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Krystal Biotech Announces Publication of Phase 1 and 2 Clinical Trial (GEM 1/2 Study) of Beremagene Geperpavec (B-VEC) Data in Nature Medicine
Treatment with B-VEC demonstrated robust functional COL7 expression followed by its assembly into basement membrane-associated anchoring fibrils Treatment
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"Treatment with B-VEC demonstrated robust functional COL7 expression followed by its assembly into basement membrane-associated anchoring fibrils Treatment with B-VEC improved durable wound closure in patients with recessive dystrophic epidermolysis bullosa (RDEB) compared with placebo with minimal adverse events PITTSBURGH, March 28, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced that results from the Phase 1 and 2 study of topical beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB) were published in Nature Medicine. The publication provides a comprehensive analysis of the data from the Phase 1 and 2 study showing that repeat topical applications of B-VEC were associated with durable wound closure, full-length cutaneous type VII collagen (COL7) expression, and anchoring fibril assembly with minimal reported adverse events. “In this first-ever clinical trial of a redosable topical gene therapy, we are pleased to see that these data show the potential of B-VEC to address the underlying cause of the disease and delineate B-VEC as an easily administered, well tolerated therapy,” said Suma Krishnan, President, Research and Development, Krystal Biotech, Inc. “For so many years, all we have been able to offer DEB patients was palliative care, so it is now gratifying to have a potential corrective treatment option for this deserving group of patients. We are grateful to the trial participants who made this study possible.” The study was led by senior author M. Peter Marinkovich, M.D., Director of the Blistering Disease Clinic at Stanford Health Care and Associate Professor of Dermatology at the Stanford University School of Medicine. DEB is a rare and severe disease that affects the skin and mucosal tissues. It is caused by one or more mutations in the COL7A1 gene, which is responsible for the production of the protein COL7 that forms anchoring fibrils that bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin). The lack of functional anchoring fibrils in DEB patients leads to extremely fragile skin that blisters and tears from minor friction or trauma. DEB patients suffer from open wounds, leading to skin infections, fibrosis that can cause fusion of fingers and toes,...