Research includes innovations in mRNA and
protein engineering and delivery science to improve therapeutic
properties
Highlights research results from ongoing
collaborations with International AIDS Vaccine Initiative (IAVI),
National Institute of Allergy and Infectious Diseases (NIAID) and
Bill & Melinda Gates Foundation toward development of an HIV
vaccine
Moderna, Inc., (Nasdaq: MRNA) a clinical stage biotechnology
company pioneering messenger RNA (mRNA) therapeutics and vaccines
to create a new generation of transformative medicines for
patients, today announced new research to be highlighted at the
Company’s third annual Science Day, held virtually this year. The
program is designed to provide insight into the continued diverse
efforts underway at Moderna and with collaborators to better
understand how to use mRNA as a medicine, and underscores the
Company’s continued commitment to basic science and innovation.
“Science Day is an opportunity for us to provide insights into
the advancements in our platform science and our further
understanding of how to use mRNA as a medicine. Our substantial
investments in basic science to date have resulted in major steps
forward in our platform’s capabilities, and these have allowed us
to open new therapeutic areas and new scientific directions,” said
Stephen Hoge, M.D., President of Moderna. “Today, we're excited to
highlight novel approaches to our lipid nanoparticle technology,
which will be used with mRNA-3745, our GSD1a candidate in
preclinical development. We are also pleased to provide an update
on our collaboration with IAVI, NIAID and Bill & Melinda Gates
Foundation toward the development of an HIV vaccine using Moderna’s
mRNA platform. We remain firmly committed to further advancing our
mRNA science to create a new generation of transformative medicines
for patients.”
At this year’s Science Day, Moderna will present new platform
science and preclinical research, including:
Engineering mRNA and Proteins to Improve Therapeutic
Properties
An advantage of mRNA medicines over small molecule drugs is the
ease of engineering their properties to achieve desired
pharmacology. New research will be presented on efforts to engineer
both mRNAs and encoded proteins to improve and extend therapeutic
effect.
These efforts include modifications to mRNA and proteins to
prevent degradation and lengthen half-life. Presentations will
include data on the addition of an inverted deoxythymidine (idT) to
the 3’-end of mRNA, which is designed to stabilize mRNA by blocking
its degradation through deadenylation. Preclinical data show that
3’-idT-stabilized mRNA encoding phenylalanine hydroxylase (PAH),
the enzyme missing or dysfunctional in the metabolic disorder
phenylketonuria (PKU), supports sustained reduction of serum
phenylalanine levels in a PKU mouse model. Data will also be
presented on removal of ubiquitination sites from PAH, designed to
prevent ubiquitin-mediated protein degradation. Results show that
combining mRNA stabilization along with PAH stabilization
maintained low serum phenylalanine levels even further, suggesting
an additive effect.
One challenge for the production of mRNA therapeutics is to
minimize or eliminate double-stranded RNA (dsRNA) impurities
produced during the synthesis process, as this can trigger
undesirable innate immune responses in vivo. At Science Day,
Moderna will present a protein engineering workflow by which it has
engineered a Moderna T7 RNA polymerase (MT7) that does not produce
dsRNA impurities.
Optimizing Lipid Nanoparticle Technology
The Company will present new research in delivery science to
optimize its lipid nanoparticles (LNPs). The presentations will
highlight a novel squaramide-based ionizable lipid designed to
enhance the interactions between the lipid and mRNA. Preclinical
data from new LNPs incorporating this novel ionizable lipid show
improved protein expression after IV administration, including
repeat dosing, and efficient delivery to the liver, as compared to
current state-of-the-art Moderna proprietary LNPs.
This squaramide-based LNP represents a new delivery system and
will be used with the Company’s glycogen storage disease type 1a
(GSD1a) candidate (mRNA-3745), which is in preclinical development.
Preclinical studies with this new LNP containing mRNA encoding for
G6Pase, the missing or dysfunctional enzyme in GSD1a, show
sustained improvements in fasting blood glucose in a mouse model of
GSD1a, achieving the target product profile.
Collaborating on an HIV Vaccine
Science Day will also include presentations from William Schief,
Ph.D., Professor, Immunology and Microbiology, Scripps Research
Institute and Executive Director, Vaccine Design, International
Aids Vaccine Initiative (IAVI) and Paolo Lusso, M.D., Ph.D., Chief,
Viral Pathogenesis, Laboratory of Immmunoregulation, National
Institute of Allergy and Infectious Diseases (NIAID) showcasing
research conducted through Moderna’s ongoing collaborations with
IAVI, NIAID and the Bill & Melinda Gates Foundation. This
research seeks to deliver an engineered HIV immunogen using
Moderna’s mRNA platform that allows for a faster, more flexible
approach to rapid iterative vaccine design and clinical testing.
