Patients and Families

Our Targets

B-VEC for Dystrophic Epidermolysis Bullosa

Beremagene geperpavec (“B-VEC”, previously “KB103”) is Krystal’s lead product candidate that seeks to use gene therapy to treat all forms of dystrophic epidermolysis bullosa, or DEB. B-VEC uses Krystal’s STAR-D technology to deliver functional human COL7A1 genes directly to the skin of affected patients. The COL7A1 genes then express functional collagen VII to form anchoring fibrils, thus stabilizing the patient’s otherwise fragile skin. The therapy is applied topically to DEB wounds. Learn more about DEB here.

B-VEC’s Mechanism of Action

KB104 for Netherton Syndrome

Krystal is also developing a new product candidate, KB104, for the treatment of Netherton Syndrome (NS). KB104, also based on our underlying STAR-D technology, is engineered to deliver functional SPINK5 directly to the skin of patients that harbor mutations in this gene. Learn more about NS here.

KB105 for TGM1-deficient ARCI

Krystal has also developed a patented gene therapy called KB105 for the treatment of TGM1-Deficient Autosomal Recessive Congenital Ichthyosis (ARCI). KB105 uses the STAR-D technology to deliver functional TGM1 genes directly to the skin in order to express the transgultaminase-1 enzyme therein. Learn more about TGM1-Deficient ARCI here.

KB105’s Mechanism of Action

Additional Dermatological Indications

In parallel to our work on B-VEC, KB104, and KB105 (directed to the severe, debilitating skin diseases described above), we are actively applying our STAR-D technology to develop novel therapeutics to treat additional dermatological indications, including both aesthetic skin conditions (e.g., wrinkles) and chronic skin diseases (e.g., chronic wounds and atopic dermatitis). Learn more about aesthetic skin conditions here. Learn more about chronic skin diseases here.