Vertex and CRISPR Collaborate Again with $330 Million Diabetes Gene Editing Deal

With a collaborative sickle cell and beta-thalassemia gene therapy rolling to the finish line, Vertex and CRISPR Therapeutics are teaming up again with their eyes set on type 1 diabetes. Vertex will pay CRISPR $100 million upfront to leverage the company’s revolutionary gene editing technology to develop hypoimmune type 1 diabetes cell therapies.

A New Target, A New Deal

In the most recent deal, which Vertex announced on March 27, the company disclosed that in addition to the $100 million for non-exclusive licensing rights to proprietary gene editing technology, CRISPR would also be eligible for up to $230 million in research and development milestones. CRISPR also has the potential to receive royalties from any products that arise from the licensing agreement. 

With a history of developing groundbreaking therapies together, the duo is moving into new territory in the type 1 diabetes market. Vertex already has substantial experience in the field with clinical prospects like VX-880 and VX-264, but CRISPR’s gene editing technology could bring Vertex’s type 1 diabetes pipeline to the next level. 

Vertex’s lead type 1 diabetes candidate, VX-880, is an allogeneic stem cell-derived, insulin-producing islet cell therapy the company is developing in-house. VX-880 is currently in Phase ½ trials, which the FDA briefly halted due to safety concerns before lifting the clinical hold in July last year.

Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex, said, “Having successfully demonstrated clinical proof of concept in T1D 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.”

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The Previous Deal’s Progress

Vertex and CRISPR have a long history, dating back to 2015 when the companies first collaborated. The initial deal brought CRISPR $105 million upfront in cash and equity investment, with the potential for additional milestones.

Eight years later, much of that hard work could start paying off very soon. With CRISPR’s gene editing technology, Vertex is developing exa-cel, an autologous cell therapy designed to treat sickle cell disease and transfusion-dependent beta-thalassemia.

Recently, CRISPR announced the FDA granted exa-cel a rolling review to treat the diseases and that Vertex will also submit the Biologics License Application (BLA) soon. The companies expect to complete the BLA submission by the end of Q1 this year. 

With exa-cel on the path to approval and VX-880 gaining momentum, Vertex’s new deal with CRISPR is exciting news in the gene editing space. Though it is unclear exactly how Vertex wants to leverage CRISPR’s technology to treat type 1 diabetes, the deal could lay the foundation for a groundbreaking new therapy. 

Author

Reed Slater