PITTSBURGH, March 18, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced that it will present late-breaking research at the 2022 American Academy of Dermatology Annual Meeting, taking place March 25-29 in Boston, Mass.
“We look forward to sharing additional results from Krystal’s Phase 3 study of the clinical efficacy and safety of beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB),” said Suma Krishnan, President, Research & Development, Krystal Biotech, Inc. “This is a terrific opportunity to raise awareness of DEB among the dermatology community and convey the need for a therapeutic option that addresses the underlying genetic cause of the disease.”
Krystal Late-Breaking Presentation
- GEM-3: A phase 3 study of beremagene geperpavec (B-VEC), an investigational, topical gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB)
M. Peter Marinkovich, MD, FAAD
Session #F045—Late-Breaking Research: Clinical Studies/Pediatric
Presentation Date & Time: Saturday, March 26, 2022, 10:50-11:00 a.m. EDT
Location: Room 253A at the Boston Convention and Exhibition Center
About Dystrophic Epidermolysis Bullosa (DEB)
DEB is a rare and severe disease that affects the skin and mucosal tissues. It is caused by one or more mutations in a gene called COL7A1, which is responsible for the production of the protein type VII collagen (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, which leads to skin infections, fibrosis which can cause fusion of fingers and toes, and ultimately an increased risk of developing an aggressive form of squamous cell carcinoma which, in severe cases, can be fatal.
About B-VEC
B-VEC is an investigational non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. B-VEC was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism.
The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have each granted B-VEC an orphan drug designation for the treatment of DEB. The FDA has also granted B-VEC fast track designation and rare pediatric designation for the treatment of DEB. In addition, in 2019, the FDA granted Regenerative Medicine Advanced Therapy (“RMAT”) to B-VEC for the treatment of DEB and the EMA granted PRIority MEdicines ("PRIME") eligibility for B-VEC to treat DEB.
About Krystal Biotech
Krystal Biotech, Inc. (NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its proprietary, redosable gene therapy platform and in-house manufacturing capabilities to potentially bring life-changing treatment options to patients with serious diseases, including rare diseases in skin, lung, and other areas. For more information, please visit http://www.krystalbio.com and follow @KrystalBiotech on LinkedIn and Twitter.
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Krystal Biotech
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Krystal Biotech
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Source: Krystal Biotech, Inc.
