Jun 11 2018 First Patient Dosed with Dystrophic Epidermolysis Bullosa Treatment, KB103
Krystal Biotech have announced that the first patient has been dosed in the Phase 1/2 clinical trial of its drug, KB103, for the treatment of dystrophic epidermolysis bullosa (DEB).
The Phase 1/2 trial at Stanford University is a single-centre, open-label, placebo-controlled study conducting an intra-subject comparison of randomized treatment and control wounds. It is designed to evaluate the safety and tolerability of KB103 in subjects with the recessive form of dystrophic epidermolysis bullosa. Efficacy is also evaluated through wound imaging and analysis of collagen VII expression and anchoring fibril formation in the basement membrane zone.
It is the mutated COL7 that means DEB patients’ skin is incredibly fragile, resulting in blistering or skin loss at the slightest friction. KB103 is a replication-defective, non-integrating viral vector that has been engineered employing Krystal’s STAR-D platform to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells.
Read Krystal Biotech’s full press release.