Caribou Biosciences Presents Positive Preclinical Data for Allogeneic Anti-BCMA CAR-T Cell Therapy Candidate CB-011 at the American Association for Cancer Research (AACR) Annual Meeting
April 08 2022 - 1:07PM
Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today announced
the presentation of positive preclinical data for its allogeneic,
immune-cloaked, anti-BCMA CAR-T cell therapy candidate, CB-011,
being developed for the treatment of relapsed or refractory
multiple myeloma (r/r MM). The data are being presented at the
American Association for Cancer Research (AACR) Annual Meeting,
April 8-13, 2022, in New Orleans.
The data demonstrate that CB-011 CAR-T cells are cytotoxic
against BCMA-expressing tumor cells and are resistant to killing by
both allogeneic T cells and natural killer (NK) cells. In a tumor
xenograft model of MM, CAR-T cells expressing a CAR containing a
proprietary humanized anti-BCMA antibody fragment (scFv) that is
used in CB-011 markedly prolonged antitumor activity compared to
CAR-T cells expressing a benchmark anti-BCMA CAR. Caribou is
conducting Investigational New Drug (IND) application-enabling
safety studies to support a planned IND application submission in
2022 for CB-011 in r/r MM.
“The data that will be presented at AACR demonstrate that T
cells expressing a CAR deploying Caribou’s proprietary anti-BCMA
scFv outperform cells expressing a known benchmark CAR,” said Steve
Kanner, Ph.D., Caribou’s chief scientific officer. “The
immune-cloaking armoring data confirm the potential value and
mechanism of our strategy using multiple genome edits involving HLA
class I disruption as an approach to increase the potential
therapeutic effect of CB-011. This strategy is enabled by our
proprietary Cas12a chRDNA genome-editing technology that
reproducibly achieves high specificity genome editing at multiple
loci.”
Manufacturing CB-011 requires four genome editing steps.
Caribou’s Cas12a chRDNA technology is used to insert a gene
encoding a proprietary, humanized anti-BCMA CAR into the TRAC gene
of the T cell, which also knocks out expression of the T cell
receptor, thereby reducing the risk of graft versus host disease
(GvHD). The immune cloaking strategy entails site-specific
insertion of a gene encoding a B2M-HLA-E-peptide fusion into the
B2M gene of the T cell. This edit knocks out B2M expression and
eliminates endogenous HLA class I presentation on the surface of
the CAR-T cells while enabling expression of the fusion transgene
of HLA-E, a minor HLA class I antigen, designed to blunt both T-
and NK-cell-mediated rejection of the CAR-T cell therapy by the
patient’s immune system.
The data demonstrate that, when co-cultured with BCMA-expressing
cell lines, CB-011 CAR-T cells proliferate in response to antigen,
secrete cytokines consistent with robust T cell activation, and
kill BCMA-positive tumor cells. When CB-011 cells are co-cultured
with either mismatched CD8+ T cells or NK cells, they are protected
from killing, potentially extending their persistence when infused
into a patient. In a tumor xenograft model of MM, CB-011 treatment
prolonged survival compared to a control. Data generated in this
model demonstrated that the anti-BCMA CAR used in CB-011 resulted
in superior survival (40% at 80 days) compared to a known benchmark
anti-BCMA CAR (0% at 30 days).
“We believe that an off-the shelf approach has the potential to
provide an important therapeutic option for people with multiple
myeloma, many of whom are not well served by current therapies,”
said Rachel Haurwitz, Ph.D., Caribou’s president and chief
executive officer. “We look forward to submitting an IND
application in 2022 for CB-011, our second cell therapy candidate.
It is an important element of our allogeneic CAR-T cell therapy
platform targeting a range of hematologic malignancies.”
Details of the poster presentation are as follows:
Title: A BCMA-specific allogeneic CAR-T cell
therapy (CB-011) genome engineered to express an HLA-E fusion
transgene to prevent immune cell
rejectionPresenter: Émilie Degagné,
Ph.D.Date and Time: Sunday, April 10, 2022, 1:30 –
5:00 pm CDTLocation: New Orleans Convention
Center
Presentations and posters will be available for registered
attendees for on-demand viewing on the AACR website on April 8,
2022 after 1:00 pm EDT. The poster is available on the
Presentations page of the Investors section of Caribou’s
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 Type II 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
occasionally edit 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
chRDNAs (pronounced “chardonnays”), RNA-DNA hybrid guides 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 genome 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, including but not limited to, its timing expectations
relating to the submission of an IND application for CB-011.
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 the risks inherent in drug development
such as those associated with being in preclinical development, and
with the initiation, cost, timing, progress, and results of 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. 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, CFA |
Investor Relations and Corporate
Communications |
afigueroa@cariboubio.com |
|
Investors: |
Elizabeth Wolffe, Ph.D., and
Sylvia Wheeler |
Wheelhouse LSA |
lwolffe@wheelhouselsa.com |
swheeler@wheelhouselsa.com |
|
Media: |
Greg Kelley |
Ogilvy |
gregory.kelley@ogilvy.com |
617-461-4023 |
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