KB111

KB111 is an investigational, topical gene therapy being developed by Krystal Biotech for the treatment of Hailey-Hailey disease (HHD), a rare genetic skin disorder. HHD is caused by mutations in the *ATP2C1* gene, leading to a deficiency in the ATPase secretory pathway Ca2+ transporting 1 protein. KB111 utilizes Krystal's proprietary Skin TARgeted Delivery (STAR-D) platform, which employs a replication-defective, non-integrating HSV-1-based vector to deliver two functional copies of the full-length, wild-type *ATP2C1* gene directly to skin cells. By restoring functional levels of the ATP2C1 protein, the therapy aims to address the underlying calcium signaling deficiency, accelerate the healing of painful skin lesions, and reduce the overall disease burden. The therapy is designed for topical application and is currently in clinical development.