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19 September 2018Americas

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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More on this story

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20 September 2018   ERS Genomics and the Broad Institute of MIT and Harvard signed CRISPR licensing deals with Thermo Fisher Scientific on Tuesday, shortly before ERS expanded a separate agreement with Oxford Genetics.
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14 December 2017   US-based Vertex Pharmaceuticals and CRISPR Therapeutics have revealed plans to jointly develop and commercialise an investigational gene-editing treatment.
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13 November 2018   Gene-editing company CRISPR Therapeutics has expanded its partnership with US-based MaxCyte, which focuses on cell-based medicines, into the immuno-oncology arena.

More on this story

Americas
20 September 2018   ERS Genomics and the Broad Institute of MIT and Harvard signed CRISPR licensing deals with Thermo Fisher Scientific on Tuesday, shortly before ERS expanded a separate agreement with Oxford Genetics.
Americas
14 December 2017   US-based Vertex Pharmaceuticals and CRISPR Therapeutics have revealed plans to jointly develop and commercialise an investigational gene-editing treatment.
Americas
13 November 2018   Gene-editing company CRISPR Therapeutics has expanded its partnership with US-based MaxCyte, which focuses on cell-based medicines, into the immuno-oncology arena.