beremagene geperpavec (Krystal Biotech, Inc.)

KB003 (also known as KB803) is an investigational, redosable ophthalmic gene therapy developed by Krystal Biotech for the treatment and prevention of ocular complications, such as corneal abrasions and scarring, in patients with dystrophic epidermolysis bullosa (DEB). The therapy utilizes a replication-deficient herpes simplex virus type 1 (HSV-1) vector to deliver two functional copies of the COL7A1 gene directly to the corneal epithelial cells. This allows the cells to produce normal type VII collagen (C7), which is deficient in DEB patients, thereby restoring the structural integrity of the basement membrane zone and anchoring fibrils in the eye. Administered as an ophthalmic solution (eye drops), KB003 aims to address the fundamental molecular cause of the disease locally. It is currently in Phase 3 clinical development following promising results in compassionate use cases.

Other names

B-VEC ophthalmic solutionberemagene geperpavec ophthalmic