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Type 1

Deal Boosts Gene-Editing Technology Aiming to Cure Type 1 Diabetes

In the latest move towards finding a stem cell-based cure for type 1 diabetes, Vertex announced a new agreement with CRISPR Therapeutics. This allows Vertex to use established gene-editing technology to develop safer stem cell therapies for T1D.

Vertex hailed its new agreement with CRISPR Therapeutics on March 27 – a move that could accelerate the development of one of the most promising potential cures for type 1 diabetes, which hopes to replace damaged insulin-producing cells with new ones, without needing drugs to suppress the recipient’s immune system.

With the deal, Vertex can now use CRISPR gene-editing technology to develop insulin-producing cells, derived from stem cells, that avoid being attacked by a recipient’s immune system. CRISPR is a Nobel Prize-winning technology that allows researchers to alter the genetic code (or DNA) of a cell with extreme precision. It’s one of the most common methods used to create genetically modified organisms, agricultural products, and certain medications.

When a person with type 1 receives a beta cell transplant with one of Vertex’s existing stem cell therapies, the implanted cells carry their own unique immune “signature” in the genetic code, which differs from that of the recipient. This is why the body’s immune system regards the cells as foreign and dangerous, and will target them for destruction in much the same way a person’s own beta cells are targeted for destruction when they have type 1.

Using the CRISPR technology to alter the DNA of the stem cells, researchers may be able to create beta cells that avoid recognition by the immune system, evading the immune system attack completely.

While Vertex is in the early stages of developing this gene-edited stem cell therapy for type 1 diabetes, the new project builds on ongoing efforts to improve the safety and tolerability of these therapies. Vertex has shown that its stem cell-derived beta cells, when protected from the immune system, have the potential to improve time in range to more than 99% in someone with T1D.

In addition, ViaCyte, another company working on T1D stem cell therapies that was acquired by Vertex in 2022, previously formed a partnership to use CRISPR technology and introduced its genetically-modified stem cells into clinical trials, which are currently ongoing. In the March 27 announcement, Vertex said that the collaboration between CRISPR and ViaCyte will continue  as these gene-edited stem cell therapies move through clinical trials. The new agreement with CRISPR will not affect clinical trials or the development of Vertex’s current therapies.

For more about stem cell therapies for T1D and using CRISPR technology, read: