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Krystal Biotech, Inc.
Krystal Biotech makes and sells gene therapies, led by a skin-treatment gel for a rare blistering disease.
Krystal Biotech is a gene therapy company. Its main product is a gel that a doctor spreads on a patient's skin to treat a rare inherited disease called dystrophic epidermolysis bullosa, where the skin is so fragile that everyday friction causes painful blisters and wounds. The gel delivers a working copy of a gene directly into skin cells, helping the skin make a protein it needs to hold itself together.
The company earns money by selling that gel, which it markets as Vyjuvek, in the United States and other countries. It got U.S. approval in 2023, making it the first gene therapy for this disease that patients can apply to the skin rather than receive through injections or infusions. That convenience matters, because many patients are children whose wounds cover much of their body, and a topical treatment is far easier to give than a hospital procedure. Krystal's bigger idea is its manufacturing platform. Gene therapies are usually expensive and slow to make, but Krystal built a system using a modified herpes simplex virus that can carry genetic instructions into cells, made in its own facilities in Pennsylvania, where the company is based. It is testing this same approach on other skin and lung problems, hoping to turn a single successful drug into a broader pipeline.
The business is still small and dependent on one product, which it sells at a high price, and it faces competitors developing their own treatments for the same disease. Its fortunes rest on how many patients can get access to Vyjuvek, whether insurers pay, and whether its other experimental programs work.
Saved answer · last researched 10 Nov 2026