Gene-edited cell replacement therapy for diabetes set to enter the clinic

By Rachel Arthur contact

- Last updated on GMT

Pic:getty/XH4D
Pic:getty/XH4D

Related tags: Stem cell, Stem cells, gene-editing, CRISPR Therapeutics, Diabetes, Type 1 diabetes

CRISPR Therapeutics and ViaCyte, Inc. have received the green light from Health Canada for their Clinical Trial Application for VCTX210: an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D).

The Phase 1 trial is expected to start enrolling patients by the end of the year, assessing the safety, tolerability and immune evasion in T1D patients.

VCTX210 is the first gene-edited cell replacement therapy for the treatment of type 1 diabetes to enter the clinic: with the two companies saying it could break new ground by broadening the population that can be treated with cell therapies.

Eliminating the need for immunosuppression

VCTX210 has been developed by applying CRISPR Therapeutics’ gene-editing technology to ViaCyte’s proprietary stem cell capabilities and has the potential to enable a beta-cell replacement product that may deliver durable benefit to patients without requiring concurrent immune suppression.

Removing the need for concurrent immune suppression

ViaCyte has pioneered the approach of generating pancreatic-lineage cells from stem cells and delivering them safely and efficiently to patients.  PEC-Direct, ViaCyte’s lead product candidate currently being evaluated in the clinic, uses a non-immunoprotective delivery device that permits direct vascularization of the cell therapy.  However, the immune system identifies these cells as foreign, meaning PEC-Direct requires long-term immunosuppression to avoid rejection (and thus is suitable only for high-risk type 1 diabetes patients).

CRISPR gene editing offers the potential to 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 (CRISPR Therapeutics is pursuing a similar approach for its allogeneic CAR-T programs and has established significant expertise in immune-evasive gene editing).

“With the approval of our CTA, we are excited to bring a first-in-class CRISPR-edited cell therapy for the treatment of type 1 diabetes to the clinic, an important milestone in enabling a whole new class of gene-edited stem cell-derived medicines,”​ said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics.

“Being first into the clinic with a gene-edited, immune-evasive cell therapy to treat patients with type 1 diabetes is breaking new ground as it sets a path to potentially broadening the treatable population by eliminating the need for immunosuppression with implanted cell therapies,”​ added Michael Yang, President and Chief Executive Officer of ViaCyte. “This approach builds on previous accomplishments by both companies and represents a major step forward for the field as we strive to provide a functional cure for this devastating disease.”

This program is part of a strategic collaboration between the Zug, Switzerland headquartered CRISPR Therapeutics and San Diego-based ViaCyte, announced in 2018. The collaboration is focused on the discovery, development, and commercialization of gene-edited stem cell therapies for the treatment of diabetes.

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