Beam Therapeutics Enrolls First Patient in BEACON Clinical Trial of BEAM-101 Base Editing Therapy Candidate for the Treatment of Sickle Cell Disease
November 14 2022 - 6:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced that the first patient has been enrolled in the company’s
BEACON trial. BEACON is an open-label, single-arm, multicenter,
Phase 1/2 clinical trial designed to evaluate the safety and
efficacy of BEAM-101 in adult patients with severe sickle cell
disease (SCD).
BEAM-101 is a patient-specific, autologous hematopoietic stem
cell (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 SCD by leading to increases in fetal hemoglobin (HbF),
which inhibits hemoglobin S (HbS) polymerization. Using base
editing, a next-generation form of CRISPR, BEAM-101 in preclinical
studies featured high levels of HSC editing (over 90% of alleles
edited), high and consistent levels of upregulation of HbF (over
60% of total hemoglobin), and significant reductions in the
disease-causing protein HbS (less than 40% of total hemoglobin) –
levels that are similar to sickle cell trait carriers, who do not
have SCD. Unlike nuclease editors, the BEAM-101 base editor is
designed to avoid double stranded breaks during editing, which can
result in unwanted chromosomal abnormalities and genotoxic
stress.
Following enrollment in the BEACON trial, each patient will
undergo a transfusion and mobilization process for HSC retrieval.
The cells are then edited, creating an autologous BEAM-101
investigational drug product. Following the drug product
manufacturing, patients in the trial will receive pre-treatment
conditioning using a standard-of-care chemotherapy regimen, after
which the edited cells are transplanted back into the patient. 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.
“The enrollment of the first patient in our BEACON trial is a
significant step forward for Beam and for the field of base
editing,” said John Evans, chief executive officer of Beam. “With
the potency and precision of base editing, we believe BEAM-101
could be a best-in-class option for SCD patients with several
advantages over other available genetic therapies. We are now
focused on activating additional clinical trial sites in the U.S.,
modifying the BEACON protocol to enable expedited future patient
enrollment and endpoint assessment, and finalizing our
commercial-ready manufacturing process. We look forward to
advancing this program for patients who suffer from the painful and
debilitating consequences of SCD.”
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: the clinical trial protocol for
BEACON; the therapeutic applications and potential of our
technology, including with respect to SCD; 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, including its impact on the global supply chain;
the uncertainty that our product candidates will receive regulatory
approval necessary to initiate human clinical studies; 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 and initiation 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, our Quarterly Report on Form 10-Q for the
quarter ended September 30, 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.
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release.
Contacts:
Investors:Chelcie ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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