KB803

KB803 is an investigational gene therapy developed by Krystal Biotech for the treatment of ocular complications associated with dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder. KB803 is formulated as an eyedrop and is based on the same technology as B-VEC (beremagene geperpavec), which is already approved for cutaneous manifestations of DEB. The therapy utilizes a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1) vector to deliver functional copies of the COL7A1 gene directly to the corneal epithelium, aiming to address corneal abrasions and other ocular complications in DEB patients. A registrational Phase 3 clinical trial (IOLITE study) evaluating its efficacy in this indication was expected to commence in the first half of 2025[3][5][7][8].

Other names

B-VEC ophthalmic solutionberemagene geperpavec ophthalmic