Krystal Biotech: FDA Grants Orphan Drug Designation For KB408

(RTTNews) - Krystal Biotech, Inc. (KRYS) announced the FDA granted Orphan Drug Designation for KB408 for the treatment of alpha-1 antitrypsin deficiency. 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 alpha-1 antitrypsin deficiency.

Suma Krishnan, President, Research & Development, Krystal Biotech, said: "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."

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