Krystal Biotech announced that the Food and Drug Administration (FDA) has granted Fast Track designation to KB103 for the treatment of dystrophic epidermolysis bullosa (DEB), the first topically-applied herpes simplex virus (HSV-1) based gene therapy to deliver a human collagen protein.
DEB is a chronic and progressive skin disease caused by mutations in the gene coding for type VII collagen (COL7), which leads to extremely fragile skin , blistering, and skin loss at the slightest friction. Currently, there is no approved treatment for DEB.
KB103 is a replication-defective, non-integrating viral vector that utilizes the Company’s STAR-D platform to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. The safety and tolerability of KB103 is being evaluated in a Phase 1/2 single-site, open-label, placebo-controlled study using wound imaging, analysis of COL7 expression, and anchoring fibril formation.
For more information call (412) 586-5830 or visit Krystalbio.com.