KB105 is a new gene therapy candidate engineered with a Transglutaminase-1 (TGM1) gene construct to treat TGM1 deficient autosomal recessive congenital ichthyosis
KB105 is the second pipeline product based on the STAR-D platform and is part of a growing pipeline of gene therapy candidates for skin diseases
Krystal Biotech Inc., (“Krystal”) (NASDAQ: KRYS), a gene therapy company developing medicines to treat dermatological diseases, today announces that it has submitted 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 KB105, an HSV-1 based gene therapy engineered to deliver a human transglutaminase-1 (TGM1) gene to patients with TGM1-deficient autosomal recessive congenital ichthyosis (ARCI).
TGM1-deficient ARCI is a debilitating rare skin disease characterized by excessive, thick scaling of the skin and causing multiple chronic health conditions. Leveraging its Skin Targeted Delivery (STAR-D) platform technology, Krystal’s approach is to use a non-replicating, non-integrating engineered HSV-1 virus, to deliver the TGM1 gene to dividing and non-dividing skin cells, causing them to produce the TGM1 protein that is lacking in this patient population. KB105 is designed to be an off-the-shelf treatment for TGM1-deficient ARCI that can be applied topically, directly to a patient’s skin.
“Advancing KB105 into the clinic is an important step for Krystal Biotech in our mission to bring life-changing treatments to people suffering from debilitating skin diseases,” said Suma Krishnan, founder and chief operating officer of Krystal. “As we head into the second half of 2019, we look forward to initiating a Phase 1/2 trial with KB105 and advancing KB103, our lead development candidate for dystrophic epidermolysis bullosa into a Phase 3 registration trial.”
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