Caribou Biosciences, Inc. (Nasdaq:CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, announced today the publication of data demonstrating that its proprietary CRISPR hybrid RNA-DNA (chRDNA) guide technology provides significantly improved specificity compared to all-RNA guides, thereby enabling high levels of intended genomic edits in cells while eliminating or minimizing inadvertent off-target events. Higher specificity is a key advantage in the development of therapeutics and of critical importance in therapies that contain multiple genome edits.
The data, which provide a mechanistic framework to elucidate the specificity of chRDNA guides, were published in an article entitled, “Conformational control of Cas9 by CRISPR hybrid RNA-DNA guides mitigates off-target activity in T cells,” in the journal Molecular Cell.
“Caribou is currently developing unique allogeneic CAR-T cell therapies with multiple genome edits designed to enhance their persistence in patients,” said Steve Kanner, Ph.D., Caribou’s chief scientific officer. “By altering the position and number of DNA residues in our chRDNA guides, we readily achieve optimal on-target editing and minimize unintended off-target edits that may be problematic in therapeutic applications.”
“These data demonstrate that our chRDNA editing platform provides an efficient and highly customizable approach to develop sophisticated allogeneic CAR-T cells for the treatment of hematologic malignancies,” said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. “The first of Caribou’s multiplex-edited product candidates, CB-010, is being evaluated in an ongoing Phase 1 clinical trial in patients with relapsed or refractory B cell non-Hodgkin lymphoma and we look forward to initial clinical data in 2022.”
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