CRISPR Gene Editing Critical to Delivering Off-the-Shelf Cell Therapy

By Vivienne Raper, PhD – Genengnews.com

The future of cell therapies should be in off-the-shelf products using multiplex gene editing during the manufacturing process. That’s according to Rachel Haurwitz, PhD, CEO of Caribou Biosciences.

Haurwitz, whose colleague Justin Skoble, PhD, spoke at at the American Biomanufacturing Summit about innovation in gene and cell therapy platforms, believes that using CRISPR gene editing can produce off-the-shelf cell therapies that meaningfully rival the performance of those tailored to individual patients.

Schematic depicting CB-010 interaction with B cell non-Hodgkin lymphoma cells. Using Caribou’s chRNDA technology, CB-010 has three genome edits: a deletion of the TRAC gene to remove T cell receptors; insertion of a CD19-specific CAR into TRAC locus to target CD19 positive tumor cells; and a knockout of PD-1 to boost the persistence of CAR-T cell antitumor activity and potentially reduce CB-010 exhaustion. [Caribou Biosciences]

 

“The future of the cell therapy field should be off-the-shelf, which means [therapies] can be available to a larger patient community,” Haurwitz explains. Off-the-shelf therapies are also seen as simpler to manufacture.

Caribou Biosciences has developed a gene-editing system that uses CRISPR hybrid RNA-DNA (chRDNA) guides to improve on traditional CRISPR Cas9 gene editing. According to Haurwitz, the chRDNA system allows Caribou to conduct multiple gene edits to, for example, remove PD-1 from the surface of T-cells used in CAR-T cell therapies.

The aim is to remove the “brakes” from the cell therapy product to maintain its high tumor activity and improve the clinical benefits, she says.

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