Gene therapy company focused on developing treatments for rare dermatological diseases.
Krystal Biotech, Inc. is a clinical-stage biotechnology company pioneering the advancement of redosable gene therapy for the treatment of severe rare diseases across the United States. At the forefront of its innovation is beremagene geperpavec (B-VEC), the company's lead product candidate currently undergoing Phase III clinical trials for the treatment of dystrophic epidermolysis bullosa, a debilitating genetic disorder.
In addition to B-VEC, Krystal Biotech is actively developing KB105, currently in Phase I/II clinical trials targeting autosomal recessive congenital ichthyosis, a rare skin disorder. The company is also progressing KB301 through Phase I/II clinical stages, aiming to address skin aging and related conditions, underscoring its commitment to diverse therapeutic applications within dermatology.
Further expanding its pipeline, Krystal Biotech is advancing KB407 through preclinical stages as a potential treatment for cystic fibrosis, while also exploring KB104 in preclinical development for netherton syndrome. The company's robust portfolio includes discovery-stage candidates like KB5xx for chronic skin diseases and KB3xx for aesthetic skin conditions, highlighting its dedication to addressing unmet medical needs across various dermatological conditions.
Founded in 2015 and headquartered in Pittsburgh, Pennsylvania, Krystal Biotech, Inc. leverages cutting-edge gene therapy technologies to pioneer transformative treatments, aiming to enhance the lives of patients grappling with rare and severe genetic disorders worldwide.