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KB104 is a herpes simplex virus type 1 (HSV-1) vector-based gene therapy developed by Krystal Biotech for the treatment of Netherton syndrome, a rare genetic skin disorder. The therapy is engineered to deliver a functional copy of the SPINK5 gene directly to the skin of patients with mutations in this gene, aiming to restore normal skin barrier function. The underlying technology platform is STAR-D, which enables targeted delivery and expression of therapeutic genes in affected tissues. As of early 2024, KB104 remains in preclinical development and has not yet entered clinical trials[1][2][8][9].
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