Caribou Biosciences Presents Data on Mechanism Underlying Superior Specificity of Caribou’s chRDNA Genome-Editing Technology in Primary Human T cells
May 16 2022 - 4:05PM
Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today announced
the presentation of studies in primary human T cells highlighting
the mechanism underlying the superior specificity of the company’s
CRISPR hybrid RNA-DNA (chRDNA) guides for genome editing.
“Our chRDNA guides provide a robust and reproducible approach
for maximizing the specificity of CRISPR genome editing, which is
key to improving the persistence and potential efficacy of our
allogeneic cell therapies,” said Steve Kanner, Ph.D., Caribou’s
chief scientific officer. “Through simple iterative engineering of
DNA positions in our chRDNA guides, we optimize the activity and
specificity of editing at different sites in the genome.”
In studies with primary human T cells, optimized chRDNA guides
were rapidly generated to provide higher specificity editing
compared to either all-RNA guides or when employing an engineered
high fidelity Cas9 variant. These high specificity chRDNA guides
can be used with either the Cas9 protein or the Cas12a protein. The
presentation highlights the underlying mechanism of this enhanced
specificity by comparing the structure of Cas9 nuclease with all
RNA guides compared with chRDNA guides. The data show differences
between the structural conformation of the complex when targeting
the intended specific site in the chromosome versus unintended
“off-target” sites, providing an explanation for the improved
specificity. Together, these data demonstrate that chRDNA guides
enable highly efficient and precise genome editing, emphasizing
their potential superiority for use in therapeutic applications.
Together, these data demonstrate that chRDNA guides enable highly
efficient and precise genome editing, emphasizing their potential
superiority for use in therapeutic applications.
The data are being presented virtually today at the 25th Annual
Meeting of the American Society for Gene and Cell Therapy (ASGCT)
which is being held May 16-19, 2022 in Washington, D.C. The
presentation, titled “Conformational Control of Cas Endonucleases
by CRISPR Hybrid RNA-DNA Guides Mitigates Off-Target Activity in T
Cells” is available on the Presentations page of the Investors
section of the Caribou website.
About Caribou’s Novel Next-Generation CRISPR
PlatformCRISPR genome editing uses easily designed,
modular biological tools to make DNA changes in living cells. There
are two basic components of Class 2 CRISPR systems: the nuclease
protein that cuts DNA and the RNA molecule(s) that guide the
nuclease to generate a site-specific, double-stranded break,
leading to an edit at the targeted genomic site. CRISPR systems are
capable of editing unintended genomic sites, known as off-target
editing, which may lead to harmful effects on cellular function and
phenotype. In response to this challenge, Caribou has developed
CRISPR hybrid RNA-DNA guides (chRDNAs; pronounced “chardonnays”)
that direct substantially more precise genome editing compared to
all-RNA guides. Caribou is deploying the power of its Cas12a chRDNA
technology to carry out high efficiency multiple edits, including
multiplex gene insertions, to develop CRISPR-edited therapies.
About Caribou Biosciences,
Inc.Caribou Biosciences is a clinical-stage CRISPR
genome-editing biopharmaceutical company dedicated to developing
transformative therapies for patients with devastating diseases.
The company’s genome-editing platform, including its proprietary
Cas12a chRDNA technology, enables superior precision to develop
cell therapies that are specifically engineered for enhanced
persistence. Caribou is advancing a pipeline of off-the-shelf CAR-T
and CAR-NK cell therapies for the treatment of patients with
hematologic malignancies and solid tumors.
Follow us @CaribouBio and visit www.cariboubio.com.
“Caribou Biosciences” and the Caribou logo are registered
trademarks of Caribou Biosciences, Inc.
Forward-Looking StatementsThis
press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
These forward-looking statements include, without limitation,
statements related to Caribou’s strategy, plans, and objectives,
and expectations regarding its clinical and preclinical development
programs. Management believes that these forward-looking statements
are reasonable as and when made. However, such forward-looking
statements are subject to risks and uncertainties, and actual
results may differ materially from any future results expressed or
implied by the forward-looking statements. Risks and uncertainties
include, without limitation, risks inherent in development of
CRISPR technologies and cell therapy products; and uncertainties
related to the initiation, cost, timing, progress, and results of
Caribou’s current and future research and development programs,
preclinical studies, and clinical trials; as well as other risk
factors described from time to time in Caribou’s filings with the
Securities and Exchange Commission, including its Annual Report on
Form 10-K for the year ended December 31, 2021, and subsequent
filings. In light of the significant uncertainties in these
forward-looking statements, you should not rely upon
forward-looking statements as predictions of future events. Except
as required by law, Caribou undertakes no obligation to update
publicly any forward-looking statements for any reason.
Caribou Biosciences, Inc.
Contacts:Amy Figueroa,
CFAInvestor Relations and
CorporateCommunicationsafigueroa@cariboubio.com
Investors and Media:Elizabeth
Wolffe, Ph.D., and Sylvia WheelerWheelhouse
LSAlwolffe@wheelhouselsa.comswheeler@wheelhouselsa.com
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