CRISPR Therapeutics and ViaCyte’s Strategic Collaboration to Develop Gene‐Edited Stem Cell‐Derived Therapy for Diabetes

Note: ViaCyte, Inc. is a grantee of Beyond Type 1. 

ZUG, Switzerland and CAMBRIDGE, Mass., and SAN DIEGO ‐ Yesterday, CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company that develops transformative gene‐based medicines for serious diseases, and ViaCyte, Inc., a regenerative medicine company, announced their collaboration. The two companies will focus on the discovery, development and commercialization of gene-edited allogeneic stem cell therapies for the treatment of diabetes.

According to National Institute of Health, “Allogeneic stem cell therapy offers advantages over the autologous counterpart. The stem cells are derived from young healthy donors, eliminating any co-morbidities associated with disease states. Allogeneic cells grown and kept in stem cell banks so that they are available for immediate delivery.”

ViaCyte has been at the forefront of generating pancreatic‐lineage cells from stem cells and delivering them safely and efficiently to patients. Their clinical data indicates that beta‐cell replacement approaches could have “curative benefits” to those who are insulin-dependent.

ViaCyte is currently using PEC‐Direct’s product in the clinic, which is a non‐immunoprotective delivery device that permits direct vascularization of the cell therapy.

This device is durable; although, because the patient’s immune system will identify the cells as foreign, PEC‐Direct will require long‐term immunosuppression drugs to avoid the body from rejecting the cell delivery device. Due to this, the PEC‐Direct will be used for a group of type 1 diabetes patients at high risk for acute complications.

With this new partnership with CRISPR, the company can offer gene editing technology that could potentially protect the transplanted cells from an attack by the patient’s immune system. This is done with the use of  ex‐vivo editing immune‐modulatory genes in the pancreatic stem-cells.

Samarth Kulkarni, Ph.D., chief executive officer of CRISPR Therapeutics, said, “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. 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.”

After the two companies have completed these studies they will jointly assume responsibility for further development and commercialization worldwide of the stem-cell replacement device that is both durable and not vulnerable to an immune attack.

“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.”

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene‐ based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG and Vertex Pharmaceuticals. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly‐ owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts and business offices in London, United Kingdom. For more information, please visit www.crisprtx.com.

About ViaCyte

ViaCyte is a privately‐held regenerative medicine company developing novel cell replacement therapies as potential long‐term diabetes treatments to achieve blood glucose control targets and reduce the risk of hypoglycemia and diabetes‐related complications. ViaCyte’s product candidates are based on the derivation of pancreatic progenitor cells from stem cells, which are then implanted in durable and retrievable cell delivery devices. Once implanted and matured, these cells are designed to secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two product candidates in clinical‐stage development. The PEC‐DirectTM product candidate delivers the pancreatic progenitor cells in a non‐immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability, and/or recurrent severe hypoglycemic episodes. The PEC‐EncapTM (also known as VC‐01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is also seeking to develop immune‐evasive ‘universal donor’ stem cell lines, from its proprietary CyT49 cell line, which are expected to further broaden the availability of cell therapy for diabetes and other potential indications. ViaCyte is headquartered in San Diego, California. ViaCyte is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF. For more information, please visit www.viacyte.com.