AAVantgarde Reports Positive Phase 1/2 Data for Gene Therapy AAVB-081 in Usher Syndrome Type 1B
核心洞察
AAVantgarde Bio (搜索) presented encouraging safety and efficacy data from its LUCE-1 Phase 1/2 trial of AAVB-081 gene therapy for Usher syndrome type 1B (搜索) at EURetina 2025.
The first four participants with ≥180 days follow-up achieved >1 line improvement in best-corrected visual acuity, with no drug-related serious adverse events reported.
The dual AAV vector approach addresses a significant unmet need for approximately 20,000 patients in the U.S. and EU who currently have no treatment options for progressive vision loss.
AAVantgarde Bio (搜索) presented updated clinical data from its LUCE-1 Phase 1/2 trial of AAVB-081, a dual AAV gene therapy for Usher syndrome type 1B (搜索), at the 25th European Society of Retina Specialists Annual Congress in Paris. The data demonstrated encouraging safety and preliminary efficacy signals in patients with this inherited retinal disease that currently has no approved treatments.
Trial Results Show Promise for Vision Improvement
The safety data encompassed the first 11 participants treated across three dose cohorts: five participants from the low dose cohort, five from the medium dose cohort, and the first participant from the high dose cohort. Prof. Francesca Simonelli, Head of the Ophthalmology Unit at the University Hospital of Campania "Luigi Vanvitelli" and Principal Investigator of the LUCE-1 trial, presented the findings.
For the first four participants receiving AAVB-081 with follow-up of ≥180 days, the treatment demonstrated a favorable safety profile with no drug-related serious adverse events or dose-limiting toxicities reported to date. The first participant has been followed for one full year. Ocular inflammation occurred infrequently and was reversible with steroid treatment.
The efficacy data showed that all four participants with ≥180 days of follow-up achieved >1 line improvement in best-corrected visual acuity (BCVA). The first two participants demonstrated >3 lines improvement in low luminance visual acuity (LLVA). Additionally, microperimetry fixation stability improved in three out of the four participants.
Addressing Critical Unmet Medical Need
Usher syndrome type 1B (搜索) affects approximately 20,000 patients in the United States and European Union. The condition is caused by mutations in the MYO7A (搜索) gene and represents a particularly challenging therapeutic target because the therapeutic gene is 6.7 kb long, making it too large to fit inside a standard AAV vector.
Children with Usher1B are born deaf, have vestibular dysfunction, and begin to progressively lose vision in their first decade of life. While surgical treatments are available for deafness, no treatments exist for the progressive vision loss and blindness associated with the condition.
"For patients living with Usher syndrome type 1B (搜索), who currently face inevitable vision loss without any therapeutic options, these findings represent an important step forward," said Prof. Simonelli. "We are optimistic that continued follow-up will further confirm the potential of this gene therapy to make a meaningful difference in patients' lives."
Dual AAV Vector Platform Innovation
AAVB-081 utilizes AAVantgarde's dual AAV vector platform to overcome the size limitations of traditional gene therapy approaches. The LUCE-1 trial 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 (搜索) administered subretinally.
Dr. Natalia Misciattelli, CEO of AAVantgarde, emphasized the significance of the results: "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 (搜索). These findings reinforce our commitment to advancing innovative gene therapies that have the potential to transform the lives of patients and their families who currently have no treatment options."
AAVantgarde Bio (搜索) is a clinical-stage biotechnology company focused on developing next-generation gene therapies for inherited retinal diseases (搜索). The company's lead programs target Stargardt disease (搜索) and retinitis pigmentosa (搜索) due to Usher syndrome type 1B (搜索), both severe inherited retinal diseases with no approved treatments.