KB103 is a gene therapy being developed by Krystal Biotech to treat dystrophic epidermolysis bullosa (DEB), a rare and incurable form of EB. The condition is characterized by blistering of the skin due to a lack of type 7 collagen (COL7) caused by mutations in the COL7A1 gene.

How KB103 works

COL7 keeps the skin’s upper layer, the epidermis, attached to its lower layer, the dermis. A lack of COL7 causes the epidermis to separate from the dermis, leading to blisters. KB103 delivers a healthy or functional copy of the human COL7A1 gene directly to the patient’s skin cells, using a modified virus as a carrier to allow them to produce normal COL7 protein, therefore preventing skin blistering.

KB103 in clinical trials

A randomized, double-blind, placebo-controlled Phase 1 study (NCT02690961) investigated the safety, tolerability and pharmacokinetics of multiple doses of KB103 in healthy volunteers. The study’s primary outcome measure was to evaluate the incidence of adverse events of KB103 compared to placebo. The now-completed study included 24 healthy volunteers aged 18 to 45. Results have not yet been released.

Other details

KB103 is Krystal’s lead candidate for the treatment of DEB. The company also intends to develop KB103 as a potential treatment for recessive dystrophic epidermolysis bullosa — the most severe form of DEB — as well as dominant dystrophic epidermolysis bullosa, the disease’s milder form.

The U.S. Food and Drug Administration has already awarded KB103 orphan drug status. If approved, the therapy may reduce the time required to treat DEB and well as costs for patients diagnosed with the disease.


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