(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.
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