Topically-Applied Gene Tx Fast-Tracked for Dystrophic Epidermolysis Bullosa

KB103 is a replication-defective, non-integrating viral vector
KB103 is a replication-defective, non-integrating viral vector

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.  

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For more information call (412) 586-5830 or visit Krystalbio.com.