RARE Daily

First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes Set for Clinical Trial

November 16, 2021

CRISPR Therapeutics and ViaCyte said that Health Canada has given a green light for the two companies to begin a clinical trial of VCTX210, an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes.

Photo: Michael Yang, president and CEO of ViaCyte

The companies expect to begin patient enrollment by the end of the year.

Type 1 diabetes is an autoimmune disease where a person’s own immune cells mistakenly attack and destroy the insulin-producing beta cells in the pancreas. Without beta cells, the body can no longer produce insulin, a hormone which controls blood glucose levels. If not controlled by insulin, high blood sugar can be life-threatening. While donor islet transplants from cadavers can be highly effective in treating type 1 diabetes, the procedure is only performed on a limited number of patients because of a severe lack of donor material and the requirement for immunosuppression.

ViaCyte and CRISPR Therapeutics formed a partnership in 2018 to discover, develop, and commercialize gene-edited allogeneic stem cell-derived therapies which could be a next-generation functional cure for all insulin-requiring type 1 and type 2 diabetes. ViaCyte’s proprietary CyT49 pluripotent human stem cell line is specifically engineered to avoid destruction by the patient’s immune system, potentially eliminating the need for immunosuppressants. The cell line is differentiated into pancreatic endoderm cells. Because the cells are being designed to be immune-evasive, they are not expected to be rejected by the immune system.

The phase 1 clinical trial of VCTX210 is designed to assess its safety, tolerability, and immune evasion in patients with T1D. This program is being advanced by CRISPR Therapeutics and ViaCyte as part of a strategic collaboration for the discovery, development, and commercialization of gene-edited stem cell therapies for the treatment of diabetes. 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 and has the potential to enable a beta-cell replacement product that may deliver durable benefit 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.”

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.