The Company
Krystal Biotech Inc. (NASDAQ:KRYS) is a clinical-stage gene therapy company based in Pittsburgh, PA, which is founded in 2015 and has its IPO in September 2017.
It develops novel, "off-the-shelf", topical gene therapies for serious genetic skin diseases.
Based on the strength of their proprietary technology (to be discussed further below), the company currently focuses on the most severe congenital genetic skin diseases, including their lead indication, Dystrophic Epidermolysis Bullosa (or DEB), as shown within the blue box of the pipeline summary below.
(Source: Company website. blue highlight by the author)
The technology
According to the company, they have developed a proprietary gene therapy platform, which they call the Skin TARgeted Delivery (or STAR-D) platform.
STAR-D platform produces gene therapies that use an engineered HSV-1 vector that the company believes can efficiently penetrate a broad range of skin cells, to then deliver to these skin cells the therapeutic (i.e. fully functional) gene.
As a result, the protein that is missing or defective due to the defective gene, that causes the disease, is able to be expressed by the patient's skin cells to carry out their normal function.
(Source: Company website)
The Vector
HSV-1 stands for type 1 herpes simplex virus, which causes cold sores and genital herpes. In recent years, HSV usage as a safe and effective vector in gene therapy has been extensively studied (here, here, here)
In 2015, Amgen's IMLYGIC (a genetically modified HSV-1) was approved by the FDA as the first oncolytic viral therapy for melanoma.
According to the company, their proprietary technology can genetically modify HSV1 so that as a gene therapy vector, it is "replication-defective," and "non-integrating" while still being able to efficiently penetrate a broad range of skin cells.
Simply put, the company believes that through their genetic modification they can get rid
