Moderna to Present Preclinical Data at 2019 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting
April 29 2019 - 8:00AM
Business Wire
The Company and academic collaborators to share
data from seven preclinical studies demonstrating the potential of
mRNA-based therapies to treat rare metabolic and genetic
disorders
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 that it will present, along with
academic collaborators, preclinical data from seven studies that
support the potential of mRNA-based therapies to treat the
underlying cause of several rare metabolic and genetic disorders.
These data, including five oral presentations, will be presented at
the 2019 American Society of Gene & Cell Therapy (ASGCT) Annual
Meeting taking place April 29 – May 2 in Washington, D.C.
“We are pleased to present these data that show the potential of
our mRNA platform and lipid nanoparticle delivery technology to
encode for missing or malfunctioning proteins that are at the root
of a diverse array of rare diseases,” said Paolo Martini, Ph.D.,
chief scientific officer of rare diseases at Moderna. “These
studies add to our growing body of published preclinical work in
rare metabolic and genetic disorders, and build upon our
understanding of the potential for mRNA-based treatments to express
required proteins or modulate immune responses as we continue our
work to bring additional investigational mRNA medicines into the
clinic.”
Presentations at the meeting include pre-clinical data from
studies of the following disorders:
- Ornithine transcarbamylase deficiency
(OTC), in collaboration with the Gene Therapy Program at the
Perelman School of Medicine, University of Pennsylvania
(Penn);
- Maple syrup urine disease (MSUD), in
collaboration with Penn;
- Arginase-1 (ARG1) deficiency, in
collaboration with the University of California, Los Angeles
Department of Surgery and Department of Molecular and Medical
Pharmacology;
- Factor VIII deficiency (hemophilia A),
in collaboration with Seattle Children’s Research Institute.
- Glycogen storage disorder type 1a
(GSD1a);
- Adult-onset type II citrullinemia
(CTLN2); and
- Progressive familial intrahepatic
cholestasis type 3 (PFIC3).
“mRNA medicines have the potential to treat the underlying cause
of many metabolic diseases, and may offer important advantages over
conventional gene and enzyme replacement therapies for eligible
patients,” said James M. Wilson, M.D., Ph.D., director of the Gene
Therapy Program in the Perelman School of Medicine at the
University of Pennsylvania. “This includes the potential to develop
controlled, dose-dependent and transient treatments that may
benefit infants and children with these disorders and patients with
diseases that are not addressable with current viral-based
approaches.”
Abstracts and presentations at ASGCT 2019
- Abstract #27: Liver-Directed Lipid
Nanoparticle mRNA Therapy Improves Survival and Reduces Serum
Branched Chain Amino Acids in a Mouse Model of Maple Syrup Urine
Disease (Greig et al., Oral Presentation, Monday, April 29, 9:00am
– 9:15am ET, Heights Courtyard 3 Room)
- Abstract #72: Systemic mRNA Therapy as
a Treatment for the Inherited Metabolic Liver Disorder Arginase
Deficiency (Truong et al., Oral Presentation, Monday, April 29,
11:45am – 12:00pm ET, Heights Courtyard 2 Room)
- Abstract #382: Immunomodulation of
Factor FVIII Inhibitors in Hemophilia A Mice Using Messenger RNA
Lipid Nanoparticles (Chen et al., Oral Presentation, Tuesday, April
30, 3:45pm – 4:00pm ET, Lincoln Room)
- Abstract #383: Efficient mRNA Therapy
for Treating Ornithine Transcarbamylase Deficiency in Two Mouse
Models (Wang et al., Oral Presentation, Tuesday, April 30, 4:00pm –
4:15pm ET, Lincoln Room)
- Abstract #709: mRNA Therapy Improves
Metabolic and Behavioral Abnormalities in a Murine Model of Citrin
Deficiency (Cao et al., Oral Presentation, Wednesday, May 1, 4:45pm
– 5:00pm ET, Heights Courtyard 1 Room)
- Abstract #768: MDR3/ABCB4 mRNA Therapy
for Treating Progressive Familial Intrahepatic Cholestasis 3
(PFIC3) (Cao et al., Poster Presentation, Date/Time: Wednesday May
1, 2019 5:00 PM - 6:00 PM, Columbia Hall)
- Abstract #797: mRNA Therapy for the
Treatment of Glycogen Storage Disease Type 1a (GSD1a) (Cao et al.,
Poster presentation, Date/Time: Wednesday May 1, 2019 5:00 PM -
6:00 PM, Columbia Hall)
At present, these research programs are not Moderna development
candidates.
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. Moderna’s platform builds on continuous
advances in basic and applied mRNA science, delivery technology and
manufacturing, providing the Company 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 and cardiovascular diseases,
independently and with strategic collaborators.
Headquartered in Cambridge, Mass., Moderna currently has
strategic alliances for development programs with AstraZeneca, Plc.
and Merck, Inc., as well as the Defense Advanced Research Projects
Agency (DARPA), an agency of the U.S. Department of Defense; the
Biomedical Advanced Research and Development Authority (BARDA), a
division of the Office of the Assistant Secretary for Preparedness
and Response (ASPR) within the U.S. Department of Health and Human
Services (HHS). Moderna has been ranked in the top ten of Science’s
list of top biopharma industry employers for the past four years.
To learn more, visit www.modernatx.com.
Special Note Regarding 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, but not limited to, statements
concerning: the potential of mRNA-based therapies to treat
rare metabolic and genetic disorders in humans, including the
potential to offer advantages over existing therapies, the
potential to develop controlled, dose-dependent, and transient
mRNA-based treatments that benefit infants and children, and the
potential to provide an option for patients with diseases that are
not addressable with current viral-based approaches; the potential
of Moderna’s mRNA platform and lipid nanoparticle delivery
technology to encode for missing or damaged proteins for a diverse
array of rare diseases; the potentially broad applicability of
Moderna’s platform technology; and the potential that preclinical
research programs may lead to additional Moderna development
candidates. In some cases, forward-looking statements can be
identified by terminology such as “will,” “may,” “should,”
“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: whether preclinical research programs will lead to
additional Moderna development candidates; whether preclinical
results will be predictive of future clinical study
results; whether mRNA-based treatments could be unsafe or
intolerable during preclinical and clinical studies; preclinical
and clinical development is lengthy and uncertain, especially for a
new class of medicines such as mRNA, and therefore our preclinical
programs may be delayed, terminated, or may never advance to the
clinic; no mRNA drug has been approved in this new potential class
of medicines, and may never be approved; mRNA drug development has
substantial preclinical, clinical and regulatory risks due to the
novel and unprecedented nature of this new class of medicines; and
those risks and uncertainties described under the heading “Risk
Factors” in Moderna’s most recent Annual Report on Form 10-K 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 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190429005133/en/
Investors:Lavina TalukdarHead of Investor
Relations617-209-5834lavina.talukdar@modernatx.comMedia:Jason
GlashowHead, Corporate
Communications617-674-5648Jason.glashow@modernatx.com
Moderna (NASDAQ:MRNA)
Historical Stock Chart
From Aug 2024 to Sep 2024
Moderna (NASDAQ:MRNA)
Historical Stock Chart
From Sep 2023 to Sep 2024