Vertex Pharmaceuticals and CRISPR Therapeutics' therapy for beta thalassemia and sickle cell disease is awaiting a regulatory decision in the U.S. and Europe, but the cystric fibrosis drug maker is not sitting back. Vertex is now looking to double dip on the gene editing partner's tech to develop Type 1 diabetes therapies.
Historically known for developing a handful of cystic fibrosis treatments, Vertex is now going deeper into diabetes with the $330 million deal, which includes $100 million in upfront cash. The companies will aim CRISPR’s gene editing tech at developing new hypoimmune cell therapies for Type 1 diabetes, according to an announcement March 27. Vertex was sure to note that the deal does not give CRISPR any interest in existing clinical-stage diabetes treatments, VTX-880 and VTX-264, the former of which has created buzz thanks to proof of concept data.
The two companies’ previous collaboration produced exa-cel, a treatment for beta thalassemia and sickle cell disease that's awaiting a decision in the U.S. and Europe. Vertex has also been able to get a glance at CRISPR’s diabetes capabilities, as recently acquired ViaCyte is working with the gene-editing company on a clinical-stage treatment. Vertex says the agreement announced Monday has no impact on the phase 1/2 trial of that therapy, called VCTX211, which remains ongoing.
The beneficiary of CRISPR’s Cas9 gene editing tech will be Vertex’s preclinical hypoimmune stem-cell-derived islet cell assets, which are third in Vertex’s diabetes roster. Vertex’s hope is that tweaking the genetic makeup would allow these cells to be more accepted by a host body, rendering them virtually invisible against the immune system.
Vertex’s clinical-stage diabetes pipeline is led by VTX-880, which also uses stem-cell-derived islet cells but couples the therapy with standard immunosuppression. Vertex revealed proof of concept data from one patient in October 2021 showing that islet cell function had been restored. The second stage of trial recruitment is now complete, and the company plans to release more data later this year.
Version 2.0 of VTX-880, known as VTX-264, is combining the same stem cells with an encapsulation device to help them evade the body’s immune system. The FDA cleared the therapy for a phase 1 trial earlier this month, and the company plans to launch the study in the first half of the year.