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There’s some good news for a company and a therapeutic approach that CIRM has been supporting for many years.

In September 2018, CRISPR Theraputics and ViaCyte entered a partnership to discover, develop and market gene-edited stem cell-derived therapies to treat type 1 diabetes (T1D). Today, they may stand one step closer to their goal. 

Last week the companies jointly announced that they have dosed the first subject in the Phase 1 clinical trial of VCTX210 for the treatment of T1D. VCTX210 is an investigational stem cell-based therapy. It was developed combining CRISPR’s gene-editing technology with ViaCyte’s stem cell expertise to generate pancreatic beta cells that can evade the immune system.

ViaCyte, a regenerative medicine company long backed by CIRM, has developed an implantable device which contains pancreatic endoderm cells that mature over a few months and turn into insulin-producing pancreatic islet cells, the kind destroyed by T1D. 

Using CRISPR technology, the genetic code of the implanted cells is modified to create beta cells that avoid all recognition by the immune system. This collaboration aims to eliminate the requirement of patients taking daily immunosuppressants to stop the immune system from attacking the implanted cells. 

The first phase of the VCTX210 clinical trial will assess the safety, tolerability, and immune evasion in patients with T1D. 

“We are excited to work with CRISPR Therapeutics and ViaCyte to carry out this historic, first-in-human transplant of gene-edited, stem cell-derived pancreatic cells for the treatment of diabetes designed to eliminate the need for immune suppression,” said James Shapiro, a clinical investigator in the trial. “If this approach is successful, it will be a transformative treatment for patients with all insulin-requiring forms of diabetes.”

CIRM has been a big investor in ViaCyte’s work for many years and has invested more than $72 million in nine different awards.