Vertex to pay $25M upfront to scale cell manufacturing technology

Vertex Pharmaceuticals will pay TreeFrog Therapeutics $25 million to license TreeFrog's cell manufacturing technology, C-Stem, to increase production of Vertex's cell therapies for type 1 diabetes (T1D).

The companies will collaborate to scale up Bordeaux, France-based TreeFrog's cell manufacturing process to produce and amplify cells for Vertex's T1D cell therapies, according the announcement. C-Stem is designed to mimic the natural microenvironment, allowing cells to grow exponentially in 3D. Scaling up the cell-manufacturing process will enhance Vertex's ability to generate large amounts of fully differentiated cells for its portfolio of T1D cell therapies.

"Our goal is to transform the treatment of T1D, and our stem cell-derived, fully differentiated islet cell VX-880 Ph 1/2 program has demonstrated this potential," said Dr. Morrey Atkinson, Vertex's executive vice president, chief technical operations officer, and head of biopharmaceutical sciences and manufacturing operations, in the announcement.

As part of the deal, Vertex may pay up to $215 million in milestones achieved in connection with the development of a scaled-up process for fully differentiated islet cells. Terms of the transaction also noted that TreeFrog is eligible to receive an additional $540 million in clinical, regulatory, and commercial milestones on up to two future products and tiered single-digit royalties.

Boston-based Vertex will fund all research and development costs related to the collaboration, and the company will be responsible for all development and commercialization of its cell therapies, according to the announcement.

For type 1 diabetes, Vertex is pursuing multiple investigational approaches using stem cell-derived islets with the aim of replacing the insulin-producing islet cells that are destroyed in people with T1D.

Among them is VX-880 and another approach, VX-264. A January update from Vertex described VX-880 as an allogeneic, stem cell-derived, fully differentiated, insulin-producing islet cell therapy, used in conjunction with standard immunosuppression, in patients with T1D and impaired awareness of hypoglycemia and recurrent hypoglycemic events. VX-264 encapsulates the same VX-880 cells in a device that is designed to eliminate the need for immunosuppressants, according to Vertex.

Ultimately, the Vertex agreement with TreeFrog aims to bring more cell therapies to the T1D patient population. 

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