A Pittsburgh-based biotech firm is poised on the edge of regulatory approval for the first of its redosable gene therapy treatments for a rare skin disease using a platform that the company is already looking at as a potential treatment for a form of cystic fibrosis and as an anti-aging formula.
Krystal Biotech Chairman and CEO Krish Krishnan told an investors conference Tuesday afternoon that the South Side-based biotech firm had applied in June for a biologics license application (BLA) for what is known as Beremagene Geperpavec or B-VEC, a gene therapy to treat a rare skin disease known as dystrophic epidermolysis bullosa (DEB) that causes fragile skin and can lead to aggressive squamous cell carcinoma.
There's no treatment or cure for DEB but Krystal's B-VEC, a topic gel, delivers one or more copies of a gene to the cell of a patient whose genetic disease either has a missing or mutated gene. The FDA has accepted for review the biologics license application.
"We are now working with the authorities, working to get the drug approved and bring this medicine to the patients," Krishnan said during the H.C. Wainwright Annual Global Investment Conference. He said the company was also working to build a commercial team in anticipation for a product launch on a global basis, with manufacturing that would be done initially at one of Krystal's two Pittsburgh manufacturing plants. The existing 10,000-square-foot plant is operational and will manufacture B-VEC. A new facility of about 150,000 square feet is to open this year to scale up its production capabilities.
Krishnan said that Krystal's pipeline is not only gene therapies for rare skin diseases but also using the same platform for respiratory and pulmonary diseases. One candidate, KB407, would treat cystic fibrosis via nebulizer that could deliver a therapy for a rare form of cystic fibrosis that isn't treatable now.
Development is also ongoing on another product line, through a wholly owned subsidiary called Jeune, that would provide gene therapy that would increase the amount of collagen being produced. Aging like wrinkles and fine lines in the skin come from declining levels of collagen.
"The idea behind Jeune is the ability to deliver the Col(lagen) 1 or Col(lagen) 3 gene and allow yourself to produce that extra amount of collagen that potentially delays the onset of fine-line syndrome," Krishnan said.
The company has completed its phase-one study and is getting ready to launch a phase-two study either late this year or early next year.
