Beam Therapeutics Reports Progress Across Ex Vivo and In Vivo Pipeline of Base Editing Therapeutics and Outlines Key Anticipated 2022 Milestones
09 1월 2022 - 11:00PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
outlined anticipated 2022 milestones across its ex vivo programs
targeting editing of hematopoietic stem cells (HSCs) and T cells
and in vivo programs targeting editing of liver cells leveraging
lipid nanoparticles (LNPs) for delivery. Updates include that the
company has selected its fourth development candidate and first in
vivo base editing candidate, BEAM-301, which aims to correct the
R83C mutation for the potential treatment of patients with glycogen
storage disorder Ia (GSDIa).
“We made significant progress across our base editing portfolio
in 2021, which culminated in U.S. Food and Drug Administration
clearance of the first investigational new drug application of a
base editing therapeutic, BEAM-101. We also further expanded our
platform, particularly with LNP delivery of base editors to the
liver and our proprietary technology for accelerating LNP delivery
to other tissues, including HSCs,” said John Evans, chief executive
officer of Beam. “We believe 2022 is set to be our most important
year yet, with preparations underway to launch the BEACON-101
clinical trial with BEAM-101 for the treatment of sickle cell
disease and to complete our transition to becoming a clinical-stage
company. We believe we are well positioned today, with four
development candidates, a rich pipeline of earlier stage programs,
and an industry-leading platform of editing and delivery
technologies enabling us to bring forward a new class of precision
genetic medicines. None of this would be possible without the
commitment of our remarkable team of fearless innovators. We look
forward to the year ahead and continuing our work to bring
potentially life-changing medicines to as many patients as
possible.”
Ex Vivo HSC Programs
- BEAM-101 is a patient-specific, autologous HSC investigational
therapy, which incorporates base edits that are designed to mimic
single nucleotide polymorphisms seen in individuals with hereditary
persistence of fetal hemoglobin. BEAM-101 aims to potentially
alleviate the effects of mutations causing sickle cell disease
(SCD) or beta-thalassemia by leading to increases in fetal
hemoglobin, which inhibits hemoglobin S (HbS) polymerization. The
BEACON-101 trial is a Phase 1/2 clinical trial designed to assess
the safety and efficacy of BEAM-101 for the treatment of SCD. The
trial is expected to include an initial “sentinel” cohort of three
patients, treated one at a time to confirm successful engraftment,
followed by dosing in up to a total of 45 patients. Beam has begun
site selection and the institutional review board approval
processes for the BEACON-101 trial and plans to enroll the first
subject in the second half of 2022.
- BEAM-102 is designed 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 more common HbA
variant and does not cause SCD. Beam plans to submit an
investigational new drug (IND) application for BEAM-102 in the
second half of 2022.
Ex Vivo T Cell Programs
- BEAM-201 is a multiplex base edited anti-CD7 CAR-T cell
investigational therapy designed to treat relapsed/refractory T
cell acute lymphoblastic leukemia, a severe disease affecting
children and adults. Beam plans to submit an IND application for
BEAM-201 in the second half of 2022.
- Beam plans to nominate a second CAR-T development candidate in
2022.
In Vivo LNP Liver-targeting
Programs
- BEAM-301, the company’s newest development candidate, is a
liver-targeting LNP formulation of base editing reagents designed
to correct the R83C mutation. R83C is the most common
disease-causing mutation of GSDIa, a life-altering genetic disease
with no approved disease-modifying treatments available today. Beam
anticipates initiating IND-enabling studies for BEAM-301 in
2022.
- Beam plans to nominate a second liver-targeted development
candidate in 2022.
J.P. Morgan Healthcare ConferenceMr. Evans will
present Beam’s pipeline and business updates during a presentation
at the 40th Annual J.P. Morgan Healthcare Conference on Monday,
January 10, 2022, at 2:15 p.m. ET. A live webcast will be available
in the investor section of the company’s website at www.beamtx.com
and will be archived for 60 days following the presentation.
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 plans, and anticipated
timing, to nominate additional development candidates, initiate
IND-enabling studies, and submit IND applications; the therapeutic
applications and potential of our technology, including with
respect to sickle cell disease, beta-thalassemia, T-ALL, GSDIa, and
LNPs; the planned initiation and design of our BEACON-101 clinical
trial, including the timing of enrolling the first subject in the
trial; our planned presentations at an upcoming conference; 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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