Rare Disease Report

First Patient Dosed with Dystrophic Epidermolysis Bullosa Treatment, KB103

MAY 10, 2018
Krista Rossi
This morning, Krystal Biotech announced that the first patient has been dosed in the Phase 1/2 clinical trial of its drug, KB103, for the treatment of dystrophic epidermolysis bullosa (DEB).

Rare Disease Report recently sat down for an exclusive interview with company CEO, Krish Krishnan, where he shared insight about KB103, which has since been granted Orphan Medicinal Product Designation (OMPD) in Europe and Investigational New Drug (IND) clearance by the US Food and Drug Administration (FDA).

KB103 is a replication-defective, non-integrating viral vector that has been engineered to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. HSV-1 is the replication-deficient, non-integrating viral vector that is able to penetrate skin cells more efficiently than other viral vectors. Due to its high payload capacity, it is able it to accommodate large or multiple genes. Additionally, its low immunogenicity makes it an appropriate choice for direct and repeated delivery to the skin (in the form of a cream).

DEB is a debilitating, genetic condition that causes the skin to be very fragile and blister easily. The slightest of injuries, such as bumping or rubbing, can cause the skin to break and blister. DEB is caused by a mutation in the gene called COL7A1. When mutated, COL7A1 doesn’t produce the required protein collagen VII to create the anchoring fibers that hold the skin together.

Stanford University is hosting the single-center, open-label, placebo-controlled Phase 1/2 trial in which an intra-subject comparison of randomized treatment and control wounds in DEB patients will be evaluated.

The primary endpoint of the study is the assessment of the safety, efficacy, and tolerability of KB103 in individuals with the recessive form of DEB. Wound imaging and analysis of collagen VII expression as well as anchoring fibril formation in the basement membrane zone will serve as primary points of evaluation in relation to efficacy.

“This is an important milestone for us, as it brings us a step closer to offering a meaningful therapeutic option for patients with dystrophic epidermolysis bullosa who do not have a treatment option presently,” commented Suma Krishnan, founder and chief operating officer of Krystal. "We are grateful to our clinical investigators and expert collaborators, who have supported Krystal Biotech and helped guide our efforts. Most of all, we are thankful to the patients and their families affected by DEB for their participation in our trial.”   

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