Vertex Pharmaceuticals and Lonza to build facility for T1D cell therapies

Vertex Pharmaceuticals announced a strategic collaboration to support the manufacture of Vertex’s portfolio of investigational stem cell-derived, fully differentiated insulin-producing islet cell therapies for people with T1D, currently focusing on the VX-880 and VX-264 programs that are currently in clinical trials. Vertex’s first clinical program, VX-880, has already demonstrated clinical proof-of-concept. In new data released last week, all six patients treated with VX-880 engrafted islet cells, produced endogenous insulin and had improved glycemic control, while reducing or eliminating insulin use. Patients with greater than 90 days of follow-up had elimination of severe hypoglycemic events in the evaluation period. Vertex’s second approach, VX-264, utilizes the same fully differentiated insulin-producing islet cells as in VX-880 and is being studied in a Phase 1/2 clinical trial. In VX-264, the cells are encapsulated in a proprietary device designed to shield the cells from the body’s immune system, thereby eliminating the need for immunosuppression. In a third approach, Vertex’s hypoimmune program, the same cells are edited to cloak them from the immune system; this program continues to progress in preclinical development. Under the terms of the collaboration, Vertex and Lonza will partner in the process development and scale-up for the manufacturing of the product portfolio and co-invest to build a dedicated new facility in Portsmouth, New Hampshire. Operated by Lonza, the facility will span more than 130,000 square feet and is anticipated to create up to 300 new jobs at peak capacity. Construction is scheduled to begin later this year.