Caribou Biosciences Announces FDA Clearance of IND Application for CB-012, an Allogeneic Anti-CLL-1 CAR-T Cell Therapy for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia
18 10월 2023 - 9:00PM
Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today announced
that it received clearance of its Investigational New Drug (IND)
application from the U.S. Food and Drug Administration (FDA) for
CB-012, an allogeneic anti-C-type lectin-like molecule-1
(anti-CLL-1) CAR-T cell therapy. CB-012 will be evaluated in the
multicenter, open-label, AMpLify Phase 1 clinical trial for
patients with relapsed or refractory acute myeloid leukemia (r/r
AML).
“Clearance of our IND application for CB-012 represents another
significant milestone for Caribou as our third off-the-shelf CAR-T
cell therapy enters the clinic,” said Rachel Haurwitz, PhD,
Caribou’s president and chief executive officer. “We look forward
to initiating patient enrollment in the AMpLify Phase 1 trial by
the middle of 2024 to evaluate the safety and tolerability of
CB-012 in patients suffering from AML.”
CLL-1 is a compelling therapeutic target because it is highly
expressed on AML cells and leukemic stem cells, but it is not
expressed on hematopoietic stem cells.
“There is an urgent need to develop new treatments for patients
with relapsed or refractory AML, for which the treatment options
are predominantly limited to salvage chemotherapy regimens,” said
Naval Daver, MD, associate professor and director, Department of
Leukemia, The University of Texas MD Anderson Cancer Center. “An
allogeneic CAR-T cell therapy that could safely and effectively
target AML blasts while preserving healthy hematopoietic stem cells
could provide a much-needed off-the-shelf option for these
patients.”
Caribou’s patented next-generation CRISPR Cas12a chRDNA
genome-editing technology platform maintains high genomic integrity
and significantly improves the specificity of genome editing.
“CB-012 was engineered with five genome edits, and is the first
allogeneic CAR-T cell therapy, to our knowledge, with both
checkpoint disruption through a PD-1 knockout, and immune cloaking
through a B2M knockout and B2M–HLA-E fusion transgene insertion,”
said Steve Kanner, PhD, Caribou’s chief scientific officer. “Both
armoring strategies are designed to improve the antitumor activity
of CB-012 that we believe are crucial for targeting this difficult
to treat indication.”
CB-012, a genome-edited allogeneic anti-CLL-1 CAR-T cell
therapy with both checkpoint disruption and immune
cloaking
A photo accompanying this announcement is available
athttps://www.globenewswire.com/NewsRoom/AttachmentNg/54559479-fba4-4f88-a241-583814242f44
About the AMpLify trialThe AMpLify Phase 1
trial is an open-label, multicenter clinical trial designed to
evaluate CB-012 in adult patients with relapsed or refractory acute
myeloid leukemia (r/r AML). Part A, a 3+3 dose escalation design,
will evaluate the safety and tolerability of CB-012 at ascending
dose levels to determine the maximum tolerated dose and/or the
recommended doses for expansion. Part B is the dose expansion
portion with the primary objective of determining antitumor
response, assessed by overall response rate (ORR), after a single
dose of CB-012. AMpLify will include patients who have not
responded to or relapsed after standard treatment and will exclude
patients who have been treated with more than 3 prior lines of
therapy and patients with proliferative disease. Caribou plans to
initiate patient enrollment in the AMpLify trial to treat patients
with a single administration of CB-012 at dose level 1 (25x106
CAR-T cells) by mid-2024.
About acute myeloid leukemiaAcute myeloid
leukemia (AML) is a cancer of the blood and bone marrow and is the
most common type of acute leukemia in adults. It is estimated there
will be 20,380 new cases of AML in the United States in 2023. The
five-year survival rate for these patients is approximately
30%. AML is currently treated with chemotherapy, targeted
therapies, and/or allogeneic or autologous stem cell transplant.
For patients with relapsed or refractory AML, there are few
treatment options and median overall survival is typically less
than seven months.
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 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.
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 its expectations relating to the timing of
initiating patient enrollment in the AMpLify Phase 1 clinical trial
for CB-012. 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; 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; and the risk that initial,
preliminary, or interim clinical trial data will not ultimately be
predictive of the safety and efficacy of Caribou’s product
candidates or that clinical outcomes may differ as patient
enrollment continues and as more patient data becomes available;
the risk that preclinical study results observed will not be borne
out in human patients or different conclusions or considerations
are reached once additional data have been received and fully
evaluated; 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
Caribou Biosciences (NASDAQ:CRBU)
과거 데이터 주식 차트
부터 4월(4) 2024 으로 5월(5) 2024
Caribou Biosciences (NASDAQ:CRBU)
과거 데이터 주식 차트
부터 5월(5) 2023 으로 5월(5) 2024