Krystal Biotech, Inc. announced that it plans to offer patients with dystrophic epidermolysis bullosa (DEB), who are enrolled in the GEM-3 open label extension study, the opportunity to be dosed in their homes by a health care professional (HCP). Krystal is offering this convenience to patients based on feedback from the U.S. Food and Drug Administration (FDA) following review of Krystal's human factors (HF) validation study report submitted in January 2022. Per the FDA's guidance, Krystal plans to address the convenience of dosing a patient at home of intend-to-market B-VEC commercial product in its BLA submission that is anticipated in 2Q, 2022.

The GEM-3 B-VEC OLE study is a 78-week (approximately a year and a half) open-label extension study of B-VEC for participants diagnosed with DEB who are aged 6 months and older that began in May 2021. The primary endpoint will be to assess long term safety and tolerability of the topical gene therapy. The study will enroll subjects who participated in GEM-3 Phase III study, as well as new participants who were unable to participate in the Phase III study and who meet all enrollment criteria.

B-VEC is an investigational non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. B-VEC was designed to treat DEB at the molecular level by providing the patient's skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism.