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Vertex’s clinical trials of lab-grown insulin-making cells 

Vertex Pharmaceuticals is conducting clinical trials where participants with type 1 diabetes receive a dose of insulin-producing cells. These cells were originally developed by Professor Doug Melton, a researcher funded by Breakthrough T1D.
Content last reviewed and updated: 13.12.2024

Vertex Pharmaceuticals logo graphic

Stem cell therapies

Beta cells are the insulin making cells in our pancreas, located within clusters called islets. Scientists around the world, many funded by Breakthrough T1D, are growing islet cells in their labs from stem cells, a type of cell which can be turned into other cell types. Using specific signalling molecules, the stem cells are instructed to develop into islets.

Vertex Pharmaceuticals is a biotechnology company that is now giving these islets to people with type 1 in clinical trials. This new type of treatment is known as stem cell therapy.

Vertex clinical trials

Vertex is currently running two clinical trials of stem cell therapies.

The VX-880 clinical trial, where people with T1D are given lab-grown islets and immunosuppressants.

The VX-264 clinical trial, testing lab-grown islets inside a protective casing in people with T1D. The casing aims to keep the cells safe from the immune system, preventing the need for immunosuppressants.

You can stay up to date with news about the type 1 diabetes clinical trials that Vertex is running on our news page.

How is Breakthrough T1D involved in this research?

For decades we have been funding stem cell research with the goal of replacing the insulin-producing cells lost in type 1 with lab-grown versions. A key Breakthrough T1D-funded researcher in this area is Professor Doug Melton. In 2015, Doug founded a company called Semma Therapeutics, named after his children Sam and Emma, who both live with type 1.

We invested in Semma through the T1D Fund and Vertex bought the company in 2019. When Vertex acquired Semma, Doug started working at Vertex and his lab-grown islets are the cells being used in both clinical trials.

Type 1 Diabetes Cell Manufacturing Facility

Vertex has partnered with a company called Lonza to open a Type 1 Diabetes Cell Manufacturing Facility, in Portsmouth, New Hampshire. The Facility will support the high manufacturing requirements of large-scale clinical trials of lab-grown islet cells.

Vertex and Lonza also plan for the facility to produce the cells as a commercially available product once testing is complete. This multi-million-dollar investment is important because it means that Vertex has the capacity to produce islet cells on a huge scale. If results from their clinical trials continue to be positive, they will be able to expand to more countries with many more people.

Related content

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Vertex Pharmaceuticals logo graphic

The VX-880 clinical trial

This trial gives lab-grown islets and immunosuppressants to people living with T1D.

Read more
Islet cell in protective casing from Vertex VX-264 clinical trial.

The VX-264 clinical trial

This trial test lab-grown islets inside a protective casing in people living with T1D.

Read more
A photo down a microscope of beta cell regeneration.

Cure research

Learn about how researchers are trying to find a cure for type 1.