Clinical studies reported in the current issue of the New England Journal of Medicine include a phase 3 trial of beremagne geperpavec (B-VEC) in patients with the rare genetic blistering skin disease dystrophic epidermolysis bullosa; phase 2 results of the bispecific antibody glofitamab in relapsed or refractory diffuse large B-cell lymphoma; and a phase 1 study of talquetamab in patients with heavily pretreated relapsed or refractory multiple myeloma that had progressed with established therapies (a median of 6 previous lines of therapy) or who could not receive these therapies without unacceptable side effects.
Editorial: Commenting on the B-VEC trial in a “science behind the study” editorial, an author makes these observations about topical gene therapy for dystrophic epidermolysis bullosa: “Approximately 70% of the wounds exposed to B-VEC showed complete closure at 6 months (including in the patient with dominant dystrophic epidermolysis bullosa), as compared with approximately 20% of the wounds exposed to placebo. In addition, durability of wound healing (defined as complete wound closure at both 3 and 6 months) was observed in 50% of the wounds exposed to B-VEC, as compared with 7% of the wounds exposed to placebo. In a previous phase 1–2 study, electron microscopy of skin biopsy samples obtained from patients with recessive dystrophic epidermolysis bullosa showed that B-VEC restored C7 expression on immunofluorescence staining at the basement-membrane zone and induced formation of anchoring fibrils. Thus, topical gene therapy with B-VEC reverses the histopathological and ultrastructural abnormalities in the skin of patients with dystrophic epidermolysis bullosa.”