AVG-003

AVG-003 is an investigational, inhaled gene therapy being developed as a potentially first-in-class, disease-modifying treatment for newborns and infants with rare, lethal ABCA3 surfactant protein disorder. This autosomal recessive condition results from mutations in the ABCA3 gene, leading to defective or absent function of the phospholipid transporter required for pulmonary surfactant production. The therapy uses AlveoGene’s proprietary lentiviral vector backbone to deliver a functional copy of the relevant transgene directly to lung tissue via inhalation. By restoring normal surfactant metabolism at the site of disease, AVG-003 aims to provide a lifelong solution for affected patients who currently have no effective treatment options and often require lung transplantation for survival[1][4][6].