Vertex enlists TreeFrog in $780M deal to scale up cell therapies for type 1 diabetes
细胞疗法引进/卖出免疫疗法临床研究基因疗法
Vertex Pharmaceuticals has entered into an exclusive licensing agreement with TreeFrog Therapeutics potentially worth over $780 million to access the French biotech's proprietary C-Stem cell manufacturing technology. The deal aims to enhance Vertex's ability to produce large quantities of fully differentiated islet cells for its portfolio of experimental cell therapies for type 1 diabetes (T1D).
Under the terms, Vertex will make a $25-million upfront payment to TreeFrog and an undisclosed equity investment. TreeFrog is also eligible for up to $215 million in milestones tied to developing a scaled manufacturing process using C-Stem. The agreement includes an additional $540 million in potential clinical, regulatory and commercial milestones across up to two future T1D cell therapy products, plus tiered single-digit royalties on sales.
VX-880 is an allogeneic, stem cell-derived insulin-producing islet cell therapy being investigated as an infusion into the hepatic portal vein. It requires immunosuppressive therapy to protect the islet cells from immune rejection. Vertex unveiled positive data in 2021 and updated results late last year showing that all treated patients continued to see improvements across all glycaemic measures beyond 90 days.
Its pipeline also includes VX-264, which uses a protective surgically-implanted device designed to shield islet cells from immune attack without the use of immunosuppression.
TreeFrog's C-Stem is designed to mimic the natural cell microenvironment, allowing exponential cell growth in a controlled bioreactor setting. According to the French biotech, C-Stem achieved a world-first output of 15 billion cells from a single manufacturing run in 2021. The 3D approach aims to overcome the limitations of traditional 2D cell culture methods that struggle to produce enough viable cells for therapeutic use.
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