Krystal Application for KB408 for Type 1 Alpha-1 Antitrypsin Deficiency OKd

Orphan Drug Designation Granted to KB408

Krystal Biotech Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug Application (IND) for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD).

KB408 is a modified, replication-defective, non-integrating HSV-1-derived vector carrying two full-length copies of the serpin family A member 1 (SERPINA1) gene to enable expression of alpha-1 antitrypsin (AAT). KB408 is formulated for inhaled delivery to the respiratory cells of the lungs via nebulization

https://www.biospace.com/article/releases/krystal-biotech-announces-fda-clearance-of-investigational-new-drug-application-for-kb408-for-the-treatment-of-type-1-alpha-1-antitrypsin-deficiency/