Vertex cuts ties to CRISPR Therapeutics’ type 1 diabetes stem cell therapy

Vertex is severing one of its ties to CRISPR Therapeutics. The big biotech has chosen to opt out of the diabetes gene-edited stem cell therapy it gained through the acquisition of ViaCyte, leaving CRISPR to take the clinical-phase program forward itself.

CRISPR teamed up with ViaCyte in 2021. The alliance positioned CRISPR to combine its gene-editing skills with ViaCyte’s ability to generate pancreatic-lineage cells from stem cells. Through editing, CRISPR aimed to create beta cells that evade the immune system and thereby enable people with diabetes to receive insulin-producing cells without long-term immunosuppression to avoid rejection.

An initial drug candidate, VCTX210, entered the clinic in 2022, followed by a next-generation prospect the next year. CRISPR, which will wholly own the assets once the opt-out is complete, plans to keep running a phase 1 clinical trial of the next-generation candidate, now called CTX211, without the support of Vertex and ViaCyte. The completion date of the clinical trial is in 2025.

Vertex also remains active in diabetes cell therapy, including in partnership with CRISPR. In March, Vertex paid $100 million for non-exclusive rights to its partner’s CRISPR-Cas9 technology in hypoimmune cell therapies for type 1 diabetes. CRISPR received a further $70 million from Vertex later in the year and is still in line to receive up to $160 million in R&D milestones. 

The CRISPR deal is one of several pacts Vertex struck to establish diabetes as a potential growth driver. Vertex has two diabetes cell therapies in clinical development, including one asset, VX-264, that is being evaluated without the use of immunosuppressive therapy. With its own irons in the fire, the biotech has decided to do without its CRISPR-partnered program. 

CRISPR disclosed the loss of the Vertex-ViaCyte deal as part of an update on its plans for 2024. Other bits of news in the update included details of CRISPR’s plans to study its CD19-directed CAR-T candidate in lupus.