- Vertex Pharmaceuticals will license CRISPR Therapeutics’ gene editing technology to accelerate its development of therapies for Type 1 diabetes, further expanding the two companies’ collaboration.
- Under an agreement announced Monday, CRISPR will get $100 million upfront and is eligible to receive an additional $230 million for research and development milestones. Vertex and CRISPR are already partnered on a possible cure for the blood disorders sickle cell disease and beta thalassemia.
- Both companies will continue efforts to develop stem cell-derived therapies for Type 1 diabetes. Vertex is testing a treatment it acquired from Semma Therapeutics, and both companies have been working with ViaCyte — CRISPR through a collaboration and Vertex through its acquisition of ViaCyte last year.
Diabetes is a newer target for Vertex as it explores other areas beyond cystic fibrosis, for which it’s developed four approved medicines. It spent $1 billion for Semma in 2019, and two years later began testing its therapy, known as VX-880, in humans.
Unlike the more common Type 2 diabetes that develops gradually in adults who have risk factors like obesity, Type 1 is a genetic condition usually diagnosed at a young age, in which the immune system attacks the insulin-making cells in the pancreas.
VX-880 has shown promising results, but testing has not been without hiccups. The Food and Drug Administration stopped Vertex from testing a higher dose about a year ago, before allowing it to proceed two months later.
Last week, the FDA cleared Vertex to begin early clinical testing of another treatment, called VX-264, that surgically implants the same stem cell-derived pancreatic islet cells as VX-880, but within a device that shields them from the body’s immune system.
Vertex said it will use CRISPR’s gene editing to develop cell therapies that are more resistant to rejection by a body’s immune system.
“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,” said Bastiano Sanna, Vertex’s chief of cell and genetic therapies, in the statement Monday.
Vertex’s latest deal with CRISPR is smaller than the two companies’ partnership on blood disorders struck in 2021, which paid the gene-editing biotech $900 million upfront.
Viacyte and CRISPR are also testing their treatment, known as VCTX210, in a trial that began a year ago. The deal announced Monday does not involve this separate program, nor does CRISPR obtain any interest in Vertex’s Type 1 diabetes programs VX-880 and VX-264 through the deal.