Krystal Biotech’s topical gene therapy Vyjuvek in an eyedrop formulation has restored the vision of a 13-year-old boy with the genetic condition dystrophic epidermolysis bullosa that scarred his eyes, according to a case brief published Thursday in The New England Journal of Medicine.
Tag Archive for: Vyjuvek
When you hear the phrase “gene therapy,” what comes to mind? A revolutionary cure for an exotic disease? A risky experimental approach that messes with nature? Something in between?
The topical treatment Vyjuvek got the FDA’s greenlight, making it the first redosable gene therapy and the first therapeutic for the rare skin disease dystrophic epidermolysis bullosa.
