KKRYSKrystal Biotech, Inc.

Krystal Biotech treats an inherited skin disease with a gene therapy applied like a gel.

Gene therapy is usually a one-off hospital procedure with demanding preparation. Here it is different: a viral vector carrying a working copy of the gene is applied directly to the wound, and the procedure is repeated regularly. That changes both the logistics and the economics — treatment becomes a course rather than a single event.

The condition is rare and severe: the skin tears at the slightest touch, and no treatment existed at all. The company is profitable, which is unusual for a developer of this kind. What follows depends on applying the same platform in other areas — ophthalmology and lung disease. Risks: the narrowness of the current market, reimbursement and new trial outcomes. The company pays no dividend.