KRYS - Krystal Biotech files for FDA approval of topical gene therapy for rare skin disorder

Krystal Biotech (NASDAQ:KRYS) said it submitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of its gene therapy B-VEC to treat patients with dystrophic epidermolysis bullosa (DEB). DEB is a rare disease in which the skin becomes fragile and can tear or blister from minor injury/rubbing and is caused by one or more mutations in a gene called COL7A1. The company said that B-VEC (beremagene geperpavec) — which is an investigational non-invasive, topical gene therapy — provides the patient's skin cells with two copies of the COL7A1 gene to make functional COL7 protein, thus  addressing the fundamental disease-causing mechanism. The company's biologics license application (BLA) is backed by data from two trials called GEM-3 and GEM-1/2. Krystal (KRYS) added that it is also in discussions with regulatory authorities in other markets, including Europe and Japan, and plans to file an application with European Medical Agency in H2.

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Krystal Biotech files for FDA approval of topical gene therapy for rare skin disorder