Beam Therapeutics Announces Upcoming Preclinical Data Presentations at the American Society of Hematology Annual Meeting
04 11월 2021 - 10:40PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced that preclinical data highlighting the company’s Makassar
base editing approach for the potential treatment of sickle cell
disease (SCD) and its proprietary lipid nanoparticle (LNP)
screening capabilities will be presented during two poster sessions
at the 63rd American Society of Hematology (ASH) Annual Meeting. In
addition, Beam will present an overview on the application of base
editing for the treatment of beta-hemoglobinopathies and other
genetic blood disorders during a Scientific Program session titled,
“Gene Editing 2.0: Advances in Gene Editing to Treat Genetic Blood
Disorders.” The ASH Annual Meeting is being held December 11-14,
2021, virtually and in Atlanta.
“We look forward to presenting these new data at ASH, which
further support our leadership position in the field of base
editing and the significant opportunity we have to potentially make
a difference for patients in need of meaningful treatment options,”
said Giuseppe Ciaramella, Ph.D., president and chief scientific
officer of Beam. “The additional preclinical data being presented
from our Makassar program continue to validate the potential of our
base editing approach to treating SCD, which is designed to correct
sickle globin to a normally functioning hemoglobin variant. In
addition, the ability of our in vivo high-throughput LNP screening
technology to identify novel LNPs capable of delivering our editors
to stem cells is exciting and supports a promising delivery
platform that could eventually eliminate the need for transplant in
sickle cell disease and other genetic blood disorders. Taken
together, these data, coupled with our ongoing work around
non-genotoxic conditioning, give us hope to be able to provide a
more holistic treatment approach for patients with SCD and
beyond.”
Details of the poster presentations are as follows:
Poster Title: Conversion of HbS to Hb
G-Makassar by Adenine Base Editing is Compatible with Normal
Hemoglobin FunctionSession Name: 113.
Hemoglobinopathies, Excluding Thalassemia: Basic and Translational:
Poster IDate: Saturday, December 11,
2021Presentation Time: 5:30p.m -
7:30p.m.Location: Georgia World Congress Center,
Hall B5Abstract Summary: Beam is advancing
BEAM-102, its Makassar base editing approach, as a potential
treatment for SCD that is constructed to directly edit the
causative HbS point mutation to recreate a naturally occurring
normal human hemoglobin variant, HbG-Makassar. The Makassar variant
has been reported to be a functional beta-hemoglobin that is not
pathogenic. In preclinical studies, Beam sought to further
characterize Makassar hemoglobin, evaluating its size, molecular
weight, oligomerization and polymerization potential, oxygen
binding properties, as well as solving its crystal structure.
Altogether, Beam’s biophysical and biochemical characterization
shows that Makassar globin behaves as a functional hemoglobin and
represents a promising investigational approach for the treatment
of sickle cell disease.
Poster Title: Screening of Chemically Distinct
Lipid Nanoparticles In Vivo Using DNA Barcoding Technology Towards
Effectively Delivering Messenger RNA to Hematopoietic Stem and
Progenitor CellsSession Name: 801. Gene Therapies:
Poster IIDate: Sunday, December 12,
2021Presentation Time: 6:00p.m. -
8:00p.m.Location: Georgia World Congress Center,
Hall B5Abstract Summary: Beam is developing an
approach to directly deliver base editors to hematopoietic stem and
progenitor cells (HSPCs) in vivo through non-viral delivery
methods, such as LNPs for the potential delivery of base editors as
a treatment for a range of hemoglobinopathies. To identify optimal
LNPs for delivery to HSPCs, leveraging its proprietary DNA
barcoding technology, Beam screened more than 1,000 LNPs and
identified LNP-HSC1 as the most potent. LNP-HSC1 was validated in
vivo, leading to durable, dose-dependent mRNA transfection in HSPCs
of more than 40%, which was maintained for 10 weeks post-delivery.
Further, LNP-HSC1 efficiently transfected mice at doses ranging
from 0.3mg/kg-1.0mg/kg, and in non-human primates, a dose-dependent
increase in mRNA in bone marrow-derived CD34+ HSPCs was
observed.
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 and LNP
screening data presentations at an upcoming scientific conference;
the therapeutic applications and potential of our technology,
including with respect to SCD and LNP screening; 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, 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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