Gene Therapy Gets Orphan Drug Designation for Severe Genetic Skin Disease

KB105 is a replication-defective, non-integrating viral vector
KB105 is a replication-defective, non-integrating viral vector

Krystal Biotech announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to KB105 for the treatment of patients with transglutaminase 1 (TGM-1) deficient autosomal recessive congenital ichthyosis (ARCI). 

Patients with TGM-1 deficiency may have chronic pronounced scaling of the skin with increased transepidermal water loss, as the TGM-1 enzyme is essential for facilitating the formation of the epidermal barrier, which prevents dehydration. Other complications can include sepsis, fluid and electrolyte imbalances due to impaired skin barrier, and failure to thrive. Current treatments only relieve some symptoms of ARCI.

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KB105, a replication-defective, non-integrating viral vector, utilizes the STAR-D (Skin TARgeted Delivery) platform to deliver functional human TGM-1 gene directly to the patients' skin cells. The modified HSV-1 viral vector can penetrate a broad range of skin cells with its high payload capacity and low immunogenicity. 

The Company anticipates filing an Investigational New Drug Application (IND) in the fourth quarter of 2018. 

For more information visit KrystalBio.com.