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Potekaev N.N.

Moscow Scientific and Practical Center of Dermatovenereology and Cosmetology;
Patrice Lumumba Peoples’ Friendship University of Russia

Porshina O.V.

Moscow Scientific and Practical Center of Dermatovenereology and Cosmetology

Zhukova O.V.

Moscow Scientific and Practical Center of Dermatovenereology and Cosmetology

First experience of the application of beremagene geperpavec genetically engineered drug for treatment of congenital dystrophic epidermolysis bullosa in the Russian Federation

Authors:

Potekaev N.N., Porshina O.V., Zhukova O.V.

More about the authors

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To cite this article:

Potekaev NN, Porshina OV, Zhukova OV. First experience of the application of beremagene geperpavec genetically engineered drug for treatment of congenital dystrophic epidermolysis bullosa in the Russian Federation. Russian Journal of Clinical Dermatology and Venereology. 2025;24(6):822‑827. (In Russ., In Engl.)
https://doi.org/10.17116/klinderma202524061822

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References:

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  2. Has C, Nyström A, Saeidian AH, BrucknerTuderman L, Uitto J. Epidermolysis bullosa: Molecular pathology of connective tissue components in the cutaneous basement membrane zone. Matrix Biol. 2018;7172:31329. https://doi.org/10.1016/j.matbio.2018.04.001
  3. Fine JD, Bruckner-Tuderman L, Eady RA, et al. Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification. J Am Acad Dermatol. 2014;70:1103-1126. https://doi.org/10.1016/j.jaad.2014.01.903
  4. Fine JD, Mellerio J. Extracutaneous manifestations and complications of inherited epidermolysis bullosa: Part I. Other organs. J Am Acad Dermatol. 2009;61:367-384.  https://doi.org/10.1016/j.jaad.2009.03.053
  5. Fine JD, Mellerio J. Extracutaneous manifestations and complications of inherited epidermolysis bullosa: Part II. Other organs. J Am Acad Dermatol. 2009;61:387-402.  https://doi.org/10.1016/j.jaad.2009.03.053
  6. El Hachem M, Zambruno G, Bourdon-Lanoy E, et al. Multicentre consensus recommendations for skin care in inherited epidermolysis bullosa. Orphanet Journal of Rare Diseases. 2014;9:76.  https://doi.org/10.1186/1750-1172-9-76
  7. Denyer J, Pillay E, Clapham J. Best practice guidelines for skin and wound care in epidermolysis bullosa. An International Consensus. Wounds International. 2017.
  8. Vyjuvek. Beremagene geperpavec. Overview. Accessed April 10, 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/vyjuvek
  9. Has C, Fischer J. Inherited epidermolysis bullosa: New diagnostics and new clinical phenotypes. Exp Dermatol. 2019 Oct;28(10):1146-1152. Epub 2018 Jun 28.  https://doi.org/10.1111/exd.13668
  10. Remington J, Wang X, Hou Y. Injection of recombinant human type VII collagen corrects the disease phenotype in a murine model of dystrophic epidermolysis bullosa. Mol Ther. 2009;17:26-33.  https://doi.org/10.1038/mt.2008.234
  11. Wong T, Gammon L, Liu L. Potential of fibroblast cell therapy for recessive dystrophic epidermolysis bullosa. J Invest Dermatol. 2008;128:2179-2189. https://doi.org/10.1038/jid.2008.78
  12. Wagner JE, Ishida-Yamamoto A, McGrath JA. Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. N Engl J Med. 2010; 363:629-639.  https://doi.org/10.1056/NEJMoa0910501
  13. Krystal Biotech receives FDA approval for the first-ever redosable gene therapy, Vyjuvek™ (beremagene geperpavec-svdt) for the treatment of dystrophic epidermolysis bullosa. News release. May 19, 2023. Accessed April 16, 2025. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene
  14. Marinkovich MP, Gonzalez ME, Guide S, et al. GEM-3: A Phase 3 Study of Beremagene Geperpavec (B-VEC), an Investigational Topical Gene Therapy, for the Treatment of Dystrophic Epidermolysis Bullosa (DEB). 2022 American Academy of Dermatology Annual Meeting. Boston, MA, United States. March 25-29, 2022. Accessed Apr 16, 2025. https://ir.krystalbio.com/static-files/bb74b04e-7e3d-44f8-afda-5469a3cf16b4
  15. Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB (GEM-3). Accessed April 16, 2025. https://clinicaltrials.gov/study/NCT04491604
  16. Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the Treatment of Dystrophic Epidermolysis Bullosa. Accessed April 16, 2025. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene
  17. Zorina AI, Zorin VL, Kopnin PB, Isaev AA, Manturova NE, Abdullaev RT, Ustyugov AYu. Current status of biomedical products for gene and cell therapy of recessive dystrophic epidermolysis bullosa (Part I). Russian Journal of Clinical Dermatology and Venereology. 2025;24(2):123-131. (In Russ.). https://doi.org/10.17116/klinderma202524021123

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