CRISPR, ViaCyte team up to develop stem cell therapies for diabetes

18 September 2018 (Last Updated September 18th, 2018 12:23)

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.

CRISPR, ViaCyte team up to develop stem cell therapies for diabetes
CRISPR’s gene editing technology will be combined with ViaCyte’s stem cell technologies to develop a curative therapy for diabetes. Credit: yourgenome.

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.