RDEB is a devastating, rare genetic blistering disease with no FDA-approved treatments currently available.

The U.S. Food and Drug Administration (FDA) Office of Orphan Products Development awarded Castle Creek Biosciences a $1.825 million research grant to support the Phase 3 development program of the investigational gene therapy FCX-007 for treatment of recessive dystrophic epidermolysis bullosa (RDEB).

"We are honored to be awarded this highly competitive research grant for the ongoing clinical development of FCX-007, which has the potential to transform the lives of people suffering from dystrophic epidermolysis bullosa (DEB)," says Mary Spellman, M.D., Chief Medical Officer of Castle Creek Biosciences, and Principal Investigator for the grant, in a news release.  "We are committed to advancing our study of FCX-007 to develop a durable personalized therapy for the localized treatment of chronic wounds due to RDEB and will continue to work closely with the FDA as our program progresses."

 The Congressionally funded Orphan Products Development Grants Program is designed to enhance the development of medical products for patients with rare diseases.   Recessive dystrophic epidermolysis bullosa (RDEB) is a progressive, devastatingly painful and debilitating, rare genetic disorder, and one of the most chronic and severe forms of DEB. 

 The FDA has granted Orphan Drug designation to FCX-007 for the treatment of DEB. In addition, FCX-007 has been granted Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy designations by the FDA for treatment of RDEB.

About FCX-007

Castle Creek Biosciences' dabocemagene autoficel (FCX-007, D-Fi) is being developed to address the deficiency of functional type VII collagen protein (COL7) in patients with dystrophic epidermolysis bullosa (DEB). FCX-007 is comprised of autologously-derived dermal fibroblasts genetically modified with a lentiviral vector containing the COL7A1 gene to express COL7. FCX-007 is locally administered by injection directly into the papillary dermis of persistent and non-healing recurrent wounds of DEB where the COL7 protein can support the formation of anchoring fibrils in the skin. 

About the Phase 3 Study of FCX-007 (DeFi-RDEB)

DeFi-RDEB is a multi-center, within-patient randomized, controlled, open-label, Phase 3 clinical trial of FCX-007 designed to enroll approximately 24 people living with RDEB. Each participant's target wounds are paired and then randomized to receive FCX-007 or remain untreated. Up to three target wound pairs are identified for each participant. The primary outcome measure is complete wound closure of the first wound pair at week 24. Currently, there are five trial locations recruiting participants including Stanford University in Stanford, California; Children's Hospital Colorado in Aurora, Colorado; Dell Children's Medical Group in Austin, Texas; Solutions Through Advanced Research, Inc. in Jacksonville, Florida; and Mayo Clinic in Rochester, Minnesota.