Krystal Biotech's Gene Therapy Gel Approved for Rare Skin Disease.
The US FDA has approved the first-ever gene therapy gel for the most severe forms of "butterfly disease," a rare and painful skin blistering condition. The gel, called Vyjuvek, delivers working copies of the defective collagen gene directly into patients' cells. The treatment significantly improved the rate at which patients' wounds closed, compared to a placebo gel, and helped prevent the skin from immediately blistering again. The most common side effects included itching, chills, redness, rash, cough, and runny nose. The gene therapy gel is now approved for use in DEB patients who are at least 6 months old.