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Krystal Submits Investigational New Drug Application for KB103 Topical Gene Therapy Candidate for Dystrophic Epidermolysis Bullosa
Mar. 27, 2018 09:14AM PST
Genetics InvestingKrystal Biotech (NASDAQ:KRYS), a gene therapy company developing “off-the-shelf” treatments for rare dermatological diseases, announces the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1/2, first in-human trial of KB103, an HSV-1 based gene therapy engineered to deliver a human collagen-producing gene to patients …
Krystal Biotech (NASDAQ:KRYS), a gene therapy company developing “off-the-shelf” treatments for rare dermatological diseases, announces the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1/2, first in-human trial of KB103, an HSV-1 based gene therapy engineered to deliver a human collagen-producing gene to patients with Dystrophic Epidermolysis Bullosa (“DEB”).
As quoted in the press release:
“KB103 has the potential to become a first-in-class “off-the-shelf” topical gene therapy treatment for DEB. It is the result of an extensive research and preclinical effort by our internal team that included engineering, screening and testing a library of in-house constructed vectors and complementing cell lines. This reflects our deep expertise in our proprietary Skin Targeted Delivery Platform (“STAR-D”),” said Suma Krishnan, founder and chief operating officer of Krystal. “As we look ahead, we believe that the productive STAR-D platform and our intent to bring GMP manufacturing in-house by the end of 2018 will support rapid advancement of clinical programs to treat debilitating skin diseases.”