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: Krystal Biotech
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.