Beam Therapeutics Reports Preclinical Data Highlighting Multiplex Base Editing Approach to Create Anti-CD5 CAR T-cell Investigational Therapies for T-Cell Malignancies
12 11월 2021 - 9:00PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced new preclinical research demonstrating the ability of the
company’s multiplex edited CAR T cells to target CD5-positive
tumors, leading to tumor clearance in vivo. The data are
highlighted in a poster titled, “CD5 knockout enhances the potency
of multiplex base-edited allogeneic anti-CD5 CAR T-cell therapy for
the treatment of T-cell malignancies,” at the Society for
Immunotherapy of Cancer’s (SITC) 36th Annual Meeting, being held
Nov. 10-14, 2021.
CAR T therapies have the potential to address a number of T-cell
malignancies, but their therapeutic potential is often hindered by
technological limitations in their development and application.
Beam has developed a process using base editing to simultaneously
silence five target genes, including CD5 and PD1, to create
allogeneic anti-CD5 CAR T-cells with enhanced effector function for
potential use as off-the-shelf treatments for T-cell
malignancies.
“There is a significant unmet need in the treatment of
T-cell lymphomas, as some indications have particularly poor
prognoses and patients with relapsed or refractory
disease have few treatment options that can produce deep
and durable responses,” said Giuseppe Ciaramella, Ph.D.,
president and chief scientific officer of Beam. “We believe our
approach using multiplex editing of T-cells to produce
allogeneic anti-CD5 CAR T investigational therapies may offer
enhanced potency and more durable responses for patients with
T-cell malignancies. Importantly, our base editing approach is
designed to avoid making double-stranded breaks in DNA that are
produced by multiplex editing with nucleases, thereby decreasing
the potential for translocations and other genomic aberrations with
potential for unforeseen consequences, increasing their potential
safety and tolerability.”
Key findings reported in the poster include:
- Multiplexed base editing resulted in anti-CD5 CAR T cells with
94-98% editing efficiency at all 5 target loci and transduction
efficiency of over 85%, which Beam believes is an efficiency at
which the likelihood of graft versus host disease, CAR T rejection,
and immunosuppression would be greatly reduced;
- CD5 knockout improved production of a combination of
proinflammatory cytokines, including a greater than 300% increase
in IL-2 production;
- CD5 knockout greatly improved in vivo efficacy of anti-CD5 CAR
T cells in a xenograft mouse model of T-ALL compared to unedited
cells. CD5 edited anti-CD5 CAR T cells were able to clear
established tumor in a dose dependent manner; however, unedited
anti-CD5 CAR T cells failed to clear initial tumor at all doses
despite demonstrating in vitro efficacy.
- Mice previously cleared of tumor underwent additional tumor
challenges to assess the persistence and functionality of anti-CD5
CAR T cells and were cleared of tumor both a second and third time,
indicating extended persistence of functional anti-CD5 CAR T cells
in vivo.
About Beam TherapeuticsBeam Therapeutics
(Nasdaq: BEAM) is a biotechnology company committed to establishing
the leading, fully integrated platform for precision genetic
medicines. To achieve this vision, Beam has assembled a platform
that includes a suite of gene editing and delivery technologies and
is in the process of building internal manufacturing capabilities.
Beam’s suite of gene editing technologies is anchored by base
editing, a proprietary technology that enables precise, predictable
and efficient single base changes, at targeted genomic sequences,
without making double-stranded breaks in the DNA. This enables a
wide range of potential therapeutic editing strategies that Beam is
using to advance a diversified portfolio of base editing programs.
Beam is a values-driven organization committed to its people,
cutting-edge science, and a vision of providing life-long cures to
patients suffering from serious diseases.
Cautionary Note Regarding Forward-Looking
StatementsThis press release contains forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995. Investors are cautioned not to place undue
reliance on these forward-looking statements, including, but not
limited to, statements related to: our planned base editing data
presentations at an upcoming scientific conference; the therapeutic
applications and potential of our technology, including with
respect to T-cell malignancies; and our ability to develop
life-long, curative, precision genetic medicines for patients
through base editing. Each forward-looking statement is subject to
important risks and uncertainties that could cause actual results
to differ materially from those expressed or implied in such
statement, including, without limitation, risks and uncertainties
related to: our ability to develop, obtain regulatory approval for,
and commercialize our product candidates, which may take longer or
cost more than planned; our ability to raise additional funding,
which may not be available; our ability to obtain, maintain and
enforce patent and other intellectual property protection for our
product candidates; the potential impact of the COVID-19 pandemic;
that preclinical testing of our product candidates and preliminary
or interim data from preclinical studies and clinical trials may
not be predictive of the results or success of ongoing or later
clinical trials; that enrollment of our clinical trials may take
longer than expected; that our product candidates may experience
manufacturing or supply interruptions or failures; risks related to
competitive products; and the other risks and uncertainties
identified under the headings “Risk Factors Summary” and “Risk
Factors” in our Annual Report on Form 10-K for the year ended
December 31, 2020, our Quarterly Report on Form 10-Q for the
quarter ended March 31, 2021, our Quarterly Report on Form 10-Q for
the quarter ended June 30, 2021, our Quarterly Report on Form 10-Q
for the quarter ended September 30, 2021, and in any subsequent
filings with the Securities and Exchange Commission. These
forward-looking statements speak only as of the date of this press
release. Factors or events that could cause our actual results to
differ may emerge from time to time, and it is not possible for us
to predict all of them. We undertake no obligation to update any
forward-looking statement, whether as a result of new information,
future developments or otherwise, except as may be required by
applicable law.
Contacts:
Investors:Chelcie ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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