Caribou Biosciences Presents New Preclinical Data on CB-012, an Allogeneic Anti-CLL-1 CAR-T Cell Therapy, at the 2023 AACR Annual Meeting
April 17 2023 - 1:30PM
Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today presents a
poster of preclinical data demonstrating the promise of CB-012, a
next-generation CRISPR-edited allogeneic anti-CLL-1 CAR-T cell
therapy, as a therapeutic candidate for adult patients with
relapsed or refractory acute myeloid leukemia (r/r AML). The
presentation takes place at the 2023 American Association for
Cancer Research (AACR) Annual Meeting today from 1:30 pm to 5:00 pm
EDT at the Orange County Convention Center, Orlando,
Florida.
“CLL-1 is a compelling target for AML because it is highly
expressed on myeloid cancer cells and is enriched on leukemic stem
cells, but it is not expressed on hematopoietic stem cells,” said
Steve Kanner, PhD, Caribou’s chief scientific officer. “CB-012 is
engineered with 5 edits, using our Cas12a chRDNA technology, which
armor the CAR-T cells for improved antitumor activity through
checkpoint disruption and immune cloaking. The preclinical data in
the poster demonstrate that CB-012 exhibits enhanced antitumor
activity against established AML xenografts.”
Caribou’s patented next-generation CRISPR Cas12a chRDNA
genome-editing technology platform, which maintains high genomic
integrity and significantly improves the specificity of genome
edits, was used to engineer the 5 genome edits implemented in the
manufacture of CB-012. CB-012 is the first allogeneic CAR-T cell
therapy, to Caribou’s knowledge, with both checkpoint disruption,
through a PD-1 knockout (KO), and immune cloaking, through a B2M KO
and B2M–HLA-E fusion transgene insertion. These armoring strategies
were designed to promote the durability of antitumor activity.
Preclinical data presented at the AACR meeting show:
- CB-012 targets, becomes activated, proliferates, and
demonstrates antitumor activity against a broad panel of AML cancer
cell lines
- Immune cloaking protects CB-012 from NK cell-mediated
cytotoxicity
- Mice harbouring AML xenograft models treated with CB-012 having
a PD-1 KO showed extended survival relative to mice injected with
control CAR-T cells that express PD-1 and that only contain 4 out
of the 5 edits
- CB-012 demonstrated significant antitumor efficacy and
prolonged survival in AML xenograft models
“The preclinical data presented at AACR further support the
clinical development of CB-012, Caribou’s third program from our
allogeneic CAR-T cell therapy platform,” said Rachel Haurwitz, PhD,
Caribou’s president and chief executive officer. “Preclinical
studies supporting our planned IND submission for CB-012 in
relapsed or refractory AML are advancing and we are on track to
submit our IND to the FDA in the second half of this year.”
Details of the poster presentation are below and the full poster
can be found on Caribou’s website under Scientific
Publications.
Title: CB-012, an allogeneic anti-CLL-1 CAR-T
cell therapy engineered with next-generation CRISPR technology to
resist both the immunosuppressive tumor microenvironment and immune
cell-mediated rejection, for patients with relapsed or refractory
acute myeloid leukemiaPresenter: Tristan Fowler,
PhD, associate director of preclinical pharmacology, Caribou
BiosciencesSession: PO.CL07.04 - Adoptive Cell
Therapy 2Session Date: Monday, April 17,
2023Presentation Time: 1:30 - 5:00 pm
EDTLocation: Orange County Convention Center,
section 37Abstract number: 3201
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
have exhibited editing at 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 Cas12a chRDNA technology,
enables superior precision to develop cell therapies that are
armored to potentially improve antitumor activity. Caribou is
advancing a pipeline of off-the-shelf cell therapies from its CAR-T
and CAR-NK platforms as readily available treatments for 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 Statements This 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, including expectations relating to the submission of its
IND application for CB-012 as well as additional updates and
results from its IND-enabling preclinical studies. 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 the development of cell
therapy products, including CB-012; 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, including those for CB-012; and the
risk that future initial or interim clinical trial data will not
ultimately be predictive of the safety and efficacy of Caribou’s
product candidates, including CB-012 or that clinical outcomes may
differ as more patient data becomes available; the risk that
preclinical study results, including those for CB-012 will not be
borne out in human patients; 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, 2022 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:Investors:Amy Figueroa,
CFAinvestor.relations@cariboubio.com
Media:Peggy Vorwald,
PhDmedia@cariboubio.com
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