AAVantgarde Bio is a clinical-stage biotechnology company and Fondazione Telethon/UNINA spin-off based in Milan, Italy. The company develops next-generation gene therapies for inherited retinal diseases, focusing on conditions caused by large gene mutations and areas of high unmet medical need. Its mission is to help retain and rejuvenate sight by targeting the genetic causes of disease.

AAVantgarde’s pipeline centers on Stargardt disease and Usher syndrome type 1B. Its technology is designed to address the challenge of delivering large genes, including ABCA4, which is associated with Stargardt disease. The company reports that preclinical studies have shown gene transfer, expression and long-term safety in relevant models. Its clinical development includes the LUCE-1 Phase 1/2 trial of AAVB-081 for Usher 1B-associated retinitis pigmentosa, as well as work on Stargardt disease, including the STELLA natural history study. AAVantgarde also provides information and resources for patients and families, covering inherited retinal diseases, gene therapy, clinical trials and expanded access. The company says its technology may have potential applications beyond its current programs.