ViaCyte & CRISPR Therapeutics team up to battle diabetes

ViaCyte
The PEC-Encap™ (also known as VC-01) product candidate is designed to deliver pancreatic progenitor cells in an immunoprotective device.  The PEC-Direct™ product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device that is designed for direct vascularization of the implanted cells.

What happens when one of the most exciting stem cell biotechs ViaCyte teams up with a firm like CRISPR Therapeutics to use a combination of gene editing and cell therapy? Hopefully a synergistic partnership emerges that in this case can develop an effective stem cell-based therapy for diabetes. You can see a press release on the news. (Update, see my new interview after the news broke with ViaCyte CEO Paul Laikind)

ViaCyte
The PEC-Encap™ (also known as VC-01) product candidate is designed to deliver pancreatic progenitor cells in an immunoprotective device.  The PEC-Direct™ product candidate delivers the pancreatic progenitor cells in a non-immunoprotective device that is designed for direct vascularization of the implanted cells.

ViaCyte has been a frequent subject of posts on The Niche related to their development of pluripotent stem cell-based therapies for diabetes. You can see my interview from just under a year ago with the company’s CEO Paul Laikind here. It goes into the differences between their current two main devices (pictured at right) and the company’s future.

One challenge for ViaCyte is that their product in its current form (see image of PEC-Direct at right) could be rejected by patients’ immune systems so the idea here with the collaboration with CRISPR therapeutics is to gene edit the ViaCyte cells to make them less likely to invoke an immune response. From the PR:

“Under the terms of the agreement, CRISPR and ViaCyte will jointly seek to develop an immune-evasive stem cell line as a first step on the path to an allogeneic stem-cell derived product.  Upon successful completion of these studies and identification of a product candidate, the parties will jointly assume responsibility for further development and commercialization worldwide.  Upon execution of the agreement ViaCyte will receive $15 million from CRISPR, which at CRISPR’s election may be paid in either cash or CRISPR stock.  ViaCyte also has the option, under certain circumstances, to receive an additional $10 million from CRISPR in the form of a convertible promissory note.”

This is a nice boost for ViaCyte to get at least $15 million for this and for CRISPR Therapeutics in the form of exciting new partnership. Hopefully a win-win. From Laikind in the PR:

“Creating an immune-evasive gene-edited version of our technology would enable us to address a larger patient population than we could with a product requiring immunosuppression.  CRISPR Therapeutics is the ideal partner for this program given their leading gene editing technology and expertise and focus on immune-evasive editing.  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,” commented Paul Laikind, Ph.D., Chief Executive Officer and President of ViaCyte.  “We also believe that this approach may have many other applications which we and CRISPR may explore in the future.”

Other groups including Semma Therapeutics formed by Doug Melton of Harvard are working on using stem cells for diabetes as well including efforts at making a beta cell product that could be delivered in a capsule.

The gene editing company’s stock (CRSP) was down a bit on the news so far today, while ViaCyte remains at this point a privately held firm so has no publicly-traded stock.

Today’s news fits with the first of my top 20 predictions for 2018 that I made in late 2017 that expected us to see a lot of action on the combined cell-gene therapy front. In a couple months I’ll make my predictions for 2019.

2 Comments


  1. Just wondering- how many ways are there to make cells immune-evasive?
    Astellas bought Universal Cells, a small company that is getting rid of HLA on hESCs and adding a suicide gene and an HLA-E single chain to evade NK cells. Last week at the Phacillitate conference I heard about a half dozen ways that people are modifying T cells so they can use allogeneic CAR-T therapy. Where does CRISPR Therapeutics sit in the CRISPR patent wars?


    • Hey Jeanne,
      Great questions. Universal Cells also came to my mind. I’m betting that ViaCyte & CRISPR Therapeutics cannot really discuss their strategy in detail at this point. I’d imagine there are going to be many ways, but one could imagine IP issues arising in some cases. Paul

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