KB409

KB409 is a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-based gene therapy vector developed by Krystal Biotech for the treatment of primary ciliary dyskinesia (PCD). The therapy is designed to be delivered via non-invasive inhalation to the lungs, where it transduces respiratory epithelial cells to express a functional copy of the human DNAI1 (dynein axonemal intermediate chain 1) gene. DNAI1 is a critical component of the ciliary outer dynein arm, and mutations in this gene are a leading cause of PCD, a condition characterized by impaired ciliary motility, chronic respiratory infections, and bronchiectasis. By restoring DNAI1 protein expression, KB409 aims to address the underlying genetic cause of the disease and improve mucociliary clearance.