Company Spotlight

Gene therapy has long promised to change the future of medicine—but few companies have managed to turn that promise into real-world success. Pittsburgh-based Krystal Biotech is one of the rare exceptions.

In August 2023, Krystal launched VYJUVEK®, the first FDA-approved redosable gene therapy—and the first genetic medicine for dystrophic epidermolysis bullosa (DEB), a rare and devastating skin disorder. The topical treatment delivers a functional COL7A1 gene to skin cells, enabling them to produce the collagen necessary for healing. For patients living with recurrent and chronic wounds and no prior treatment options, it has been life-changing.

Unlike many gene therapies still navigating lengthy clinical trial timelines, VYJUVEK is already reaching patients and improving lives. Since its launch, it has generated over $429 million in revenue and recently gained regulatory approval in Europe and Japan, with continued commercial expansion globally underway.

Krystal’s success is rooted in its proprietary gene delivery platform based on an engineered version of the HSV-1 virus—the same virus responsible for the common cold sore, but here modified into a safe and effective therapeutic tool. The vector was chosen for its ability to avoid triggering immune responses, work in both dividing and non-dividing cells, not alter the patient’s DNA, and allow for repeat, non-invasive dosing. In short, it solves many of the delivery challenges that have long held gene therapy back.

“We are pushing forward a broad and expanded pipeline which we expect will ultimately demonstrate the power of HSV-1 based gene delivery in the lung, eye, and skin—and most importantly—deliver meaningful benefit to patients,” said Krystal Chairman and CEO Krish Krishnan.

That pipeline is moving quickly. Krystal recently dosed the first patient in a Phase 1/2 trial for KB801, a redosable gene therapy eyedrop for neurotrophic keratitis (NK), a degenerative corneal disease with no approved treatments. The company is also running a Phase 3 trial for treating and preventing corneal abrasions in DEB patients. Additional programs in oncology, respiratory disease, dermatology, ophthalmology, and aesthetics are also in development.

Supporting all of this are serious infrastructure and assets: two in-house, commercial-scale CGMP manufacturing facilities, more than 30 issued patents, and a growing team of nearly 300 employees.

Despite its global aspirations, Krystal’s roots are firmly planted in Pittsburgh, with southwestern Pennsylvania’s deep local talent pool fueling Krystal’s rapid growth and success. 

“I believe it’s thanks to Pittsburgh that Krystal is where we are today. We have a team of people who are relentless in their work ethic and truly care about the patient communities we are focused on. The grit they exhibit is just unparalleled,” Krishnan said. “We are truly grateful for this city and its people.”

That commitment to the region continues to pay off. With an expanding pipeline, global momentum, and therapies already transforming lives, Krystal Biotech is proving that gene therapy’s future isn’t just possible—it’s already here.

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