Moderna to Present Preclinical Data at 2019 American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

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

CAMBRIDGE, Mass.–(BUSINESS WIRE)–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.

Contacts

Investors:
Lavina Talukdar
Head of Investor Relations
617-209-5834
[email protected]

Media:
Jason
Glashow
Head, Corporate Communications
617-674-5648
[email protected]

error: Content is protected !!