Krystal Biotech Announces FDA Clearance Of IND For KB408

(RTTNews) - Krystal Biotech, Inc. (KRYS) reported on Thursday that the FDA cleared the Investigational New Drug Application (IND) for the company's orphan drug designated KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD).

KB408 is an inhaled (nebulized) formulation of the Company's new replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, which encodes for human alpha-1 antitrypsin protein, for the treatment of AATD.

The first patient in a Phase 1 clinical study is expected to be dosed in the first quarter of 2024, according to the company.

The Phase 1 clinical trial is a single-dose escalation study in adult patients with AATD who have the PI*ZZ genotype. According to the company, the three planned dose levels of KB408 will be assessed with three patients in each cohort to assess the drug's safety, tolerability, and effectiveness.