Beam Therapeutics Announces Updated Preclinical Data Highlighting Optimized LNP Delivery Approaches for In Vivo Base Editing to the Liver and Other Tissues
23 9월 2021 - 7:30PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced new preclinical data highlighting advancements with the
company’s approach to developing novel lipid nanoparticle (LNP)
formulations for in vivo liver editing. In addition, the company
reported initial in vivo data demonstrating its delivery
capabilities to tissues outside of the liver. The data will be
presented today, September 23, 2021, from 11:15 – 11:45 a.m. ET at
the TIDES USA Oligonucleotide & Peptide Therapeutics Conference
(TIDES 2021) in a presentation titled “Optimization of LNP for in
vivo base editing.”
“We are committed to advancing innovative genetic medicines to
reach as many patients as possible, and our progress toward that is
exemplified by the data reported today with our novel delivery
technologies,” said Giuseppe Ciaramella, Ph.D., president and chief
scientific officer of Beam. “Continued optimization of our LNPs has
led to a substantial increase in liver editing potency to what we
believe could be a clinically relevant dose for our lead LNP
program, which we plan to finalize later this year. Importantly,
data from our studies in non-human primates (NHPs) showed that
these formulations were well tolerated and had good early storage
stability in ongoing studies. We’ve also generated encouraging data
using our novel LNP formulations gained through our acquisition of
Guide Therapeutics, which could have meaningful application for
delivery of our base editors to tissues beyond the liver,
broadening the potential reach of our genetic medicines.”
Using an mRNA-encoding adenine base editor (ABE) and guide RNA,
Beam evaluated various LNP formulations and mRNA production
processes to optimize its LNPs for improved in vivo liver editing
and to avoid immune stimulation in NHPs. The findings highlight key
improvements in Beam’s LNP delivery system for the liver,
including:
- Increased editing potency in NHPs, demonstrating up to 60%
editing at 1.0 mg/kg;
- Well-tolerated formulations in NHPs treated with up to 1.5
mg/kg LNP, with minimal to mild increases in transient liver enzyme
elevations that were resolved by day 15 post-treatment; and
- Stable formulations with potency maintained at -20 and -80 ⁰C
out to three months.
In addition, Beam reported an update on its proprietary approach
to developing LNPs to deliver base editors to tissues beyond the
liver, including hematopoietic stem and progenitor cells (HSPCs).
Leveraging its DNA barcoding technology, Beam identified a family
of LNPs for delivery of base editors to HSPCs in mice, with
administration at 1.0 mg/kg leading to 40% expression of mRNA cargo
in cells. Beam is evaluating this delivery approach for potential
application in hemoglobinopathies and other genetic blood
disorders.
About Beam Therapeutics
Beam 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.
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 data presentations at upcoming scientific
conferences; our belief that we may have identified a clinically
relevant dose for our lead LNP program; our ability to deliver base
editors to tissues beyond the liver; and the therapeutic
applications and potential of our technology, including our ability
to develop life-long, curative, precision genetic medicines for
patients through base editing. Each forward-looking statement is
subject to 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 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 (except as
otherwise noted) 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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