KB105

KB105 is a topical gene therapy candidate developed by Krystal Biotech for the treatment of autosomal recessive congenital ichthyosis (ARCI) caused by transglutaminase 1 (TGM1) deficiency. It utilizes a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1) vector engineered to deliver a functional human TGM1 gene directly to dividing and non-dividing skin cells. The therapy aims to restore TGM-1 activity in the epidermis, which is essential for forming the skin barrier and preventing dehydration and infection. KB105 has shown safety and evidence of functional TGM-1 expression in early clinical trials, with no significant adverse events or systemic exposure reported.

Other names

TGM1 gene therapyTGM-1 gene therapyTGM 1 gene therapy