This approach could significantly accelerate early clinical trials
and transform HIV vaccine science and development efforts. The data
presented today will highlight progress toward creating a
protective HIV-1 vaccine designed to elicit broadly neutralizing
antibodies to prevent HIV-1 infection.
Moderna currently has 23 mRNA development candidates in its
portfolio with 13 in clinical studies. Across Moderna’s pipeline,
more than 1,900 participants have been enrolled in clinical
studies. The Company’s updated pipeline can be found at
www.modernatx.com/pipeline. Moderna and collaborators have
published more than 45 peer-reviewed papers.
Science Day Webcast Information
Moderna will host its annual Science Day for analysts and
investors at 8:00 a.m. ET on Tuesday, June 2. A live webcast will
be available under “Events and Presentations” in the Investors
section of the Moderna website at investors.modernatx.com. A replay
of the webcast will be archived on Moderna’s website for one year
following the presentation.
About Moderna
Moderna is advancing messenger RNA (mRNA) science to create a
new class of transformative medicines for patients. mRNA medicines
are designed to direct the body’s cells to produce intracellular,
membrane or secreted proteins that can have a therapeutic or
preventive benefit and have the potential to address a broad
spectrum of diseases. The company’s platform builds on continuous
advances in basic and applied mRNA science, delivery technology and
manufacturing, providing Moderna the capability to pursue in
parallel a robust pipeline of new development candidates. Moderna
is developing therapeutics and vaccines for infectious diseases,
immuno-oncology, rare diseases, cardiovascular diseases, and
autoimmune and inflammatory diseases, independently and with
strategic collaborators.
Headquartered in Cambridge, Mass., Moderna currently has
strategic alliances for development programs with AstraZeneca PLC
and Merck & Co., Inc., as well as the Defense Advanced Research
Projects Agency (DARPA), an agency of the U.S. Department of
Defense, and BARDA. Moderna has been named a top biopharmaceutical
employer by Science for the past five years. To learn more, visit
www.modernatx.com.
Forward Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended, including regarding the Company’s mRNA and
encoded protein engineering efforts, the Company’s new
squaramide-based LNP and its expected use with mRNA-3745 and other
new development candidates, the potential for the Company’s mRNA
platform to significantly accelerate early clinical trials and
transform HIV vaccine science and development efforts, and the
presentation of research and data at the Company’s upcoming Science
Day. In some cases, forward-looking statements can be identified by
terminology such as “will,” “may,” “should,” “could”, “expects,”
“intends,” “plans,” “aims,” “anticipates,” “believes,” “estimates,”
“predicts,” “potential,” “continue,” or the negative of these terms
or other comparable terminology, although not all forward-looking
statements contain these words. The forward-looking statements in
this press release are neither promises nor guarantees, and you
should not place undue reliance on these forward-looking statements
because they involve known and unknown risks, uncertainties, and
other factors, many of which are beyond Moderna’s control and which
could cause actual results to differ materially from those
expressed or implied by these forward-looking statements. These
risks, uncertainties, and other factors include, among others: the
fact that there has never been a commercial product utilizing mRNA
technology approved for use; potential adverse impacts due to the
global COVID-19 pandemic such as delays in regulatory review,
manufacturing and supply chain interruptions, adverse effects on
healthcare systems and disruption of the global economy; and those
other risks and uncertainties described under the heading “Risk
Factors” in Moderna’s most recent Quarterly Report on Form 10-Q
filed with the U.S. Securities and Exchange Commission (SEC) and in
subsequent filings made by Moderna with the SEC, which are
available on the SEC’s website at www.sec.gov. Except as required
by law, Moderna disclaims any intention or responsibility for
updating or revising any forward-looking statements contained in
this press release in the event of new information, future
developments or otherwise. These forward-looking statements are
based on Moderna’s current expectations and speak only as of the
date hereof.
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version on businesswire.com: https://www.businesswire.com/news/home/20200602005212/en/
Moderna Contacts
Media: Colleen Hussey Senior Manager, Corporate
Communications 203-470-5620 Colleen.Hussey@modernatx.com
Dan Budwick 1AB 973-271-6085 Dan@1abmedia.com
Investors: Lavina Talukdar Head of Investor Relations
617-209-5834 Lavina.Talukdar@modernatx.com
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