Krystal Biotech has dosed the first patient in a Phase I/II trial evaluating KB103 for the treatment of dystrophic epidermolysis bullosa (DEB).
The single-centre, open-label, placebo-controlled trial is being conducted at Stanford University, California, US.
The trial also aims to analyse the safety and tolerability of KB103 in patients with the recessive form of DEB.
Furthermore, it will examine the efficacy of KB103 through wound imaging and analysis of collagen VII expression and anchoring fibril formation in the basement membrane zone.
Krystal Biotech founder and chief operating officer Suma Krishnan said: “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.”
DEB is a chronic, progressive and extremely painful skin disease caused by mutations in the gene coding for type VII collagen (COL7).
Mutated COL7 makes the DEB patients' skin incredibly fragile, resulting in blistering or skin loss at the slightest friction.
No approved treatments are currently available for DEB.
KB103 is a replication-defective, non-integrating viral vector that intends to use gene therapy to treat DEB.
It has been developed using Krystal’s STAR-D platform to provide functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells.
Last month, KB103 secured an investigational new drug (IND) approval from the US Food and Drug Administration (FDA).