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.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


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