Krystal Biotech, Inc., the leader in redosable gene therapy, today announced that the United States Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application to evaluate KB407 in a clinical trial for cystic fibrosis (CF).
KB407 is a modified HSV-1 vector carrying two copies of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to the respiratory cells in the lungs. By inducing expression of full length, normal CFTR protein in the lung, treatment with KB407 has potential to restore ion and water flow into and out of lung cells to correct the lung manifestations of the disease in patients regardless of their underlying genetic mutation.
CF is a genetic disease, affecting people of every racial and ethnic group, by causing a buildup of mucus in the lungs leading to persistent lung infections and progressive pulmonary disease. Sources including the Cystic Fibrosis Foundation indicate that there are close to 40,000 children and adults living with cystic fibrosis in the US, and an estimated 105,000 people diagnosed with CF across 94 countries.
