In 2016, veteran drug developer Suma Krishnan was in her early 50s when she began to seriously consider a radically different way to treat one of the rarest and most devastating skin disorders in the world: dystrophic epidermolysis bullosa, sometimes called “butterfly skin disease” for the fragility of the skin it afflicts. The condition leaves patients—often children—covered in wounds, in constant pain, and at risk of severe complications, including skin cancer. At the time, there were no approved treatments.
Krishnan’s idea was to deliver gene therapy as a topical gel, applied directly to the skin. The approach was unconventional, both in its use of a modified herpes simplex virus to deliver healthy copies of the damaged gene, and in the setting: at home, rather than in a hospital. With her husband and longtime biotech collaborator, Krish Krishnan, she co-founded Krystal Biotech in Pittsburgh, far from the typical biotech hubs. The couple self-funded the company’s early work with $5 million from prior ventures, avoiding venture capital and telling friends who invested to think of it as philanthropy.
Eighteen months later, Krystal went public on Nasdaq, raising $45 million. Six years after that, in 2023, the company achieved what had once seemed improbable: FDA approval for Vyjuvek, the first at-home gene therapy for butterfly skin disease. Clinical trials showed that the gel could promote healing by delivering a functional copy of the gene that produces a crucial type of collagen.
The approval was unusually swift for a new gene therapy—especially one cleared during the Covid-19 pandemic, when regulators were cautious about treatments involving viruses. Priced at $24,250 per vial, Vyjuvek’s annual cost for an average patient is roughly $631,000, though costs decline as wounds heal.
Krystal’s commercial success has been striking. In 2024, revenue surged to $291 million, up from $51 million the year before, with net income rising from $11 million to $89 million. The Krishnans retain a 12% stake, and Suma’s net worth is estimated at $300 million.
For Krishnan, who grew up in Bombay resisting the path her family expected—marriage over career—the achievement is the culmination of decades in drug development. Her career includes leading the approval of Vyvanse for ADHD and holding more than 70 patents. Now, with Vyjuvek approved in the U.S., Europe, and Japan, she is turning Krystal’s attention to other conditions, including ocular complications of butterfly skin disease, cystic fibrosis, and lung cancer.
Her goal is not just to make one breakthrough, but to prove the model works across diseases. “The first one is always harder,” she says. “Now we can use what we’ve built to do more.”
