First-in-Human Gene-Edited Stem Cell Trial? Check.

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Gene Editing via Scissors

ViaCyte, a beta cell replacement company long supported by JDRF, and CRISPR Therapeutics have a new first: Gene-editing for type 1 diabetes (T1D). By the end of the year, they will start a clinical trial of VCTX210, a gene-edited stem cell replacement therapy for this disease. Combining ViaCyte’s leading stem cell capabilities, which were developed with significant support from JDRF, with CRISPR Therapeutics’ pre-eminent gene-editing platform, has significant potential in the development of a cell replacement therapy that does not require immune suppression, advancing ViaCyte’s mission of providing a cure for diabetes and JDRF’s vision of a world without T1D.

“JDRF applauds ViaCyte and CRISPR for bringing their VCTX210 therapy into human clinical trials. This is an enormous step forward in alignment with JDRF’s vision to bring insulin independence to people with type 1 diabetes without the need for immunosuppression,” says Sanjoy Dutta, Ph.D., JDRF Vice President of Research. “JDRF is proud of the work we have done with ViaCyte over the past 10+ years that has led to this transformative step and looks forward to forthcoming results.”

The phase I clinical trial to evaluate the safety, efficacy, and immune evasiveness of the therapy will begin in Canada this year.

JDRF Leadership: JDRF has been a long-time and significant supporter of ViaCytesupporting the company through research funding, as well as advocating for government funding for the California Institute of Regenerative Medicine, which also supported ViaCyte. Our funding 15 years ago (when ViaCyte was called CyThera) underwrote development of the proprietary line of precursor stem cells used in their treatment. JDRF also funded the preclinical and clinical studies of ViaCyte’s PEC-01™ therapies, which are designed to mature into islet tissue in humans, including glucose-responsive insulin-secreting beta cells. This includes the first ever clinical trial to test a stem cell-derived cell replacement therapy for T1D, in 2014.

This funding is one of several approaches to cell replacement JDRF has advanced through research funding  as part of the JDRF Cell Replacement Consortium. Visit here to find out more about our program.