Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced that the company will present new preclinical data from
several programs across its base editing portfolio at the American
Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting. The
meeting is being held May 16-19, 2022, in Washington, DC.
Beam’s presentations showcase a key component of the company’s
long-term strategy: making early and deliberate investments in
preclinical assessments to better understand levels of editing
required to generate therapeutic responses and to potentially
restore physiological function. Updated findings to be presented at
the meeting include data from two of the company’s in vivo,
liver-targeted programs – BEAM-301, its base editing program in
development for the treatment of glycogen storage disease type Ia
(GSDIa), and its base editing program in development for the
treatment of alpha-1 antitrypsin deficiency (Alpha-1). In addition,
Beam will present research highlighting its proprietary lipid
nanoparticle (LNP) delivery capabilities for potential in vivo
delivery to both T cells and natural killer (NK) cells, which could
have broad applications in both immunology and oncology.
“Beam research continues to advance on many fronts in parallel,
including both ex vivo and in vivo programs as well as novel
delivery technologies, and we are excited to present new
preclinical findings at ASGCT,” said Giuseppe Ciaramella, Ph.D.,
president and chief scientific officer of Beam. “Updated data from
our BEAM-301 in vivo candidate for GSDIa continue to show potent
and durable liver editing that translated into improved metabolic
parameters and survival in a mouse model of homozygous GSDIa,
combined with a favorable off-target profile, providing further
support for its advancement toward IND-enabling studies this year.
In addition, updated data from our Alpha-1 program demonstrate
Beam’s capabilities to optimize base editing for a challenging
target site, achieving significantly improved editing potency and
therapeutically relevant levels of gene correction in a clinically
relevant dose range.”
Dr. Ciaramella added, “Lastly, we’ve leveraged our proprietary
LNP capabilities to deliver mRNA to immune cells both ex vivo and
in vivo. In vivo delivery of mRNA encoding therapeutic transgenes
such as CARs to T and NK cells could circumvent certain critical
challenges associated with current-generation autologous CAR-T
therapies, potentially complementing our existing platform for
multiplex-edited allogeneic ex vivo CAR-T therapies. Collectively,
this research is exciting for Beam and the entire gene editing
field, bringing us closer to delivering important new
disease-modifying therapies to patients suffering from a wide range
of severe diseases.”
Details of the presentations are as
follows:
Title: Single, systemic administration of
BEAM-301 mitigates fasting hypoglycemia and restores metabolic
function in a transgenic mouse model of glycogen storage disease
type IaDate & Time: Monday, May 16, 2022, from
4:15-4:30 p.m. ETData Summary: GSDIa is an
autosomal recessive disorder caused by mutations in the G6PC gene
that disrupt a key enzyme, glucose-6-phosphatase (G6Pase), involved
in maintaining glucose homeostasis. Inhibition of G6Pase activity
results in low fasting blood glucose levels that can be fatal. Beam
is advancing BEAM-301, composed of a guide RNA and an mRNA encoding
an adenine base editor (ABE) delivered via LNP, which aims to
directly correct the R83C mutation, one of the primary
disease-causing mutations of GSDIa. Beam has previously shown
editing efficiencies of approximately 60% in liver extracts, with
survival of a GSDIa mouse model to three weeks of age without
hypoglycemia-induced seizures. Today’s data build on those
findings, showing that in a GSDIa mouse model, treated mice, which
otherwise have poor survival outcomes if left untreated, grew
normally to at least 35 weeks following administration of BEAM-301,
with survival ongoing in the study. Notably, as low as single digit
percentage base-editing rates were sufficient to restore
physiologically relevant levels of hepatic G6Pase activity,
normalize serum metabolites, and most importantly, prevent
hypoglycemia during a 24-hour fast. In addition, preliminary
off-target assessments have suggested a favorable profile of
BEAM-301.
Title: Optimized base editing reagents yield
more potent genetic correction in a mouse model of alpha-1
antitrypsin deficiency (poster M-123)Date &
Time: Monday May 16, 2022, from 5:30-6:30 p.m.
ETData Summary: Alpha-1 is a rare, inherited
genetic disorder that can cause progressive lung and liver disease.
It is most commonly caused by a G-to-A point mutation – referred to
as the PiZ mutation – within the SERPINA1 gene, which produces
alpha-1 antitrypsin (AAT) protein. In healthy individuals, the AAT
protein is secreted from the liver and, in circulation, protects
the lungs from damage. In individuals with the PiZ mutation, AAT is
misfolded, preventing secretion and resulting in damaging build-up
in the liver, as well as loss of its protective function in the
lungs. Previous studies conducted by Beam, have shown the ability
of LNP-delivered base editing reagents to correct the PiZ mutation
in the livers of mouse models, suggesting the potential of Beam’s
base editors to treat both lung and liver manifestations of the
disease. In updated research to be presented at ASGCT, Beam
scientists sought to optimize both the ABE and the guide RNA used
to correct the disease-causing PiZ mutation, with the improvements
over the original reagents leading to a greater than two-fold
increase in editing potency and therapeutically relevant increases
in circulating AAT in mice treated at clinically relevant doses
(<1mg/kg). Further, similar results were seen in adult mice
dosed at greater than 37 weeks, a treatment context more similar to
what might be encountered in a clinical setting.
Title: Efficient LNP delivery of mRNA in vivo
and in vitro to T and NK cells (poster Tu-107)Date &
Time: Tuesday May 17, 2022, from 5:30-6:30 p.m.
ETData Summary: Chimeric antigen receptor (CAR) T
cell therapy has demonstrated tremendous therapeutic potential in
the treatment of some cancers, but certain challenges such as
complex manufacturing and logistics associated with autologous
therapies, immunological rejection of allogeneic therapies, cost of
goods, and patient lymphodepletion prior to receipt of the therapy
limit the utility of current approaches. Leveraging its proprietary
LNP screening and delivery technology, Beam researchers evaluated
the potential to deliver mRNA directly to T and NK cells in vivo to
overcome these challenges. In mouse models, dose dependent
expression of reporter proteins in T and NK cells was observed
following a single intravenous LNP dose. LNP transfection was
further evaluated in several additional studies, showing:
- In human cells, ex vivo delivery of LNP resulted in reporter
protein expression in over 90% of T cells and 70% of NK cells.
- In an in vivo humanized mouse model, reporter protein
expression was observed in approximately 10% and 20% of T and NK
cells in bone marrow, respectively, and 40% of both T and NK cells
in the spleen.
- In non-human primates, reporter protein expression was observed
in up to 7% of bone marrow T cells.
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 presentations at ASGCT, our
plans, and anticipated timing, to initiate IND-enabling studies;
the therapeutic applications and potential of our technology,
including with respect to GSDIa, Alpha-1, CAR-T and CAR-NK cells,
and LNPs, including our ability to deliver base editors to target
organs in and beyond the liver; 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, 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
Beam Therapeutics (NASDAQ:BEAM)
과거 데이터 주식 차트
부터 6월(6) 2024 으로 7월(7) 2024
Beam Therapeutics (NASDAQ:BEAM)
과거 데이터 주식 차트
부터 7월(7) 2023 으로 7월(7) 2024