CRISPR, ViaCyte beat Vertex to the clinic with diabetes cell therapy that evades the immune system
The race to develop a cell therapy for type 1 diabetes is heating up, and the team at ViaCyte and CRISPR Therapeutics appears to have pulled ahead by a hair.
ViaCyte announced Tuesday it’s putting a new therapy in the clinic derived from “off-the-shelf,” gene-edited, immune-evasive stem cells, the first time such a treatment will be tested in humans. The move comes after its clinical trial application was accepted by Canadian regulators, and patient enrollment is expected to start by the end of the year.
“This approach builds on previous accomplishments by both companies and represents a major step forward for the field as we strive to provide a functional cure for this devastating disease,” ViaCyte CEO Michael Yang said in a statement.
Both squads had tested T1D cell therapies in humans before, but there’s a particular distinction with ViaCyte and CRISPR’s new program. Previously, the in-human treatments had used infused, naked, lab-grown islet cells that stimulate an immune response, with the body usually killing the therapeutic cells before they can produce insulin.
Those therapies, therefore, likely would have been limited only to patients with severe disease, given the potential need for additional immunosuppressive medicines. However, Tuesday’s news involves cells engineered to be better at naturally avoiding the immune system’s triggers.
It’s one of a few different approaches the ViaCyte/CRISPR and Vertex teams are attempting. ViaCyte had previously tried encapsulating the cells in a companion device, but that method failed miserably — most patients didn’t see the cells engraft and almost no change to their diseases.
The immune-evasive stem cell plan also comes after ViaCyte revealed data from a stem cell transplant patient, who required immunosuppressive treatment, earlier this year. Though extremely limited, experts said at the time they provided the first proof-of-concept for that approach, potentially providing a path to a functional cure for both type 1 and type 2 diabetes.
It remains to be seen how ViaCyte and CRISPR’s new study will turn out, but Vertex isn’t sitting idly by. Last month, the biotech released its own data for the immunosuppressive-required method in one patient, reporting their need for insulin injections vanished almost entirely after suffering five nearly life-threatening low blood sugar episodes in the year before treatment.
And though Vertex and CRISPR are longtime collaborators, they’re competing against each other in this race. CRISPR originally partnered with ViaCyte in 2018 to develop off-the-shelf gene and cell therapies for diabetes, with Tuesday’s news coming from their first program together.