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These Companies Want to Combine Stem Cells and CRISPR Gene Editing to Treat Diabetes

September 17, 2018, 9:35 PM UTC

Some news from the land of CRISPR gene-editing to start off this week: The appropriately named CRISPR Therapeutics—a biotech that, it should be noted, was co-founded by Dr. Emmanuelle Charpentier, one of the pioneers of the genome-tweaking tech that’s at the center of major IP litigation that’s pitted scientific titans against one another—is partnering with San Diego-based ViaCyte in an effort to tackle type 1 diabetes. The initial deal is worth up to $25 million, including $15 million that CRISPR will pay ViaCyte upfront.

Type 1 diabetes is a different beast from the type 2 variety that often soaks up the public health media spotlight. The disorder tends to afflict the young, and it’s caused by a defect in the pancreas which leads to minimal or non-existent insulin production (type 2 diabetes, on the other hand, is associated with insulin resistance and an eventual breakdown in its production—both conditions result in excess blood sugar levels).

ViaCyte has set out on an ambitious quest to treat type 1 diabetes by creating certain kinds of pancreatic cells out of stem cells. But there’s a fly in the ointment—these cells are of an “off-the-shelf” variety, meaning that they aren’t necessarily created to precisely match each individual patient’s cells. That can provoke attacks from the immune system that reject these foreign bodies as, well, foreign.

The hope is that CRISPR gene-editing could could essentially transform ViaCyte’s manufactured pancreatic cells to make them non-threatening to patients’ immune systems.

“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,” said CRISPR Therapeutics CEO Samarth Kulkarni in a statement. “ViaCyte is a pioneer in the regenerative medicine field, and has built a compelling clinical program, robust manufacturing capabilities, and assembled a strong intellectual property position. 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.”

Kulkarni’s comments on manufacturing and intellectual property are particularly telling. Since ViaCyte’s technology is of the off-the-shelf variety, producing it on a large scale could potentially be less expensive than, say, personalized immunotherapies that are created on a patient-by-patient basis. And, of course, every life sciences firm involved in the CRISPR space is well-versed in the struggles of a strung-out patent litigation process.

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