{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T22:31:06Z","timestamp":1793313066145,"version":"4.5.0"},"reference-count":29,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2025,7,16]],"date-time":"2025-07-16T00:00:00Z","timestamp":1752624000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["The Journal of Dermatology"],"published-print":{"date-parts":[[2025,10]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    Dystrophic epidermolysis bullosa (DEB) patients have pathogenic variants in\n                    <jats:italic>COL7A1<\/jats:italic>\n                    , leading to skin fragility, blistering, and scarring. Beremagene geperpavec\u2010svdt (B\u2010VEC) is a replication\u2010defective herpes simplex virus type 1 (HSV\u20101)\u2010based gene therapy vector administered topically to deliver functional\n                    <jats:italic>COL7A1<\/jats:italic>\n                    to DEB wounds. In a United States (US) Phase 3 study, B\u2010VEC significantly improved wound healing at 3 and 6\u2009months compared to placebo, and in a US open\u2010label extension (OLE) study, weekly B\u2010VEC administration was well tolerated for up to 112\u2009weeks. The present study was conducted to confirm the efficacy and safety of B\u2010VEC in a cohort of Japanese DEB patients receiving weekly B\u2010VEC treatment (4.0\u2009\u00d7\u200910\n                    <jats:sup>9<\/jats:sup>\n                    plaque forming units (PFU)\/mL) for 52\u2009weeks. Wound healing assessments were conducted on a Primary Wound at visits corresponding to Month 3 (the secondary efficacy endpoint), Month 6 (the primary efficacy endpoint), and Months 9 and 12 (exploratory durability endpoints). Patient\u2010reported outcome (PRO) measures were employed as exploratory analyses of efficacy. Safety was assessed by adverse events (AEs) and clinical laboratory tests. Five subjects enrolled in the study; one discontinued due to challenges with following wound dressing disposal guidelines. The study met its primary and secondary efficacy endpoints with 100% of Primary Wounds demonstrating complete closure at Months 6 and 3, respectively; durable complete closure was observed in 3\/4 (75%) of wounds at Months 9 and 12. PROs indicated decreased pain, improvement in skin\u2010specific quality of life, and moderate to high treatment satisfaction. Four subjects reported ten AEs; all were assessed as mild or moderate in severity and unrelated to treatment by Investigators. None were serious, severe, or led to treatment\/study discontinuation. The results of the Japan OLE study are in agreement with the US Phase 3 and OLE studies, demonstrating the efficacy and safety of B\u2010VEC in Japanese patients with DEB.\n                  <\/jats:p>\n                  <jats:p>\n                    <jats:bold>Trial Registration:<\/jats:bold>\n                    Japan Registry of Clinical Trials: jRCT2053230075\n                  <\/jats:p>","DOI":"10.1111\/1346-8138.17863","type":"journal-article","created":{"date-parts":[[2025,7,17]],"date-time":"2025-07-17T02:24:50Z","timestamp":1752719090000},"page":"1494-1502","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Efficacy and Safety of the Topical Gene Therapy Beremagene Geperpavec\u2010\n                    <scp>svdt<\/scp>\n                    (B\u2010VEC) in an Open\u2010Label Study of Japanese Subjects With Dystrophic Epidermolysis Bullosa"],"prefix":"10.1111","volume":"52","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3865-6366","authenticated-orcid":false,"given":"Ken","family":"Natsuga","sequence":"first","affiliation":[{"name":"Department of Dermatology Graduate School of Medicine, Hokkaido University  Sapporo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daisuke","family":"Tsuruta","sequence":"additional","affiliation":[{"name":"Department of Dermatology Graduate School of Medicine, Osaka Metropolitan University  Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shota","family":"Takashima","sequence":"additional","affiliation":[{"name":"Department of Dermatology Graduate School of Medicine, Hokkaido University  Sapporo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiharu","family":"Tateishi","sequence":"additional","affiliation":[{"name":"Department of Dermatology Graduate School of Medicine, Osaka Metropolitan University  Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masaaki","family":"Takatoku","sequence":"additional","affiliation":[{"name":"Krystal Biotech, Inc.  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Pittsburgh Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael J.","family":"Johnston","sequence":"additional","affiliation":[{"name":"Krystal Biotech, Inc.  Pittsburgh Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ramakrishna","family":"Edukulla","sequence":"additional","affiliation":[{"name":"Krystal Biotech, Inc.  Pittsburgh Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suma M.","family":"Krishnan","sequence":"additional","affiliation":[{"name":"Krystal Biotech, Inc.  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