Just two weeks after launching the first company-backed trial of a CRISPR treatment, CRISPR Therapeutics inked a deal with ViaCyte to develop off-the-shelf, gene-edited stem cell therapies for diabetes. ViaCyte is picking up $15 million upfront, with a potential $10 million to follow.
ViaCyte’s lead asset, PEC-Direct, is designed to treat diabetes by administering stem cell-derived pancreatic progenitor cells in an implantable device. Once delivered, the cells mature into human pancreatic cells, including insulin-secreting beta cells. There’s a snag though: the treatment must be given with immunosuppressive drugs to prevent rejection. Because of this, the treatment is being developed for high-risk Type 1 diabetes patients, those who experience severe hypoglycemic episodes, whose blood sugar levels swing up and down, or who are less able or unable to spot symptoms of low blood sugar.
CRISPR and ViaCyte plan to clear this hurdle first by developing an immune-evasive stem cell line and eventually creating an allogeneic stem cell-derived treatment. Editing the stem cells with CRISPR could protect them from the patient’s immune system, the companies said. Specifically, they are seeking to edit immune-modulatory genes in the stem cell line used to produce the therapy. CRISPR is already working on this with its allogeneic CAR-T programs and in August started a human trial of its autologous gene-edited hematopoietic stem cell therapy with partner Vertex.
Under the deal, which goes through commercialization, the pair will share costs and profits. ViaCyte could also “under certain circumstances” earn $10 million from CRISPR in the form of a convertible promissory note, but the companies did not detail what those circumstances would be.
“Creating an immune-evasive gene-edited version of our technology would enable us to address a larger patient population than we could with a product requiring immunosuppression,” said ViaCyte CEO Paul Laikind, Ph.D., in a statement. “We also believe that this approach may have many other applications which we and CRISPR may explore in the future.”
In a similar tack, researchers from the Salk Institute are investigating the use of CRISPR-edited liver cells to treat hemophilia B. Patients with the disorder produce very little clotting factor IX, or none at all, which causes potentially life-threatening prolonged bleeding. The team found that the treatment restored the ability of mice with hemophilia B to form blood clots for a year. The hope is that this one-and-done treatment might replace the frequent injections of clotting factors that are currently used to treat hemophilia B.