Canadian Regulators Give Go-Ahead for Diabetes Trial Using CRISPR, ViaCyte’s Gene-Edited Stem Cell-Derived Therapy

Swiss-US firm, CRISPR Therapeutics is a gene-editing company that aims to rapidly translate potential CRISPR/Cas9 gene-editing platforms into therapies for hemoglobinopathies, cancer, diabetes, and other diseases.

In September 2018, it entered a collaboration with ViaCyte Inc. to discover, develop, and commercialize gene-edited allogeneic stem cell therapies for the treatment of diabetes. As per the deal, ViaCyte was eligible to receive an upfront of $15 million either as cash or CRISPR stock, on top of another $10 million in the form of a convertible promissory note.

Earlier today both companies announced that Canadian regulators have approved their Clinical Trial Application (CTA) for VCTX210, an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D).

“With the approval of our CTA, we are excited to bring a first-in-class CRISPR-edited cell therapy for the treatment of type 1 diabetes to the clinic, an important milestone in enabling a whole new class of gene-edited stem cell-derived medicines,” said Samarth Kulkarni, Ph.D., CEO of CRISPR Therapeutics.

“The combination of ViaCyte’s leading stem cell capabilities and CRISPR Therapeutics’ pre-eminent gene-editing platform has the potential to meaningfully impact the lives of patients living with type 1 diabetes.”

Allogeneic Stem Cell Therapy for Diabetes

The insulin hormone produced by one of the cell types in pancreatic islets called beta cells metabolizes glucose and maintains blood glucose levels. However, the beta cells are destroyed by the immune system in patients with T1D and they require insulin injections.

VCTX210 is an allogeneic, gene-edited, stem cell-derived product developed by applying CRISPR Therapeutics’ gene-editing technology to ViaCyte’s proprietary stem cell capabilities. The candidate has the potential to enable a beta-cell replacement product that may deliver durable benefits to patients without requiring concurrent immune suppression.

“Being first into the clinic with a gene-edited, immune-evasive cell therapy to treat patients with type 1 diabetes is breaking new ground as it sets a path to potentially broadening the treatable population by eliminating the need for immunosuppression with implanted cell therapies,” said Michael Yang, President, and CEO of ViaCyte.

“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.”

Meanwhile, the candidate from CRISPR and ViaCyte is not without competition. Boston-based Vertex is also trialing its VX-880, an investigational stem cell-derived, fully differentiated pancreatic islet cell replacement therapy for T1D. Last month, the company announced encouraging 90-day data for the first patient dosed in a Phase 1/2 trial.

Author

Rajaneesh K. Gopinath