KB408

KB408 is an investigational inhaled gene therapy developed by Krystal Biotech for the treatment of alpha-1 antitrypsin deficiency (AATD). It utilizes a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-based vector to deliver two copies of the SERPINA1 gene, which encodes human alpha-1 antitrypsin protein. The therapy is administered via nebulization to target respiratory epithelial cells in the lung. Its mechanism involves functional gene replacement, enabling expression of normal AAT protein in patients with AATD. Early clinical data show successful transduction and increased AAT levels in lung tissue and serum, with reductions in active neutrophil elastase[1][3][4][5][6][8].