KB407

KB407 is an investigational, redosable gene therapy designed to treat cystic fibrosis. It uses a replication-defective, non-integrating herpes simplex virus 1 (HSV-1) vector to deliver two full-length functional copies of the human cystic fibrosis transmembrane conductance regulator (CFTR) gene directly to respiratory cells in the lungs. This approach aims to restore normal CFTR protein production and function regardless of the specific underlying CFTR mutation in a patient. The therapy is administered via inhalation (nebulization). Preclinical studies have shown that KB407 can induce expression of functional CFTR protein in cell and organoid models derived from patients with various CFTR mutations. The drug has received orphan drug status for cystic fibrosis from both the US FDA and European Commission and is currently being evaluated in Phase 1 clinical trials for safety and tolerability in adults with cystic fibrosis[1][2][3][4][5][6][7].