Inês Martins, PhD,  managing science editor—

The gene therapy company Krystal Biotech has received a $700,000 equity-based award from the Epidermolysis Bullosa Research Partnership (EBRP) and the Epidermolysis Bullosa Medical Research Foundation (EBMRF) to continue developing a treatment for dystrophic epidermolysis bullosa (DEB). EBRP and EBMRF are nonprofit organizations dedicated to funding medical research in…

Abeona Therapeutics, a biopharmaceutical developing new gene therapies for rare diseases, announced the opening of the first anticipated commercial gene therapy manufacturing facility in Cleveland, Ohio. The facility is named The Elisa Linton Center for Rare Disease Therapies and will have the capacity to produce advanced gene and cell…

Fibrocell Science recently reported positive interim results from its Phase 1/2 clinical trial testing FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). FCX-007 is a gene therapy product candidate, developed in collaboration with Intrexon Corporation, for the treatment of RDEB, a congenital and progressive orphan skin disease…

The U.S. Food and Drug Administration (FDA) recently granted orphan drug status to ProQR Therapeutics‘ investigational drug QR-313, developed for the treatment of dystrophic epidermolysis bullosa (DEB). A first-in-class ribonucleic acid (RNA)-based oligonucleotide, QR-313 was designed to address the underlying cause of DEB due to mutations in…