Krystal Biotech (KRYS) started trading on the Nasdaq on 20 Sept 2017

• HQ: Pittsburgh, PA
• http://www.krystalbio.com/
• Founded: 2015

The Pittsburgh-based gene therapy company, which develops treatments for dermatological diseases, upped its offering to 3.96 million shares at $10 a share to raise up to $39.6 million. An additional 594,000 shares are available to cover overallotments. Last week, Krystal's Securities and Exchange Commission filing offered 3 million shares at a price range of $9 to $11 a share. In late August, Krystal Biotech said it hoped to raise up to $35 million in its IPO.

Krystal Biotech is a gene therapy company that is looking to bring products developed via its gene platform to the commercial market.

The company plans “to develop treatments for rare or orphan dermatological indications caused by the absence of or a mutation in a single gene, and plan to leverage our platform to expand our pipeline to include other dermatological indications in the future,” it said in its IPO filing.

The company’s leading product is known as KB103 and uses gene therapy to treat a rare genetic skin disease, dystrophic epidermolysis bullosa (DEB).

The disease currently has no approved treatment and those with it are often called “butterfly children” because the skin appears as fragile as butterfly wings.

KB103 is not yet in clinical trials.

The severest form of DEB is recessive DEB (RDEB), which “is characterized by severe skin blistering, extremely fragile skin, mutilating scarring of the hands and feet, joint contractures, strictures of the esophagus, and often, eventually the development of aggressive squamous cell carcinomas which may shorten the patient’s life,” the company said.

“Nearly 10% of RDEB patients die before the age of 10, almost 40% die by the age of 20, and over 70% die before the age of 30,” Krystal Biotech said.


Just One Gene Therapy Product Has the FDA’s OK

The market for the treatment would be 125,000 global patients, the company said, citing DEBRA International. There are about 3,200 to 3,500 patients in the U.S., European Union, Japan and Canada.

But here’s the big warning with the market opportunity for a gene therapy company.

“To date, only one gene therapy product has received marketing authorization from the FDA, and only two gene therapy products have received marketing authorization from the EMA,” Krystal Biotech said.