AAVantgarde presents positive clinical data from its AAVB-081 program for Usher 1B and preclinical data from its AAVB-039 program for Stargardt at the ARVO 2025 annual meeting

MILAN, May 12, 2025 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announces the presentation of two abstracts at the Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), held May 4-8, 2025 in Salt Lake City (US).

Prof. Simonelli, Head of the Ophthalmology Unit at the University Hospital of Campania “Luigi Vanvitelli” (Naples), presented safety data of the first 8 subjects dosed (full low dose cohort and 3 patients from the medium dose cohort) and efficacy data at day 180 of the first subject dosed. Results show no drug-related serious adverse events or dose-limiting toxicities to date. Ocular inflammation has been infrequent, minimal and reversible with steroid treatment. Subject 1 showed >1 line improvement in BCVA and > 3 lines improvement in LLVA, along with multiple positive trends on functional endpoints observed in several subjects.

The poster presented by AAVantgarde's CMO, Dr. Jayshree Sahni, showed preliminary data from the NHP GLP study, designed to evaluate the safety and pharmacokinetics of subretinal administration of dual AAV8.ABCA4. Overall, findings are consistent with subretinal administration of AAVs, confirming a positive safety profile that supports the initiation of a first-in-human clinical study.

Prof. Francesca Simonelli, Head of the Ophthalmology Unit at the University Hospital of Campania “Luigi Vanvitelli” (Naples) and Principal Investigator of the LUCE-1 clinical trial, expressed her enthusiasm about the study's progress:​ “I am delighted to be involved as Principal Investigator in this first-in-human Phase 1/2 clinical study of AAV-081 for patients with retinitis pigmentosa related to USH1B and I am very excited to present updated positive safety results in 8 patients and day 180 results for our first subject dosed, showing preliminary efficacy signal. Through this innovative program, we aim to revolutionize our approach to understanding and treating these underserved patients. We are poised to generate robust evidence that will not only advance scientific knowledge, but also directly impact patient care.”

Dr. Jayashree Sahni, CMO of AAVantgarde also commented on her presentation that “the presented data of our GLP-NHP study showing a positive safety profile for our AAVB-039 program is promising and supports the initiation of a first-in-human clinical study, providing hope for underserved Stargardt patients that don't have any therapeutic options to prevent them from losing their sight.”

About AAVantgarde
AAVantgarde Bio is a clinical stage, International biotechnology company that has developed two proprietary Adeno-Associated Viral (AAV) vector platforms to address the gene therapy cargo capacity limitations of AAV vectors. The AAVantgarde platforms are being used to deliver large genes to the Company's two lead programs in Usher 1B and Stargardt disease, two inherited retinal diseases with clear unmet need, with the platform also having the potential for applicability in non-ocular tissues. For more information, please visit: www.aavantgarde.com

Contact:
Dr. Magda Blanco – Head of Corporate Development
Email: info@aavantgarde.com