AVG-002

AVG-002 is a novel, inhaled gene therapy developed by AlveoGene for the treatment of lethal neonatal Surfactant Protein B (SP-B) deficiency, an ultra-rare monogenic disorder that causes fatal respiratory distress syndrome in newborns. The therapy uses AlveoGene’s proprietary InGenuiTy platform, which employs a unique pseudotyped lentiviral vector to deliver a functional SP-B gene directly to the deep lung alveolar region via respiratory instillation. This approach aims to restore lung function and enable lifelong local production of surfactant from a single administration. Preclinical studies in SP-B knockout mouse models have shown that AVG-002 can substantially extend survival and restore normal lung histology and function compared with other candidates. The drug has received Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA, with clinical development underway and potential marketing authorization targeted by 2028[1][2][4][5][6].