Vertex jumps to TreeFrog to enhance production of Type 1 diabetes cell therapy candidates

Following success in cystic fibrosis and sickle cell disease, Vertex’s next big leap is Type 1 diabetes (T1D). Helping the Boston company develop three pipeline candidates in the indication will be French cell therapy developer and manufacturer TreeFrog Therapeutics.

The companies announced their partnership on Tuesday, with Vertex licensing exclusive use of TreeFrog’s cell manufacturing platform, C-Stem, which will optimize production by allowing cells to grow “exponentially in 3D,” Vertex said.

“The technology will enhance Vertex’s ability to generate large amounts of fully differentiated cells for its portfolio of cell therapies,” the companies wrote.

Terms of the milestone-heavy partnership include a $25 million payment upfront, with $215 million available related to the development of a process to produce differentiated islet cells. TreeFrog can also gain sales royalties and another $540 million when clinical, regulatory and commercial milestones are met on up to two future products.

Vertex will apply TreeFrog’s technology to its primary T1D candidate VX-880, which successfully completed a phase 1/2 trial last year. Both VX-880 and VX-264 are stem-cell-derived, fully differentiated insulin-producing islet cell therapies.

“Our expectation is that C-Stem will enhance our ability to generate large amounts of fully differentiated cells for our portfolio of T1D cell therapies,” a Vertex spokesperson wrote in an email. “We will be implementing TreeFrog’s technology into our current processes to potentially enhance stem cell production and scale-up.”

In addition to developing a pipeline of cell therapy products—including a lead candidate for Parkinson’s disease—six-year-old TreeFrog licenses out its C-Stem technology. 

Three years ago, the company touted C-Stem as a “leap forward in cell manufacturing,” allowing companies to mass-produce off-the-shelf cell therapies with standardized quality.

“This is a fantastic opportunity to apply and further develop our C-Stem technology,” Frédéric Desdouits, Ph.D., CEO of TreeFrog, said in a release. “This collaboration with Vertex advances our business strategy of helping bring best-in-class cell therapies to millions of people—both through our own therapeutic programs and with best-in-class partners like Vertex.”

Last year, Vertex struck a manufacturing deal with Lonza in which the companies are co-investing in a new facility in Portsmouth, New Hampshire, which will employ up to 300 people. The plant will support the scale-up and manufacture of Vertex’s two clinical T1D programs.

“Our scale up at Lonza is ongoing and we will determine the right time to incorporate TreeFrog’s technology into that process as the collaboration progresses,” the Vertex spokesperson said.

Last year, Chicago startup CellTrans scored the first approval for a T1D cell therapy when the FDA signed off on Lantidra (donislecel). Stem cell treatments—like those under development by Vertex—are believed to be more reliable and healthier than those from deceased donors, as is the case with Lantidra.