Vertex and TreeFrog Therapeutics Enter Licensing Agreement and Partnership on Production of Type 1 Diabetes Cell Therapies

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Vertex Pharmaceuticals, a global biotechnology company, and TreeFrog Therapeutics, a biotechnology company based in France, announced that Vertex has acquired an exclusive license to C-Stem, TreeFrog’s proprietary cell manufacturing technology, in order to optimize production of Vertex’s cell therapies for type 1 diabetes (T1D). The collaboration aims to scale-up TreeFrog’s process to produce and amplify cells for Vertex’s T1D therapies. C-Stem is designed to imitate the natural microenvironment, which allow cells to continuously grow in 3D. Through C-Stem, Vertex can create large amounts of full differentiated cells for T1D cell therapies.

Under the agreement, TreeFrog is set to receive $25 million upfront, an equity investment from Vertex, and up to $215 million in milestones connected to the development of a scaled-up process for fully differentiated islet cells. TreeFrog may also receive an additional $540 million in regulatory, clinical, and commercial milestones on up to two future products and tiered single-digit royalties (1). Vertex has committed to fund all research and development costs for the collaboration, and it will be responsible for all development and commercialization of its 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 Morrey Atkinson, PhD, executive vice president, chief technical operations officer, and head of Biopharmaceutical Sciences and Manufacturing Operations at Vertex, in a Vertex press release. “We’re excited to explore TreeFrog’s C-Stem to scale up stem cell production and deliver for the large number of people living with T1D.”

This is not the first collaboration Vertex has entered into to manufacture its cell therapies. On Dec. 11, 2023, Vertex and RoslinCT, a UK-based cell and gene therapy contract development and manufacturing organization, announced that RoslinCT would manufacture Vertex’s Casgevy, a clustered regularly interspaced short palindromic repeats (CRISPR)-based gene therapy that treats sickle cell disease and β thalassemia. This announcement came shortly after FDA’s approval of Casgevy on Dec. 8, 2023, in which the therapy was granted priority review, orphan drug, fast track, and regenerative medicine advanced therapy designations.

RoslinCT worked closely with Vertex on an adaptive basis during the development of Casgevy, and the collaboration put RoselinCT in a position to support the manufacturing of a large range of highly complex autologous and allogenic cell therapies beyond Casgevy. In a press release, FDA said that Casgevy is “the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.”

References

1. Vertex, Vertex and TreeFrog Therapeutics Announce Licensing Agreement and Collaboration to Optimize Production of Vertex’s Cell Therapies for Type 1 Diabetes. Press Release, Apr. 23, 2024.
2. Mirasol, F. RoslinCT to Manufacture CRISPR-Based Gene Therapy Casgevy. PharmTech.com. Dec. 14, 2023.