Krystal's topical gene therapy meets endpoints in two patients with skin blistering disease

Krystal Biotech Inc. (NASDAQ:KRYS) reported interim data from the Phase I/II GEM trial showing that topical gene therapy KB103 met all primary efficacy and safety endpoints in two adults with recessive dystrophic epidermolysis bullosa.

KB103 is a replication-defective, non-integrating HSV-1 viral vector engineered using the company's STAR-D platform to deliver functional human collagen type VII (COL7A1) genes directly to the patients' dividing and non-dividing skin cells. The company said the high payload capacity of the HSV-1 vector to accommodate large or multiple genes and its low immunogenicity make it a suitable candidate for direct and repeat delivery to the skin...