AVG-001

AVG-001 is a novel, inhaled gene therapy developed to treat Alpha-1 Antitrypsin Deficiency (AATD) lung disease. It utilizes a third-generation lentiviral vector delivered via nebulization to the lungs, promoting localized and durable production of human alpha-1 antitrypsin (hAAT) directly in the respiratory tract. This approach aims to address the limitations of current therapies, which rely on intravenous infusions of plasma-derived protein and are often clinically sub-optimal for lung disease. Preclinical data indicate that a single dose can provide long-term protection against neutrophil elastase-mediated lung injury without significant safety or toxicology concerns. The therapy is being positioned as an alternative to standard-of-care IV protein replacement for AATD patients, with preparations underway for clinical development[2][3][4].