Krystal Biotech Inc (KRYS)

[Monksdream]

From Cathie Wood
A Novel Gene Therapy Promises Relief For A Rare Genetic Skin Disorder
ARK Invest_Illustration_Pierce Jamieson_Final_Circle 400 px By Pierce Jamieson | @PierceARK
Research Associate
Recently, the US Food and Drug Administration (FDA) approved Krysta Biotech’s (KRYS) groundbreaking advance1 in gene therapy, Vyjuvek, to treat wounds in patients suffering from dystrophic epidermolysis bullosa (DEB), a rare and serious genetic skin disorder caused by mutations in the collagen Type VII alpha 1 chain (COL7A1) gene. As the first FDA-approved gene therapy for DEB, Vyjuvek is a significant breakthrough.

Vyjuvek uses a genetically modified HSV-1 to deliver normal copies of the COL7A1 gene to wounds, restoring the integrity of the skin with fibrils that hold skin layers together. Treated with Vyjuvek, 65% of wounds closed completely.