Krystal Gains as First Topical Gene Therapy Wins Approval (3)

(Adds William Blair comments starting in second sentence.)

Krystal Biotech is up 8% in Friday trading after winning US approval for the first topical gene therapy, indicated to treat wounds in patients with a rare blistering skin disorder called DEB.

William Blair (outperform) called it a “transformational” moment for the drug developer, estimating $1 billion in worldwide sales from the product by 2030.

• The FDA approved the Vyjuvek treatment for dystrophic epidermolysis bullosa (DEB) with mutations in the COL7A1 gene
• Regulators cleared use in both recessive and dominant forms of DEB, and also allowed at-home dosing
  • “Receiving both items in the ...