In The Spotlight: Krystal Biotech

(RTTNews) - Shares of Krystal Biotech Inc. (KRYS) touched a new high of $13.00 on Thursday, and are up 30% since the beginning of this year.

Krystal is a gene therapy company focused on developing novel treatments for patients suffering from dermatological diseases through its proprietary gene therapy platform, known as Skin TARgeted Delivery platform, or STAR-D platform.

The Company's lead product candidate KB103, currently under phase 1/2 clinical study to treat dystrophic epidermolysis bullosa, or DEB, was initiated last month.

KB103 is a replication-defective, non-integrating viral vector that has been engineered using the STAR-D platform to deliver functional human COL7A1 genes directly to patients' dividing and non-dividing skin cells. It is designed to be an off-the-shelf treatment for DEB that can be applied as needed, either intra-dermally or topically to a patient's skin.

The FDA and European Medicines Agency have granted Orphan drug designation for KB103 for the treatment of Dystrophic epidermolysis bullosa.

Dystrophic epidermolysis bullosa, or DEB, is an incurable, often fatal skin blistering condition caused by a lack of collagen protein in the skin. It is caused by mutations in the gene coding for type VII collagen, or COL7. The most severe form of DEB is recessive DEB, or RDEB, which is caused by null mutations in the COL7A1 gene, and the milder form of DEB occurs in the form of dominant DEB, or DDEB.

There is currently no cure for dystrophic epidermolysis bullosa, and the current standard of care for DEB patients is limited to palliative treatments. It is estimated that there may be as many as 125,000 patients worldwide who suffer from DEB.

The Company's phase 1/2 clinical study of KB103 will initially enroll 3 adult patients, followed by 3 adult and pediatric patients. The initial data from the study is expected in the second half of this year.

Also in the pipeline are, KB105, to treat Lamellar Ichthyosis, an autosomal recessive disorder that is disfiguring and causes considerable psychological stress to affected patients, and KB107 for chronic wounds.

The Company is on track to file its Investigational New Drug application on KB105 for the treatment of lamellar ichthyosis this year, and begin clinical studies in the first quarter of 2019. As for KB107, it is in early stage of research.

*Key Numbers...

Accumulated deficit: $11.22 million Cash on hand: $47.23 million Shares Outstanding: 10.31 million Float: 4.78 million

(*As of March 31, 2018)

Krystal Biotech went public on the NASDAQ Capital Market on September 20, 2017, offering its shares at a price of $10 each. The stock has thus far hit a low of $8.03 and a high of $13.00.