Vertex and Lonza Collaborate to Build Dedicated Manufacturing Facility for Diabetes Cell Therapies

‍Vertex Pharmaceuticals and Lonza have recently announced a strategic collaboration aimed at supporting the manufacturing of Vertex’s portfolio of investigational stem cell-derived, fully differentiated insulin-producing islet cell therapies for people with Type 1 Diabetes.

The 130,000ft² site will be run by Lonza and will create approximately 300 new jobs.

Briefing

• Vertex Pharmaceuticals and Lonza collaborate to manufacture investigational stem cell-derived islet cell therapies for Type 1 Diabetes (T1D) patients.
• Promising results from Vertex’s VX-880 program show engraftment of islet cells, insulin production, and improved glycemic control in T1D patients.
• A dedicated manufacturing facility will be constructed in New Hampshire, operated by Lonza, to support the efficient production of Vertex’s T1D cell therapies.

Manufacturing Partnership

Under the terms of their collaboration, Vertex and Lonza will partner in the process development and scale-up for the manufacturing of Vertex’s T1D cell therapy portfolio. To support this effort, the companies will co-invest in the construction of a dedicated manufacturing facility in Portsmouth, New Hampshire. The facility, spanning over 130,000 square feet, will be operated by Lonza and is expected to create up to 300 new jobs at peak capacity. Construction is scheduled to begin later this year.

The dedicated manufacturing facility will complement Lonza’s existing cell and gene technologies manufacturing network, which supports the development and commercialisation of emerging therapies. Lonza’s scientific, regulatory, and manufacturing expertise, combined with Vertex’s innovative T1D cell therapies, is anticipated to accelerate the development and commercialisation of these potentially transformative treatments

“Vertex’s allogeneic, fully differentiated, insulin-producing islet cells, and the results from the Phase I/II VX-880 programme, represent a scientific breakthrough that offers the potential to transform the treatment of T1D.

“Establishing this strategic partnership with Lonza, a world-class manufacturing organisation, is a critical milestone in this journey and underscores our long-term commitment to patients with T1D.”

Vertex Pharmaceuticals CEO and President Reshma Kewalramani

Three programmes

• The VX-880 programme is Vertex’s first clinical programme focused on T1D cell therapy. In recent data released by Vertex, all six patients treated with VX-880 demonstrated engraftment of islet cells, production of endogenous insulin (C-peptide), and improved glycaemic control. Furthermore, patients with more than 90 days of follow-up experienced a reduction or elimination of severe hypoglycemic events during the evaluation period.

• Vertex’s second approach to T1D cell therapy is the VX-264 programme. This programme utilises the same fully differentiated insulin-producing islet cells as in the VX-880 programme but incorporates a proprietary device that encapsulates the cells. The purpose of this encapsulation is to shield the cells from the body’s immune system, eliminating the need for immunosuppression. The VX-264 programme is currently being studied in a Phase 1/2 clinical trial, and its progress holds great potential for the treatment of T1D.

• Vertex is also advancing a third approach called the hypoimmune programme. This programme focuses on editing the islet cells to make them “invisible” to the immune system. By cloaking the cells, Vertex aims to prevent immune rejection and improve the long-term viability of the therapy. The hypoimmune programme is currently in preclinical development and shows promise for further advancements in T1D cell therapy.

What’s Next

Construction begins later this year for a facility which will provide the infrastructure necessary for the efficient production of these innovative therapies. Simultaneously, Vertex will continue to advance its clinical programmes.