Designed to cure diabetes, CRISPR-edited cell replacement therapy enters the clinic

On November 16, 2021, ViaCyte and CRISPR Therapeutics jointly announced that Health Canada has approved a clinical trial application (CTA) for CRISPR gene-edited stem cell-derived therapy VCTX210 for the treatment of type 1 diabetes (T1D)
.

VCTX210 uses CRISPR gene editing to prevent allogeneic cell therapy from being recognized by the patient's immune system

VCTX210 uses CRISPR gene editing to prevent allogeneic cell therapy from being recognized by the patient's immune system for the treatment of patients with type 1 diabetes.

The islet cells derived from the differentiation and expansion of stem cells in vitro will cause immune rejection, which will cause the body’s immune system to attack these cells and reduce the durability of the therapy
.

In order to overcome immune rejection, pancreatic islet cell transplantation usually needs to be combined with immunosuppressive agents

The fully differentiated islet cell therapy derived from allogeneic stem cells developed by Vertex Pharmaceuticals needs to be combined with immunosuppressive therapy to protect islet cells from immune rejection

▲Islet cells modified by gene editing are expected to become the next-generation therapeutic drugs for all diabetic patients who need insulin treatment (photo source: ViaCyte official website)

▲Islet cells modified by gene editing are expected to become the next-generation therapeutic drugs for all diabetic patients who need insulin treatment (photo source: ViaCyte official website)

VCTX210 is derived from ViaCyte's pluripotent stem cell line CyT49
.

Using CRISPR gene editing technology, knock out the protein expression related to T cell attack, and then achieve the purpose of protecting transplanted cells from immune rejection

Dr.
Samarth Kulkarni, CEO of CRISPR Therapeutics, said: “We are excited to advance a potential'first-in-class' CRISPR-edited cell therapy for the treatment of type 1 diabetes to the clinic, which is the development of a new type of gene-edited stem cell-derived An important milestone for drugs.
The combination of
ViaCyte’s stem cell capabilities and the CRISPR Therapeutics gene editing platform is expected to meaningfully affect the lives of patients with type 1 diabetes
.

"

An important milestone in the development of a new class of gene-edited stem cell-derived drugs

Reference materials:

[1] CRISPR Therapeutics and ViaCyte, Inc.

to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes.