CRISPR Therapeutics unites with ViaCyte to treat diabetes
Gene-editing company CRISPR Therapeutics has partnered with regenerative medicine-focused ViaCyte to focus on diabetes treatments.
Announced on Monday, September 17, the deal will see the two companies collaborate on the discovery, development and commercialisation of gene-edited allogeneic stem cell therapies for the treatment of diabetes.
According to the release, ViaCyte pioneered the approach of generating pancreatic-lineage cells from stem cells and delivering them safely and efficiently to patients.
ViaCyte’s lead candidate, PEC-Direct, uses a non-immunoprotective delivery device that allows for direct vascularisation of the cell therapy.
The companies said that PEC-Direct could potentially deliver durable benefit but, because a patient’s immune system will identify these cells as foreign, it will require long-term immunosuppression to avoid rejection.
Because of this, PEC-Direct is being developed as a therapy for the subset of patients with type 1 diabetes at high risk for acute complications.
This is where CRISPR comes in—the gene-editing technique could protect the transplanted cells from the immune system by “ex vivo editing immune-modulatory genes within the stem cell line used to produce the pancreatic-lineage cells”.
CRISPR Therapeutics and ViaCyte will collaborate to develop an immune-evasive stem cell line, the first step on the path to an allogeneic stem-cell derived product.
Following successful completion of these studies and identification of a product candidate, CRISPR Therapeutics and ViaCyte will both assume responsibility for further development and commercialisation worldwide.
ViaCyte will receive $15 million from CRISPR Therapeutics, with the potential to receive an additional $10 million.
Samarth Kulkarni, CEO of CRISPR Therapeutics, said that the combination of regenerative medicine and gene-editing has the potential to offer “durable, curative therapies to patients in many different diseases”.
Kulkarni added: “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.”
Paul Laikind, CEO and president of ViaCyte, believes that creating an immune-evasive gene-edited version of ViaCyte’s technology will allow the companies to address a larger patient population than they could with a product requiring immunosuppression.
He said: “We are thrilled to have the opportunity to partner with CRISPR Therapeutics on what we believe could be a transformational therapy for patients with insulin-requiring diabetes.”
CRISPR Therapeutics is also pursuing a similar approach for its allogeneic CAR-T programmes.
In August last year, CRISPR Therapeutics announced that it had united with the cancer centre at Massachusetts General Hospital to develop T cell therapies for treating the disease.
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