Assessing Gene Therapy in Recessive Dystrophic Epidermolysis Bullosa

Video

Matthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed the company and its pipeline.

“We have the lentiviral vectors as a backbone that we can leverage both on the ex vivo and in vivo side, so we have broader therapeutic areas that we can access. We're really able to dial in and optimize the right therapy for treating the disease in question. Typically, ex vivo based approaches are for more chronic treatments in nature and are more disease-modifying, whereas the in vivo lentiviral approach allows us to develop curative approaches. So, depending on the disease, we can pick the right approach and we think that gives us a distinct advantage.”

Castle Creek Biosciences is developing cell and gene therapies for rare dermatologic diseases and now, following the acquisition of Novavita Thera, rare metabolic and liver diseases.1 The company is using lentiviral vectors in both the in vivo and ex vivo setting with their dual cell and gene therapy platform.

Castle Creek’s lead program is dabocemagene autoficel (FCX-007, D-Fi), a gene therapy for the potential treatment of recessive dystrophic epidermolysis bullosa (RDEB). D-Fi is currently being assessed in a phase 3 trial (NCT04213261) that plans to recruit up to 24 participants with RDEB.2

GeneTherapyLive spoke with Matthew Gantz, president and chief executive officer, Castle Creek, to learn more about the company, the indications they are targeting, and the dual in vivo and ex vivo platform they leverage. He also discussed D-Fi for RDEB and the phase 3 study currently recruiting as well as how the Novavita Thera acquisition and research collaboration with Mayo Clinic has broadened Castle Creek’s pipeline.

REFERENCES
1. Castle Creek Biosciences acquires Novavita Thera to expand innovative cell and gene therapy platform. News release. Castle Creek Biosciences. January 10, 2022. https://castlecreekbio.com/castle-creek-biosciences-acquires-novavita-thera-to-expand-innovative-cell-and-gene-therapy-platform/
2. Castle Creek Biosciences awarded FDA orphan products development grant to support DeFi-RDEB, a pivotal phase 3 study of FCX-007 investigational gene therapy for recessive dystrophic epidermolysis bullosa. News release. Castle Creek Biosciences. October 21, 2021. https://castlecreekbio.com/castle-creek-biosciences-awarded-fda-orphan-products-development-grant-to-support-defi-rdeb-a-pivotal-phase-3-study-of-fcx-007-investigational-gene-therapy-for-recessive-dystrophic-epidermolysis-bull/
Recent Videos
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
David Porter, MD, the director of cell therapy and transplant at Penn Medicine
Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Ben Samelson-Jones, MD, PhD, assistant professor pediatric hematology, Perelman School of Medicine, University of Pennsylvania and Associate Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Related Content
© 2025 MJH Life Sciences

All rights reserved.