According to Krystal Biotech, the FDA has granted orphan drug designation to the company’s KB408 nebulized gene therapy for the treatment of alpha-1 antitrypsin (AAT) deficiency. Krystal, which is also developing inhaled gene therapies for the treatment of cystic fibrosis (KB407) and solid tumors (KB707), says that KB408 is intended to deliver 2 copies of the SERPINA1 gene, which encodes for AAT.
Krystal Biotech President, Research & Development, Suma Krishnan commented, “This important designation is a milestone in the advancement of KB408, and this decision by the FDA underscores the need for potential new treatment options for patients with AATD. We are encouraged by the profile of KB408 in preclinical studies to date and look forward to dosing patients once we receive clearance from the FDA.”
Read the Krystal Biotech press release.
