Krystal Biotech

First-of-its-kind gene therapy restores vision in DEB patient

27 July 2023

A dystrophic epidermolysis bullosa (DEB) patient at the Bascom Palmer Eye Institute, University of Miami, US, has regained his vision after being the first person to receive a formulation of gene therapy Vyjuvek suitable for the eyes. DEB is a rare genetic condition that makes the skin and mucous membranes so fragile that any friction […]

FDA approves first gene therapy for rare skin disorder

29 May 2023

The US Food and Drug Administration has approved Krystal Biotech’s Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB). It is approved for treating wounds in patients six months of age and older with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. “Vyjuvek […]

Regenerative medicine switches to VEGF as top drug target

5 April 2023

Regenerative medicines in early-stage development have seen a change in drug targets compared to therapies in late-stage development. According to analysts GlobalData, early-stage therapies are now focused on vascular endothelial growth factor (VEGF) as the top drug target, with three drugs currently in development. Surprisingly, the majority of the targets for drugs in early-stage development […]

Child with cystic fibrosis on ventilator

Gene therapy for cystic fibrosis receives EC orphan drug designation

25 January 2023

The European Commission has granted orphan designation for Krystal Biotech’s KB407 for the treatment of cystic fibrosis (CF). KB407 is an investigational, redosable gene therapy designed to correct the underlying cause of cystic fibrosis by delivering two copies of the CFTR gene directly to the airway epithelial cells when delivered via a nebuliser. By inducing […]