CRISPR Therapeutics and ViaCyte dosed their first patient in Phase I clinical trial of a novel gene-edited cell replacement therapy to treat Type 1 Diabetes.
CRISPR Therapeutics and ViaCyte have announced that they dosed their first patient in Phase I clinical trial of novel gene-edited cell replacement therapy to treat Type 1 Diabetes (T1D). The drug, VCTX210, is an investigational, allogenic (patient-derived), gene-edited, immune-evasive, stem cell-derived therapy intended to help T1D patients produce their insulin.
In patients with T1D, the body’s immune system mistakenly attacks healthy pancreatic cells that produce insulin. Patients with T1D must take prescription insulin to maintain normal blood sugar levels because their body can no longer do so on its own.
By combining CRISPR Therapeutics’ gene-editing technology and ViaCyte’s proprietary pluripotent stem cell line, the companies have developed VCTX210, which produces pancreatic cells designed to evade recognition from the immune system, prohibiting them from being destroyed by an aberrant immune system. The cell line will be differentiated into pancreatic endoderm cells, generating glucose-responsive insulin-secreting cells in the patient.
VCTX210 will most likely be delivered via a PEC-Direct pouch, a product candidate developed by ViaCyte. The pouch is designed for blood vessels to enter the device and directly interact with implanted cells. Direct vascularization allows for robust and consistent engraftment. Because the cells are designed to be immune evasive, the companies do not expect them to be rejected by the immune system.
The Phase I clinical trial of VCTX210 will assess the safety, tolerability and immune evasion in patients with T1D. Ten participants will be included in the trial estimated to be completed in December 2022.
CRISPR Therapeutics and ViaCyte have produced this program as part of a strategic collaboration to discover, develop, and commercialize gene-edited stem cell-derived therapies. The companies hope that these therapies can provide a functional cure for people with T1D and those with insulin-requiring Type 2 Diabetes without immunosuppression.
“We are excited to work with CRISPR Therapeutics and ViaCyte to carry out this historic, first-in-human transplant of gene-edited, stem cell-derived pancreatic cells for the treatment of diabetes designed to eliminate the need for immune suppression,” said James Shapiro, M.D., Ph.D., Canada research chair, director of the Islet Transplant Program at the University of Alberta, Canada, and a clinical investigator in the trial. “If this approach is successful, it will be a transformative treatment for patients with all insulin-requiring forms of diabetes.”
