Preliminary Success in Gene Therapy for Inherited Blistering Skin Disease (Dystrophic Epidermolysis Bullosa) Described at Precision Medicine World Conference (PMWC 2023)

The closing presentation of Day 1, Track1, Gene & Cell Therapies, on Wednesday, January 25, at the Precision Medicine World Conference (PMWC 2023) in Santa Clara, California, featured the description of a possibly major breakthrough in gene therapy treatment for the genetic blistering skin disease known as dystrophic epidermolysis bullosa. The presentation was delivered by M. Peter Marinkovich, MD, an Associate Professor of Dermatology at Stanford, where he directs the Stanford Blistering Disease Clinic. Dr. Marinkovich’s laboratory has had a longstanding focus on the development of molecular therapy for various subtypes of epidermolysis bullosa. In a major advance, Dr. Marinkovich and colleagues published the following article in the December 15, 2022 issue of the New England Journal of Medicine: “Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa.” The conclusion of this work was that complete wound healing at 3 and 6 months in patients with dystrophic epidermolysis bullosa was more likely with topical administration of B-VEC than with placebo. Pruritus and mild systemic side effects were observed in patients treated with B-VEC. Dystrophic epidermolysis bullosa is a rare genetic blistering skin disease caused by mutations in COL7A1, which encodes a protein involved in assembling type VII collagen (C7). Beremagene geperpavec (B-VEC) is a topical investigational herpes simplex virus type 1 (HSV-1)–based gene therapy designed to restore C7 protein by delivering normal COL7A1. Longer and larger trials are warranted to determine the durability and side effects of B-VEC for this disease, the authors advised.

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