Krystal Biotech Doses First Patient in Phase 1/2 trial of KB103 Gene Therapy for the Treatment of Dystrophic Epidermolysis Bullosa

May 11, 2018

Krystal Biotech Inc., (“Krystal”) (NASDAQ:KRYS), announced on 5/11/18 that the first patient has been dosed in the Phase 1/2, first in-human trial of KB103, a first-in-class topical gene therapy for the treatment of dystrophic epidermolysis bullosa (DEB).

DEB is a chronic, progressive and incredibly painful skin disease caused by mutations in the gene coding for type VII collagen, or COL7. As a result of mutated COL7, DEB patients' skin is incredibly fragile, resulting in blistering or skin loss at the slightest friction. There are currently no approved treatments for DEB.

“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,” said 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.

KB103 is Krystal’s lead product candidate that seeks to use gene therapy to treat dystrophic epidermolysis bullosa, or DEB, an incurable skin blistering condition caused by a lack of collagen in the skin. KB103 is a replication-defective, non-integrating viral vector that has been engineered employing Krystal’s STAR-D platform to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. HSV-1 is Krystal’s replication-deficient, non-integrating viral vector that can penetrate skin cells more efficiently than other viral vectors. Its high payload capacity allows it to accommodate large or multiple genes and its low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.

Krystal has two other candidates in preclinical development.

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