Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
shared a three-stage, long-term development strategy for its base
editing approach to treat sickle cell disease (SCD). The plan, and
supportive data, are being presented during a Scientific Program
Session oral presentation and two poster sessions at the 63rd
American Society for Hematology (ASH) Annual Meeting &
Exposition.
SCD is a severe, hereditary monogenic blood disorder that alters
the structure and function of oxygen-carrying hemoglobin in red
blood cells and is caused by a single letter spelling error in the
beta globin gene. To fully address SCD and support broad
accessibility, Beam is deploying a stepwise strategy that includes
advancements of its ex vivo programs, BEAM-101 and BEAM-102,
improvements in patient conditioning regimens, and enablement of in
vivo base editing with delivery directly into patients via lipid
nanoparticles (LNPs).
“Our goal is to make disease-altering, lifelong treatments with
base editing broadly accessible to SCD patients around the globe,
and, over time, improve on the delivery of these therapies so
patients everywhere can opt for a potentially curative therapy,”
said John Evans, chief executive officer of Beam. “We have focused
our initial SCD efforts on our ex vivo programs, BEAM-101 and
BEAM-102, which leverage established clinical and regulatory
strategies and have the potential to offer transformative
therapeutic options for SCD patients. To further improve the
therapeutic options for SCD patients, we also aim to develop
lower-toxicity conditioning methods for improved transplant, as
well as pursuing in vivo delivery of our base editors using our
innovative LNP delivery capabilities. We believe this suite of
technologies – base editing, improved conditioning and in vivo
delivery for editing HSCs – can maximize the potential of our SCD
programs as well as create a powerful platform for the treatment of
many other severe genetic blood disorders.”
During the presentations at ASH, Beam is highlighting its
stepwise strategy for treating SCD and is presenting complementary
data supporting several components of its approach:
Wave 1: Ex Vivo Base
Editing via Autologous TransplantBeam is advancing ex vivo
base editing programs, in which cells are collected from a patient,
edited and then infused back into the patient following a
conditioning regimen, such as treatment with busulfan, the standard
of care in hematopoietic stem cell (HSC) transplantation today.
This approach is being deployed in the company’s BEAM-101 and
BEAM-102 base editing programs, and allows the company to pursue an
efficient path for development using increasingly validated
clinical endpoints and regulatory strategies.
BEAM-101 is an investigational, patient-specific, autologous HSC
therapy which incorporates base edits that mimic single nucleotide
polymorphisms seen in individuals with hereditary persistence of
fetal hemoglobin to potentially alleviate the effects of mutations
causing SCD or beta-thalassemia. The company recently received
clearance of its Investigational New Drug application for this
program and is working to initiate its Phase 1/2 BEACON-101
trial.
BEAM-102 aims to treat SCD by directly editing the causative HbS
point mutation to recreate a naturally occurring normal human
hemoglobin variant, HbG-Makassar. The Makassar variant has been
reported to have the same function as the wild-type variant and
does not cause SCD.
During ASH, Beam is presenting updated preclinical data further
characterizing Makassar hemoglobin created by BEAM-102 and
demonstrating biophysical and biochemical properties consistent
with normal hemoglobin, as expected:
- Makassar globin did not polymerize in vitro
- Cells co-expressing Makassar globin and sickle globin had
properties similar to sickle trait cells, which are cells with one
normal globin gene and one sickle globin gene and which do not
sickle
- Oxygen binding of Makassar globin was comparable to the most
abundant normal adult hemoglobin (HbA)
- The crystal structures of the Makassar globin and of HbA were
superimposable, suggesting no meaningful structural
differences
Wave 2: Improved Conditioning In parallel with
Wave 1 development, Beam also aims to improve the transplant
conditioning regimen for SCD patients, reducing toxicity challenges
associated with standard of care. Conditioning is a critical
component necessary to prepare a patient’s body to receive the ex
vivo edited cells that must engraft in the patient’s bone marrow in
order to be effective. Today’s conditioning regimens rely on
nonspecific chemotherapy or radiation, which are associated with
significant toxicities. Beam has a collaboration with Magenta
Therapeutics to evaluate the potential utility of MGTA-117,
Magenta’s novel antibody drug conjugate that is designed to
precisely target only hematopoietic stem and progenitor cells,
sparing immune cells. Importantly, improved conditioning regimens
could potentially be paired with BEAM-101 and BEAM-102, as well as
other base editing programs in hematology.
Wave 3: In Vivo Base
Editing via HSC-targeted LNPsBeam is also exploring the
potential for in vivo base editing programs for SCD, in which base
editors would be delivered to the patient through an infusion of
LNPs targeted to HSCs, eliminating the need for transplantation
altogether. This approach could provide a more accessible option
for patients, particularly in regions where ex vivo treatment is
challenging. Building on its acquisition of Guide Therapeutics,
Beam has established a DNA-barcoded LNP screening technology to
enable high-throughput in vivo identification of LNPs with novel
biodistribution and selectivity for target organs beyond the
liver.
During ASH, Beam is presenting updated preclinical data using
this technology to screen more than 1,000 LNPs for potential to
deliver to HSCs and identified LNP-HSC1 as the most potent, with
efficient transfection in both mice and non-human primates. Updated
findings in the poster showed:
- LNP-HSC1 was validated in vivo, leading to durable,
dose-dependent mRNA transfection in HSCs and resulting in
fluorescent reporter expression in more than 40% of cells, now
maintained out to 16 weeks post-delivery
- LNP-HSC1 efficiently transfected human CD34+ cells in
vitro
- LNP-HSC1 efficiently transfected nearly 20% of CD34+ HSCs in
humanized mice and non-human primates at a dose of 1.0 mg/kg.
Beam is continuing work to identify more potent LNPs for
applications in in vivo base editing of HSCs. As with Wave 2, Beam
can potentially leverage the same base editing payloads used in
BEAM-101 and BEAM-102 for future in vivo programs, as well as
expanding the use of the technology to other indications in
hematology.
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 strategic initiatives for
our base editing program targeting SCD; 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, conditioning and LNP
screening; the planned initiation of our BEACON-101 trial; 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 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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