By Wayne Koberstein, Executive Editor, Life Science Leader magazine
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Many stakes are at play in targeting rare skin, connective tissue, liver, and metabolic disorders.
Castle Creek Biosciences is in late-stage development of gene therapies for rare connective tissue conditions. Its most advanced products, ex vivo therapies based on the company’s proprietary autologous fibroblast technology platform, are localized treatments administered at disease sites. New early-stage products from a recent acquisition have added in vivo, or inside the body, gene therapies, which act against the root causes of targeted diseases. The lead product, D-Fi (coded FCX-007), is now entering late-stage trials for localized treatment of skin blisters and wounds due to dystrophic epidermolysis bullosa (DEB), a rare but horrific inherited condition. Next in line is FCX-013, now in Phase 1/2, treating localized scleroderma. Other ex vivo therapies, including some to treat rare collagen disorders, are in discovery stages tightly focused on rare connective tissue conditions. In vivo gene therapies are in preclinical studies for rare metabolic and liver diseases.