Beam Therapeutics to Present First Research Highlighting Approach to Develop Non-Genotoxic Conditioning Regimens for Patients with Sickle Cell Disease Ahead of Autologous Transplant
27 6월 2022 - 7:30PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced that new research highlighting the company’s internal
efforts to develop improved transplant conditioning regimens for
patients with sickle cell disease (SCD) undergoing hematopoietic
stem cell transplantation (HSCT) will be presented today, June 27,
2022, at 4:00 p.m. CEST at the Federation of American Societies for
Experimental Biology (FASEB) Genome Engineering Conference by
Nicole Gaudelli, Ph.D., director, head of gene editing at Beam. The
conference is being held from June 26-30, 2022, in Lisbon,
Portugal.
Beam is advancing two ex vivo base editing programs for SCD:
BEAM-101, which incorporates base edits that are designed to mimic
single nucleotide polymorphisms seen in individuals with hereditary
persistence of fetal hemoglobin, and BEAM-102, which directly edits
the causative HbS point mutation to recreate a naturally occurring
normal human hemoglobin variant, HbG-Makassar. In the second half
of 2022, Beam plans to initiate patient enrollment in its clinical
trial with BEAM-101 and to submit an investigational new drug
application for BEAM-102.
Beam has laid out a long-term strategy to support broad
accessibility of base editing treatments for patients with SCD and
other hematologic diseases. A key component of this strategy is
focused on improving the safety of conditioning regimens, a
required pretreatment for patients receiving ex vivo gene editing
treatment via autologous transplant. Today’s conditioning regimens
rely on nonspecific chemotherapy or radiation, which are associated
with significant toxicities, including genotoxicity, primary or
secondary malignancy, and organ toxicities including infertility.
With a goal of overcoming this, 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) called ESCAPE, or Engineered Stem
Cell Antibody Paired Evasion.
“As we execute on our long-term strategy to develop base editing
treatments for SCD, we are excited to share new findings around our
pre-clinical research to identify improved conditioning regimens
for patients ahead of autologous transplant,” said Giuseppe
Ciaramella, Ph.D., president and chief scientific officer of Beam.
“The findings being presented today showcase the first data from
our efforts to advance an improved non-genotoxic conditioning
approach, coupling a monoclonal antibody with multiplex base edited
HSCs that both ‘ESCAPE’ antibody binding and contain
disease-corrective edits to potentially ameliorate the clinical
manifestations of SCD. By exploiting the unique multiplex
capabilities of base editing in these next-generation conditioning
and cell-engineering approaches, we aim to develop a curative
treatment for patients with SCD that avoids the safety and
fertility concerns associated with current conditioning regimens.
These findings are a major step forward in our efforts to enable
potentially highly efficacious conditioning options for patients
with SCD and could significantly increase the probability of
success of non-genotoxic conditioning, which is a key priority in
the field. We look forward to rapidly advancing this work as part
of our long-term strategy to bring safer and more effective options
forward for patients with SCD.”
New antibody-based conditioning agents have shown promise in
targeting CD117, an optimal conditioning target for eliminating
HSCs, but such antibodies generally cannot discriminate between
host (disease-carrying) and transplanted (disease-corrected) cells,
and therefore are designed with short half-life or dosed at low
concentrations well before transplant. To potentially solve for
this and other safety concerns associated with current conditioning
regimens, Beam scientists developed ESCAPE, whereby an
edit-antibody pair targeting CD117 was designed to enable edited
HSCs to function normally but escape the binding of the
conditioning antibody. This strategy is intended to allow the
conditioning antibody to continue clearing older unedited host
cells while selectively allowing new edited cells to proliferate in
the body during engraftment.
The findings show that in vitro the ESCAPE antibodies bound to
wild-type CD117, blocked binding of its ligand and led to depletion
of unedited cells, while enriching for edited cells which were
generally not bound by the antibody. High levels of editing
efficiency were demonstrated with both a single CD117 edit and
simultaneous CD117 and BEAM-101 edits (~85% multiplex editing).
Beam has also developed a CD117 editing strategy with greater than
75% editing efficiency that is also compatible with an edit to
correct the sickle mutation and generate HbG-Makassar, Beam’s
strategy with its BEAM-102 program. Relative to a control, ESCAPE
reduced cell viability of unedited cells while maintaining CD117
edited cells in vitro, suggesting utility as a
conditioning agent with a selective advantage to edited HSCs
post-transplant.
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 presentation at FASEB; our
plans, and anticipated timing, to initiate patient enrollment in
our BEAM-101 clinical trial and to submit an investigational new
drug application for Beam-102; the therapeutic applications and
potential of our technology, including with respect to improved
conditioning regimens and sickle cell disease; 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, 2021, under the heading “Risk Factors”
in our Quarterly Report on Form 10-Q for the quarter ended March
31, 2022, 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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