CRISPR Therapeutics and ViaCyte Present Positive In Vitro Data Towards a Potential Immune-Evasive Cell Replacement Therapy for Diabetes at EASD 2019

-New data demonstrate successful differentiation of CRISPR-edited human pluripotent stem cells to pancreatic precursor cells-

ZUG, Switzerland and CAMBRIDGE, Mass., and SAN DIEGO, September 17, 2019 – CRISPR Therapeutics (Nasdaq: CRSP), and ViaCyte, Inc., a privately-held cell therapy company, today presented data from the Companies’ regenerative medicine program targeted towards type 1 diabetes (T1D) in an oral presentation at the 55th Annual Meeting of the European Association for the Study of Diabetes (EASD) in Barcelona, Spain. The data demonstrate that the CyT49 pluripotent stem cell line, which has been shown to be amenable to efficient scaling and differentiation, can be successfully edited with CRISPR. The CyT49 pluripotent stem cell line is currently being used to generate islet progenitors for clinical trials.

“These data provide further evidence that 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, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “We look forward to advancing our T1D program in partnership with ViaCyte.”

“We are pleased with the data presented at EASD, which bring us potentially one step closer to a transformational therapy for patients with insulin-requiring diabetes through the development of an immune-evasive gene-edited version of our technology,” said Paul Laikind, Ph.D., Chief Executive Officer and President of ViaCyte. “ViaCyte has led the field over the past decade, being the first group to demonstrate a number of essential milestones on the path to a broadly applicable cell replacement therapy for diabetes. Now, in partnership with CRISPR Therapeutics, we aim to achieve yet another first, the development of an immune-evasive cell replacement therapy as a potential cure for T1D. The work being presented at EASD is an important step along that path.”

To protect pancreatic islet cells from immune rejection, researchers utilized CRISPR/Cas9 gene editing to generate CyT49 clones that lack the β2-microglobulin (B2M) gene, a required component of the major histocompatibility complex class I (MHC-I), and express a transgene encoding programmed death-ligand 1 (PD-L1) to further protect from T-cell attack. Edited clonal cells maintained karyotypic stability and showed in vitro protection against T-cell mediated cell lysis.

About the CRISPR-ViaCyte Collaboration

CRISPR Therapeutics and ViaCyte entered into a strategic collaboration in 2018 focused on the discovery, development, and commercialization of novel regenerative medicines including gene-edited allogeneic stem cell-derived therapies for the treatment of diabetes. The Companies are currently evaluating a preclinical-stage therapeutic candidate for insulin-requiring diabetes including type 1 diabetes, for which the Companies will jointly assume responsibility for development and commercialization worldwide.

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, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. 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 glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte’s product candidates are based on the derivation of pancreatic islet progenitor cells from pluripotent stem cells, which are then implanted in durable and retrievable cell delivery devices. Over a decade ago, ViaCyte scientists were the first to report on the production of pancreatic cells from a stem cell starting point and the first to demonstrate in an animal model of diabetes that, once implanted and matured, these cells secrete insulin and other pancreatic hormones in response to blood glucose levels. ViaCyte has two product candidates in clinical-stage development. The PEC-Direct™ product candidate delivers the pancreatic islet 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-Encap™ (also known as VC-01) product candidate delivers the same pancreatic islet 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 developing immune-evasive stem cell lines, from its proprietary CyT49 cell line, which have the potential 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.