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Investigational gene therapy KB103 receives priority designation in Europe for dystrophic epidermolysis bullosa

from Epidermolysis Bullosa News

The topical gene therapy candidate KB103 to treat dystrophic epidermolysis bullosa has been granted PRIME (PRIority MEdicines) designation by the European Medicines Agency. KB103 is a gene therapy candidate developed by Krystal Biotech to deliver a functional COL7A1 gene directly to skin cells using a modified and safe herpes simplex virus. Mutations in the COL7A1 gene are the underlying cause of DEB and lead to a defective production of COL7 protein, causing layers of the skin to separate and form blisters. more


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