Krystal Biotech Announces Dosing of First Patient in the Phase 1/2 trial of KB103

Krystal Biotech (NASDAQ:KRYS), a gene therapy company developing topical and intradermal “off-the-shelf” treatments for rare dermatological diseases, today announces that the first patient has been dosed in the Phase 1/2, first in-human trial of KB103, a first-in-class topical gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB).

As quoted in the press release:

The Phase 1/2 trial at Stanford University is a single-center, open-label, placebo-controlled Phase 1/2 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.

DEB is a chronic, progressive and incredibly painful skin disease caused by mutations in the gene coding for type VII collagen, or COL7. As a result of mutated COL7, DEB patients’ skin is incredibly fragile, resulting in blistering or skin loss at the slightest friction. There are currently no approved treatments for DEB.

Click here to read the full press release.