Krystal Biotech was recently granted a U.S. patent covering its herpes simplex viral vectors and related gene therapy to be used in preventive, palliative (providing relief), or therapeutic treatment for wounds and skin disorders, particularly dystrophic epidermolysis bullosa (EB).
Patent No. 9,877,990 was issued by the U.S. Patent Office.
The company stated in a press release that the exclusive rights over these vectors and their use for dermatological conditions is an important step in advancing Krystal’s pipeline and moving its dystrophic EB product candidate KB103 into clinical trials.
“The granting of the composition of matter patent and methods of using the same for skin diseases is an important milestone for KB-103, our lead product candidate with orphan-drug designation designed to treat patients with dystrophic epidermolysis bullosa,” Suma Krishnan, founder and chief operating officer of Krystal Biotech, said in the news release.
KB103 is an investigational gene therapy currently in preclinical development for dystrophic EB.
Dystrophic EB is caused by mutations in the collagen type VII gene (COL7A1). Type VII collagen is particularly important to provide strength and stability to the skin. Gene mutations in COL7A1 result in a deficient connection between the epidermis (the top layer of the skin) and the dermis (the underlying skin layer).
The only currently available treatment for patients with dystrophic EB is palliative, and Krystal Biotech is aiming to fill this treatment gap using its KB103 gene therapy.
KB103 consists of an engineered herpes simplex virus vector (HSV-1) that will not replicate or invade the patient’s genome, but will fix the COL7A1 gene mutation by delivering a normal copy of the gene to skin cells.
The company’s choice for HSV-1 is based on its high efficacy to penetrate the skin and its low ability to trigger an immune response, compared with other viral carriers.
With the new patent, Krystal Biotech hopes to become a leader in off-the-shelf therapies for skin diseases, particularly in those without effective treatments.
“We believe this issuance puts Krystal Biotech in an IP leadership position in the application of gene therapy to treat dermatological diseases.” said Krish S. Krishnan, Krystal’s chairman and chief executive officer.
Currently, KB103 is the company’s only product in preclinical development. KB103 was granted orphan drug status by the U.S. Food and Drug Administration (FDA) in November 2017.