Krystal Biotech

Gene Therapy

KB105 Clin1/Clin2 TGM1-deficient ARCI (ODD, fast track); KB407 Clin1 cystic fibrosis (ODD); KB301 Clin1 undisclosed; KB104 Clin0 Netherton syndrome; KB5XX Clin0 skin conditions; KB408 Clin0 alpha-1 antitrypsin deficiency; 6+ additional programs RD/Clin0 undisclosed

Krystal Biotech is developing gene therapies to address skin diseases. Many debilitating skin diseases lack effective treatments. Krystal is solving this by introducing therapeutic genes into patients with skin diseases. To achieve this, Krystal is using their patented gene therapy platform, Skin TARgeted Delivery (STAR-D), that consists of an engineered HSV-1 vector optimized for gene transfer in skin. The engineered HSV-1 vector is replication-defective and non-integrating such that it can penetrate a broad range of skin cells without posing a risk to genome disruption. The therapies are designed to be administered topically allowing direct and repeat delivery of therapeutic genes to the skin. Krystal’s most advanced therapy for Epidermolysis Bullosa is in Phase 3 trials.