subretinal injection.

Further details on the LUCE-1 trial can be found here.