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CRISPR and ViaCyte to develop stem cell therapies for diabetes

by JMC on September 18, 2018

Swiss gene editing company CRISPR Therapeutics and US-based regenerative medicine firm ViaCyte have formed a strategic alliance to discover, develop and commercialise stem cell therapies for diabetes patients.

The partners will focus on formulating gene-edited allogeneic stem cell therapies as curative treatment. Together, they intend to develop an immune-evasive stem cell line, which would mark the initial step for an allogeneic stem-cell derived product.

As part of the collaboration, CRISPR’s gene editing technology will be combined with ViaCyte’s stem cell technologies to devise a beta-cell replacement candidate with durable benefit.

ViaCyte candidate PEC-Direct, which is being assessed in clinic, uses a non-immunoprotective delivery device for direct vascularisation of the cell therapy. However, the patient’s immune system reacts to this and the product will require long-term immunosuppression in order to avoid rejection.

Due to the immune reaction, PEC-Direct is currently being developed to treat a subset of type-1 diabetes patients who are at high risk for acute complications.

CRISPR gene editing has the potential to protect the transplanted cells from the patient’s immune system via ex-vivo editing of immune-modulatory genes within the stem cell line that is used to generate the pancreatic-lineage cells.

The combination of this gene editing and stem cell technologies is expected to avoid triggering reactions, such as from the immune system.

CRISPR Therapeutics CEO Samarth Kulkarni said: “We believe the combination of regenerative medicine and gene editing has the potential to offer durable, curative therapies to patients in many different diseases, including common chronic disorders like insulin-requiring diabetes.

“Partnering with ViaCyte will allow us to accelerate our efforts in regenerative medicine, an area that we believe will provide a variety of longer-term opportunities for our company.”

After completion of studies and identifying a product candidate, CRISPR Therapeutics and ViaCyte will jointly carry out further development and commercialisation globally.

Following agreement execution, CRISPR Therapeutics will pay $15m to ViaCyte, which also holds an option for additional $10m as a convertible promissory note.

Source: CRISPR and ViaCyte to develop stem cell therapies for diabetes

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