Beam Therapeutics to Present New Preclinical Data Highlighting Non-Genotoxic Conditioning Regimens for Patients with Sickle Cell Disease Ahead of Autologous Transplant at the 64th ASH Annual Meeting
November 03 2022 - 9:01AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced that new data supporting the advancement of its
Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning
approach will be presented during poster sessions at the 64th
Annual American Society of Hematology (ASH) Annual Meeting and
Exposition. The meeting is being held December 10-13, 2022, in New
Orleans.
Beam is advancing ESCAPE as part of its long-term strategy to
support broad accessibility of base editing treatments for patients
with sickle cell disease (SCD) and other hematologic diseases. A
key component of the company’s Wave 2 strategy is focused on
improving the safety and tolerability of conditioning regimens, a
required pretreatment for patients receiving ex vivo gene editing
treatment via autologous transplant. To address toxicity challenges
associated with currently available conditioning regimens, Beam has
leveraged its base editing capabilities to develop a potentially
non-genotoxic approach that combines antibody-based conditioning
with multiplex gene edited hematopoietic stem cells (HSCs).
The data to be presented at ASH expand upon data presented
earlier this year for its ESCAPE-1 approach, which consists of
multiplex base edited HSCs that include a therapeutic edit for SCD
at the HGB1/2 gene designed to enable upregulation of fetal
hemoglobin and an additional simultaneous edit at CD117. Findings
showed that the edit-antibody pair targeting CD117, an optimal
conditioning target for eliminating HSCs, led edited HSCs to
function normally but escape the binding of the conditioning
antibody. In addition, Beam will present work on ESCAPE-2, in which
the company leveraged its initial ESCAPE work to screen guides that
were compatible, in a multiplex-edited HSC, with a next-generation
adenine base editor that could install the therapeutic HbG-Makassar
edit.
“Improving conditioning regimens for patients ahead of
autologous transplant is a key focus for Beam as part of Wave 2 of
our long-term strategy to bring best-in-class base editing
treatments to people with SCD,” said Giuseppe Ciaramella, Ph.D.,
president and chief scientific officer of Beam. “We are advancing
our ex vivo programs with standard conditioning under our Wave 1
strategy and are excited by this Wave 2 progress with both our
ESCAPE-1 and ESCAPE-2 approaches, which have the potential to
expand the number of patients who may benefit from our therapies.
These conditioning strategies take advantage of the strengths of
base editing for precise, efficient, multiplex editing without
double stranded breaks, and we are encouraged by the rapid progress
we are making on our vision for a potentially non-genotoxic
approach for conditioning in HSC transplant. This work is an
integral part of our SCD portfolio strategy, and we believe it may
also open many other potential applications in hematology. We look
forward to presenting these data at the upcoming ASH Annual
Meeting.”
Presentation Details: Title:
Engineered Stem Cell Antibody Paired Evasion 1 (ESCAPE-1): Paired
HSC Epitope Engineering and Upregulation of Fetal Hemoglobin for
Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy
Conditioning for the Treatment of Hemoglobinopathies
(1955)Session Name: 701. Experimental
Transplantation: Basic and Translational: Poster I Date
& Time: Saturday, December 10, 2022, 5:30-7:30 p.m.
Location: Ernest N. Morial Convention Center, Hall
D
Title: Engineered Stem Cell Antibody Paired
Evasion-2 (ESCAPE-2): Paired HSC Epitope Engineering and Direct
Editing of Sickle Allele for Antibody-Mediated Autologous
Hematopoietic Stem Cell Therapy Conditioning for the Treatment of
Sickle Cell Disease (4585)Session Name: 701.
Experimental Transplantation: Basic and Translational: Poster III
Date & Time: Monday, December 12, 2022,
6:00-8:00 p.m. Location: Ernest N. Morial
Convention Center, Hall D
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 is designed to enable
precise, predictable and efficient single base changes, at targeted
genomic sequences, without making double-stranded breaks in the
DNA. This has the potential to enable 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 upcoming presentations at
the ASH Annual Meeting and Exposition; the therapeutic applications
and potential of our technology, including with respect to SCD and
our conditioning regimens; 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,
including its impact on the global supply chain; the uncertainty
that our product candidates will receive regulatory approval
necessary to initiate human clinical studies; 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 and initiation 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, 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.
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not deemed to be incorporated by reference in this press
release.
Contacts:
Investors:Chelcie ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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