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… Read More
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… Read More
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… Read More
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… Read More
