Four Patients Treated in Phase 2 Trial Testing Topical Gene Therapy

     KB103, for Wound Healing


All four patients with dystrophic epidermolysis bullosa (DEB) enrolled in a Phase 2 study testing the safety and efficacy of Krystal Biotech’s topical gene therapy candidate KB103 have received the treatment.


Results are expected to be known by mid-year.


The protein type 7 collagen (Col7) binds the two top layers of the skin, and its deficit results in blisters and wounds. Mutations in the Col7A1 gene encoding Col7 results in either non-working or insufficient levels of Col7, causing the skin blistering observed in DEB patients.


KB103 is a gene therapy candidate designed to deliver functional Col7 directly to the impacted cells with the help of a modified herpes simplex virus (HSV-1;  this modified virus is unable to cause disease).


In the ongoing six-month study (NCT03536143; GEM-1), underway at Stanford University, KB103 or placebo were applied directly to skin wounds (topical administration) of four DEB patients, two adults and two children (age 5 or older) … read more