By Mark Terry
ViaCyte, headquartered in San Diego, and CRISPR Therapeutics, located in Switzerland and Cambridge, Massachusetts, will utilize CRISPR’s gene editing technology and ViaCyte’s PEC-Direct product to focus on type 1 diabetes. PEC-Direct is currently in clinical trials. It uses a non-immunoprotective delivery device the allows direct vascularization of a cell therapy. However, the patients’ immune systems react to these, which requires long-term immunosuppression. PEC-Direct, then, is being developed to treat a subset of type 1 diabetes patients at high risk for acute complications.
CRISPR gene editing may potentially protect the transplanted cells from the patient’s immune system by “ex vivo editing immune-modulatory genes within the stem cell line used to produce the pancreatic-lineage cells.”
Essentially, ViaCyte has been creating pancreatic cells out of stem cells, hoping to be used to treat type 1 diabetes. However, patients’ bodies treat them as foreign materials, and react to them, requiring immune system suppression. CRISPR hopes to be able to use its gene editing tech to modify these pancreatic cells in such a way that the immune system doesn’t attack them.
CRISPR is attempting to do similar work with CAR-T cells in the oncology space.
“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 Samarth Kulkarni, CRISPR Therapeutics’ chief executive officer, 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.”
Under the terms of the deal, the two companies will jointly work to develop an immune-evasive stem cell line. Once they identify a possible product candidate, they will jointly take responsibility for further development and commercialization. CRISPR Therapeutics will pay ViaCyte $15 million upfront, which CRISPR can pay in cash or CRISPR stock. ViaCyte will also have the option, based on specific circumstances, to receive another $10 million from CRISPR as a convertible promissory note.
“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,” said Paul Laikind, ViaCyte’s chief executive officer and president, in a statement. “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. We also believe that this approach may have many other applications which we and CRISPR may explore in the future.”
In June, ViaCyte released two-year data from Cohort 1 of its PEC-Encap Phase I/II clinical trial in type 1 diabetes. The data suggested the product was safe and well tolerated and when engraftment occurred, viable mature insulin-expressing endocrine islet cells were formed. And in some cases, the insulin-expressing cells lasted for up to two years.