Dystrophic Epidermolysis Bullosa
October 15, 2025
Real-World Use of the Topical Gene Therapy B-VEC in Dystrophic Epidermolysis Bullosa Patients Under 6 Months of Age
Summary: Real-world clinical data on the use of beremagene geperpavec-svdt (B-VEC) in patients under six months of age was presented at the 13th Pediatric Dermatology Research Alliance (PeDRA) Annual Conference.
- Presentation 1.1 MB
July 16, 2025
Efficacy and Safety of the Topical Gene Therapy Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Study of Japanese Subjects With Dystrophic Epidermolysis Bullosa
Summary: The Journal of Dermatology has published the results of the open-label extension study evaluating B-VEC therapy in Japanese DEB patients.
- Presentation 1.4 MB
April 21, 2025
The Utility of Collagen VII Topical Gene Therapy in Treatment of Surgical Defect After Excision of Recessive Dystrophic Epidermolysis Bullosa Associated Squamous Cell Carcinoma
Summary: The Utility of Collagen VII Topical Gene Therapy in Treatment of Surgical Defect after Excision of Recessive Dystrophic Epidermolysis Bullosa associated Squamous Cell Carcinoma
April 12, 2025
Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa
Published in the American Journal of Clinical Dermatology
Summary: The American Journal of Clinical Dermatology has published the results of the open-label extension study, which was conducted with 47 subjects receiving B-VEC weekly to target wound areas for up to 112 weeks (median 81 weeks).
March 9, 2024
Long‐Term Safety and Efficacy of Beremagene Geperpavec
(B‐VEC) in an Open‐Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa
Summary: During the AAD Annual Meeting in San Diego, California from March 8-12, clinical data from the open-label extension study of beremagene geperpavec (B-VEC) was presented. Over half of the patients in the study received B-VEC treatment for at least one year. The safety profile and wound closure rates observed in the open-label extension study were consistent with findings from the registrational Phase 3 GEM-3 trial and provide additional support for the long-term use of B-VEC for the treatment of DEB.
- Presentation 1 MB
February 29, 2024
Gene Therapy for Patients with Dystrophic Epidermolysis Bullosa
Summary: At this year’s Rare Disease Day at NIH, M. Peter Marinkovich, M.D., Associate Professor, Department of Dermatology, Stanford Medicine presented on recent approvals and advancements in gene therapy describing the path to recent approvals highlighting patient perspectives.
- Presentation 3.3 MB
June 14, 2023
Type VII Collagen Deficiency in the Oncogenesis of Cutaneous Squamous Cell Carcinoma in Dystrophic Epidermolysis Bullosa
Summary: The Journal of Investigative Dermatology has published an article that examines cutaneous squamous cell carcinoma pathophysiology in dystrophic epidermolysis bullosa with a focus on known oncogenesis pathways at play and explores the idea that therapeutic type VII collagen replacement may reduce cutaneous squamous cell carcinoma risk.
May 11, 2023
Long Term Use of Topical Beremagene Geperpavec (B-VEC) in Two Patients with Dystrophic Epidermolysis Bullosa Presented at the International Societies for Investigative Dermatology (ISID) 2023 Conference
Summary: During the ISID 2023 Conference held in Tokyo, Japan from May 10-13, data was presented on the long term use of topical B-VEC in two patients with dystrophic epidermolysis bullosa.
- Presentation 448.7 KB
December 15, 2022
Trial of Beremagene Geperpavec (B-VEC) for Dystrophic Epidermolysis Bullosa Published in the New England Journal of Medicine
Summary: The New England Journal of Medicine has published data from the pivotal Phase 3 (GEM-3) trial of beremagene geperpavec (B-VEC) for the treatment of dystrophic epidermolysis bullosa (DEB).
- Publication 724.3 KB
September 29, 2022
Clinical Characteristics, Healthcare Use, and Annual Costs Among Patients With Dystrophic Epidermolysis Bullosa Published in Orphanet Journal of Rare Diseases
Summary: Orphanet Journal of Rare Diseases has published an article detailing dystrophic epidermolysis bullosa (DEB) as a serious, ultra-rare, genetic blistering disease that requires a multidisciplinary care team and lifelong, proactive disease management.
- Publication 1.2 MB
June 29, 2022
A New Era of in Vivo Gene Therapy: the Applicability of a Differentiated HSV-1 Based Vector Platform for Redosable Medicines Published in Cell & Gene Therapy Insights
Summary: A review of Krystal Biotech’s proprietary HSV-1 based platform which is being leveraged to develop treatments for patients with serious diseases, including rare diseases in skin, lung, and other organ systems, has been published in Cell & Gene Therapy Insights.
- Publication 984.3 KB
June 15, 2022
Coverage of B-VEC Development Published in Nature Biotechnology
Summary: Nature Biotechnology has published an article covering beremagene geperpavec (B-VEC), an investigational, topical, redosable gene therapy, as the first HSV-1-based gene therapy in development for dystrophic epidermolysis bullosa (DEB).
- Publication 1.1 MB
March 28, 2022
In Vivo Topical Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa on Phase 1 and 2 Published in Nature Medicine
Summary: Nature Medicine has published data from the Phase 1 and Phase 2 trial of beremagene geperpavec (B-VEC), an investigational, topical, redosable gene therapy in development for recessive dystrophic epidermolysis bullosa (RDEB).
- Read More 9.7 MB
September 16, 2021
New Data on B-VEC and Dystrophic Epidermolysis Bullosa (DEB) Presented at the DEBRA International Conference 2021
Summary: During the DEBRA International Conference held virtually September 16-19, 2021, new data on use of beremagene geperpavec (B-VEC) in a murine corneal wound model, the proprietary Krystal Biotech HSV-1-based platform, burden of illness and socioeconomic costs of dystrophic epidermolysis bullosa (DEB) were presented.
- Presentation 1.3 MB
- Presentation 736.2 KB
May 14, 2020
New Data Across the B-VEC and KB105 Programs Presented at SID 2020
Summary: Positive results from the GEM-1 Phase 1/2 study of beremagene geperpavec (B-VEC), an investigational, topical redosable gene therapy, in patients with recessive dystrophic epidermolysis bullosa (RDEB) along with data on KB105, an investigational, redosable gene therapy, from the Phase 1/2 study in patients with TGM1-related autosomal recessive congenital ichthyosis (ARCI) were presented virtually at the Society for Investigative Dermatology (SID) Annual Meeting held May 13-16, 2020.
- Presentation 3.1 MB
- Presentation 1.5 MB
June 24, 2019
GEM-2 KB103 Phase 2 Clinical Study Update
Summary: Additional data about the GEM-2 KB103 Phase 2 clinical study were presented.
- Presentation 5.9 MB
May 18, 2018
New Preclinical Data on B-VEC Presented at the IID 2018
Summary: New in vivo data on B-VEC, an investigational, topical redosable gene therapy, being studied in recessive dystrophic epidermolysis bullosa (RDEB) skin was presented at the International Investigative Dermatology (IID) Meeting held in May 16-19, 2018 in Orlando, FL.
- Read More 2.6 MB
September 24, 2017
HSV-1 Mediated COL7A1 (KB103) Delivery to Keratinocytes and Fibroblasts for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Therapy: Preparations for Phase-I Clinical Trials
Summary: During the EB2017 Research Congress and the EB Clinet Conference, held September 24-27, 2017 in Salzburg, Austria, preclinical assessment data of HSV-1 mediated COL7A1 delivery in vitro and in vivo were presented.
- Presentation 752.8 KB