Krystal Biotech

This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.

KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to dystrophic epidermolysis bullosa (DEB).

The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI\*ZZ or PI\*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Repeat dosing will be evaluated at the mid dose level. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. In the repeat dose and the high dose cohorts, subjects must wash out from IV AAT for at least 10 days, as applicable.Show more

This study is a non-interventional, observational study that will evaluate the natural history of corneal abrasions in patients with Dystrophic Epidermolysis Bullosa (DEB).

The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc. which have a shared backbone of HSV-1, in participants who received at least one dose of investigational product (IP).Show more

Krystal Biotech is a Pittsburgh gene therapy company founded in 2016 by the husband-and-wife team of Krish Krishnan, its chairman and CEO, and Suma Krishnan, its president of research and development. Its idea is simple to state and was hard to pull off: use a disabled herpes simplex virus (HSV-1) as a delivery vehicle that can carry a full-size healthy gene into cells, can be given again and again because it does not trigger the immune response that limits other gene therapy vectors, and can be applied as a gel, an eye drop, or an inhaled mist rather than an intravenous infusion. Krystal makes everything itself at two facilities in the Pittsburgh area, ANCORIS and ASTRA, and trades on Nasdaq under KRYS.

The proof is VYJUVEK (beremagene geperpavec-svdt), approved by the FDA on May 19, 2023 as the first redosable gene therapy and the first treatment to address the root cause of dystrophic epidermolysis bullosa, a genetic skin disease in which a missing collagen protein (COL7A1) leaves the skin so fragile that it blisters and tears from minor friction. VYJUVEK is a topical gel applied to wounds by a nurse, a caregiver, or the patient at home, and it delivers working copies of the COL7A1 gene to skin cells so they can produce the missing collagen. A September 2025 label update extended it to patients from birth. It is also approved in Europe (April 2025), Japan (July 2025), and the United Kingdom (May 2026). Sales reached $389 million in 2025 and $236 million in the first half of 2026, and the company ended June 2026 with $1.1 billion in cash.

Krystal is now applying the same vector to the eye, the lung, and other skin diseases. KB803, an eye-drop version of VYJUVEK for the painful corneal abrasions many people with dystrophic EB experience, finished enrolling its pivotal IOLITE study in April 2026 with results expected in the fourth quarter of 2026. KB801, an eye drop that produces nerve growth factor, is in a registrational trial for neurotrophic keratitis with data expected around the end of 2026. In the lung, KB407 is an inhaled therapy that delivers the CFTR gene for cystic fibrosis, aimed first at the roughly 10% of patients who cannot take or do not benefit from CFTR modulator pills. In January 2026 Krystal reported that the therapy reached airway cells and produced normal CFTR protein in treated patients; a repeat-dose safety study is underway, and the company plans to agree on a registrational trial design with the FDA in late 2026 and start it in 2027. KB408 delivers the SERPINA1 gene for alpha-1 antitrypsin deficiency and is enrolling a repeat-dose cohort, with results now expected in 2027. KB111 for Hailey-Hailey disease, a rare blistering skin disorder, started its first study in 2026 with early results expected before year end. In oncology, an inhaled version of KB707, which prompts lung tumors to produce immune-stimulating proteins, showed a 31% response rate in combination with pembrolizumab in heavily pretreated lung cancer at ASCO 2026, and a related program was expanded in 2026 to Gorlin syndrome, a genetic condition that causes hundreds of basal cell skin cancers over a lifetime.

One program has gone quiet: KB105 for a form of congenital ichthyosis has not appeared in the company's updates since early 2025 and should be considered dormant.

Krystal Biotech has 1 FDA-approved drug with a rare disease use that we track, as of October 2026.

Vyjuvek (beremagene geperpavec-svdt), approved 2023: Epidermolysis Bullosa.

Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Yes, for 1 of its FDA-approved rare disease medicine that we track, each checked on Krystal Biotech's own website.

Vyjuvek: Krystal Connect, 1-844-557-9782. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked September 29, 2026.

Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.

Eligibility is decided by each program, so call the number listed before a prescription is filled.