Krystal Biotech’s KB408 granted Orpah Drug Designation in AATD

Krystal Biotech announced that the FDA granted Orphan Drug Designation for KB408 for the treatment of alpha-1 antitrypsin deficiency, or AATD. AATD is caused by mutations in the SERPINA1 gene that lead to decreased levels and/or decreased functionality of alpha-1 antitrypsin protein. Over time, the deficiency can lead to progressive enzymatic destruction of the lung tissue, ultimately causing life-threatening pulmonary impairment and severe respiratory insufficiency. In severe cases, current disease management includes intravenous augmentation therapy which requires weekly infusions, the clinical benefit of which remains to be established. KB408 is an inhaled formulation of the company’s replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment of AATD.