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

▎ WuXi AppTec content team editor 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
.

The phase 1 clinical trial of VCTX210 aims to evaluate its safety, tolerability and immune escape characteristics in patients with type 1 diabetes, and it is expected to start patient registration before the end of the year
.

The press release states that this is the first gene-edited cell replacement therapy to enter clinical trials 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
.

For example, 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
.

PEC Direct (VC-02), developed by ViaCyte, encapsulates the islet progenitor cells derived from stem cells in a "pocket" that can be implanted under the skin of a patient.
This graft allows blood vessels to grow into the "pocket", and implant cells.
Direct interaction
.

These cells can respond to blood sugar levels and secrete insulin and other pancreatic hormones
.

Since the implanted cells will cause the immune system to respond, patients also need to use immunosuppressive regimens to protect the cells
.

▲Islet cells modified by gene editing are expected to become the next-generation therapeutic drug for all diabetic patients who need insulin therapy (picture 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
.

"References: [1] CRISPR Therapeutics and ViaCyte, Inc.
to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes.
Retrieved November 16, 2021, from https://crisprtx.
gcs-web .
com / news-releases / news- release-details / crispr-therapeutics-and-viacyte-inc-start-clinical-trial-first DISCLAIMER: Advances in biomedical research on global health WuXi team focused content presentation
.

This article only for For the purpose of information exchange, the opinions in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views
in the
article .
This article is not a treatment plan recommendation
.

If you need treatment plan guidance, please go to a regular hospital
.