Pharma, BioPharma

Vertex Pays CRISPR Therapeutics $100M to Bring Gene-Editing to Type 1 Diabetes

Vertex Pharmaceuticals aims to use CRISPR Therapeutics’ gene-editing technology to develop type 1 diabetes cell therapies that don’t prompt the immune system to reject them. This approach could compete with a Sana Biotechnology program expected to reach its first test in humans this year.

Vertex Pharmaceuticals has two clinical-stage programs for type 1 diabetes, each employing different methods for preventing the immune system from rejecting the cell therapies. For a third program in preclinical development, the drug developer wants to use gene editing to avoid rejection and it has agreed to pay CRISPR Therapeutics $100 million to apply that company’s technology to the effort.

The upfront payment announced Monday gives Boston-based Vertex non-exclusive rights to CRISPR’s gene editing technology to develop these new edited cell therapies for type 1 diabetes. The deal adds to a longstanding relationship between the companies. Vertex is already partnered with Zug, Switzerland-based CRISPR on a rare blood disorders therapy that’s made by the ex vivo editing of a patient’s stem cells.

Type 1 diabetes is an autoimmune disorder in which the immune system destroys insulin-producing islet cells of the pancreas. Vertex joined the pursuit of a cell therapy for the disorder with the 2019 acquisition of Semma Therapeutics, a company that was developing a therapy comprised of stem cell-derived human islets. Vertex says this therapy, VX-880, requires standard immunosuppression to protect the implanted cells. This program is currently being evaluated in a Phase 1/2 clinical trial. Last June, the company demonstrated clinical proof of concept for this approach with data for two patients in the study.

For Vertex’s second program, VX-264, insulin-producing islet cells are housed and implanted in an immunoprotective device. While the company has received the green light to proceed with clinical testing in Canada, its application for U.S. trials is still under an FDA clinical hold.

The third approach Vertex is taking involves developing cells that are hypoimmune, meaning they are less prone to prompting an immune response. This research is preclinical, but the company said Monday that using CRISPR’s gene-editing technology could accelerate the development of such cell therapies for type 1 diabetes. If this approach works, it could avoid the need for immunosuppressive therapies that introduce additional complication risks.

“Having successfully demonstrated clinical proof of concept in T1D [type 1 diabetes] in our VX-880 program, we are excited to deepen our relationship with CRISPR Therapeutics with this agreement, which will allow us to further accelerate our goal of generating fully differentiated, insulin-producing hypoimmune islet cells for T1D,” Bastiano Sanna, Vertex’s executive vice president and chief of cell and genetic therapies said in a prepared statement.

Vertex’s potential competition for a hypoimmune cell therapy includes Sana Biotechnology. Seattle-based Sana is developing pancreatic islet cells derived from stem cells. In preclinical research, these islet cells, which were modified to be hypoimmune, were able to survive without immunosuppression. These cells were also able to evade immune responses that could lead to rejection. Results were presented last June during the annual meeting of the International Society for Stem Cell Research.

In its report of 2022 financial results earlier this month, Sana said it expects an investigator-sponsored study testing hypoimmune-modified islet cells transplanted into humans could have data later this year. The company aims to file its own investigational new drug application for this program in 2024.

Vertex has an additional presence in type 1 diabetes through ViaCyte, a company it acquired last fall in a $320 million deal. While ViaCyte has two type 1 diabetes programs that require immunosuppressive therapies, it has a third program that—thanks to gene-editing—potentially will not. This program stems from a collaboration that ViaCyte struck up with CRISPR Therapeutics prior to the company’s acquisition by Vertex. Vertex said the ViaCyte/CRISPR collaboration will continue under the terms of their agreement. That program has reached Phase 1/2 testing.

Under the new agreement between Vertex and CRISPR, the Swiss company could receive up to $230 million in research and development milestone payments, plus royalties from sales of any commercialized hypoimmune cell therapies for type 1 diabetes. The deal does not give CRISPR any stake in any programs in Vertex’s type 1 diabetes drug pipeline, including VX-880 and VX-264.

The previous partnership that Vertex and CRISPR struck to develop a gene-editing therapy for rare blood disorders is making progress. Last November, Vertex began a rolling FDA submission seeking regulatory approval in sickle cell disease and transfusion-dependent beta thalassemia. In its report of 2022 financial results last month, Vertex said it expects the application will be complete by the end of the first quarter of this year.

Photo: David L. Ryan/The Boston Globe, via Getty Images

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