Krystal Biotech announces publication in NEJM on application of B-VEC

Krystal Biotech announced data on the compassionate use of beremagene geperpavec, B-VEC, administered as an eyedrop to treat a patient with dystrophic epidermolysis bullosa with cicatrizing conjunctivitis has been published in the New England Journal of Medicine, NEJM. The full manuscript, titled “Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa,” can be found in the February 8, 2024 issue of the NEJM. A patient presented with severe cicatrizing conjunctivitis secondary to DEB. Surgical symblepharon lysis of the patient’s right eye with pannus removal was conducted and regular administration of B-VEC as an eyedrop directly to the eye were added to routine post-surgical care, three times weekly for the first two weeks and then once weekly. B-VEC application frequency was further decreased to once monthly once the corneal epithelium was healed. B-VEC was well tolerated and associated with full corneal healing by 3 months as well as significant visual acuity improvement from hand motion to 20/25 at 8 months.