Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, announced today its participation in a $2.4
million research grant awarded to the University of Pennsylvania under
the "American Recovery and Reinvestment Act of 2009”. The new grant will
fund research over two years in the laboratories of Daniel Rader, M.D.
of the University of Pennsylvania School of Medicine, Sekar Kathiresan,
M.D. of Massachusetts General Hospital (MGH) and Harvard Medical School,
and Alnylam.
The goal of this initiative is to mechanistically evaluate the metabolic
and molecular effects of 38 novel genes implicated by human genetic
studies in prevention of cardiovascular disease and to discover and
develop RNAi therapeutics towards these gene candidates. Through an
agreement with the University of Pennsylvania and MGH, Alnylam will
retain the rights to develop and commercialize RNAi therapeutics from
the research effort.
"We are excited to participate in this important work led by Dr. Dan
Rader of the University of Pennsylvania, who is widely recognized for
his outstanding work in lipid research and lipoprotein metabolism,” said
Victor Kotelianski, M.D., Ph.D., D.Sc., Senior Vice President,
Distinguished Alnylam Fellow. "We are also pleased to be collaborating
in this research effort with Dr. Kathiresan of MGH, where we will be
able to interrogate the findings of large-scale genome-wide association
studies in cardiovascular disease, including genes associated with
abnormal blood lipid levels and early onset myocardial infarction. As
evidenced by our RNAi therapeutic program targeting PCSK9,
cardiovascular disease remains an area of great interest for Alnylam as
we believe there is significant potential for an RNAi therapeutic to
make an impact in the lives of patients.”
"We are honored to be awarded this new grant as part of the NIH
stimulus,” said Rader, the Cooper-McClure Professor of Medicine at the
University of Pennsylvania School of Medicine. "Together with my team at
the University of Pennsylvania and my collaborators at the Massachusetts
General Hospital and the Broad Institute, I am looking forward to
working with Alnylam as leaders in the RNAi therapeutics field. Our
knowledge of lipid disorders and their association with human disease
combined with Alnylam’s expertise of development of RNAi therapeutics
represents a powerful and rare opportunity to advance new innovative
medicines.”
The work conducted under this grant intends to validate novel genes as
targets for new therapies for lipid disorders and heart disease and will
establish early proof of concept on how genetic association studies can
yield important new insights into biology that have the potential to
impact cardiovascular disease. Coronary heart disease is the leading
cause of mortality in both men and women worldwide and its incidence is
highly correlated with levels of cholesterol and triglycerides in the
blood. Plasma concentrations of low-density lipoprotein (LDL, or "bad”
cholesterol) and high-density lipoprotein (HDL, or "good” cholesterol),
as well as triglycerides, have a strong inherited basis and recent
genetic association studies have linked previously unsuspected genes to
these complex traits.
The project described was supported by Award Number RC2HL101864 from the
National Heart, Lung, And Blood Institute. The content is solely the
responsibility of the authors and does not necessarily represent the
official views of the National Heart, Lung, And Blood Institute or the
National Institutes of Health.
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing a
breakthrough in understanding how genes are turned on and off in cells,
and a completely new approach to drug discovery and development. Its
discovery has been heralded as "a major scientific breakthrough that
happens once every decade or so,” and represents one of the most
promising and rapidly advancing frontiers in biology and drug discovery
today which was awarded the 2006 Nobel Prize for Physiology or Medicine.
RNAi is a natural process of gene silencing that occurs in organisms
ranging from plants to mammals. By harnessing the natural biological
process of RNAi occurring in our cells, the creation of a major new
class of medicines, known as RNAi therapeutics, is on the horizon. Small
interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise
Alnylam’s RNAi therapeutic platform, target the cause of diseases by
potently silencing specific mRNAs, thereby preventing disease-causing
proteins from being made. RNAi therapeutics have the potential to treat
disease and help patients in a fundamentally new way.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics
based on RNA interference, or RNAi. The company is applying its
therapeutic expertise in RNAi to address significant medical needs, many
of which cannot effectively be addressed with small molecules or
antibodies, the current major classes of drugs. Alnylam is leading the
translation of RNAi as a new class of innovative medicines with
peer-reviewed research efforts published in the world’s top scientific
journals including Nature, Nature Medicine, and Cell.
The company is leveraging these capabilities to build a broad pipeline
of RNAi therapeutics; its most advanced program is in Phase II human
clinical trials for the treatment of respiratory syncytial virus (RSV)
infection and is partnered with Cubist and Kyowa Hakko. In addition, the
company is developing RNAi therapeutics for the treatment of a wide
range of disease areas, including liver cancers, hypercholesterolemia,
Huntington’s disease, and TTR amyloidosis. The company’s leadership
position in fundamental patents, technology, and know-how relating to
RNAi has enabled it to form major alliances with leading companies
including Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko,
and Cubist. To reflect its outlook for key scientific, clinical, and
business initiatives, Alnylam established "RNAi 2010” in January
2008 which includes the company’s plan to significantly expand the scope
of delivery solutions for RNAi therapeutics, have four or more programs
in clinical development, and to form four or more new major business
collaborations, all by the end of 2010. Alnylam is a joint owner of
Regulus Therapeutics, a joint venture focused on the discovery,
development, and commercialization of microRNA therapeutics. Founded in
2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For
more information, please visit http://www.alnylam.com.
Alnylam Forward-Looking Statement
Various statements in this release concerning Alnylam’s future
expectations, plans and prospects, constitute forward-looking statements
for the purposes of the safe harbor provisions under The Private
Securities Litigation Reform Act of 1995. Actual results may differ
materially from those indicated by these forward-looking statements as a
result of various important factors, including the company's ability to
successfully research and develop products and to successfully prosecute
and enforce its patents around the world, as well as those risks more
fully discussed in the "Risk Factors” section of its most recent
quarterly report on Form 10-Q on file with the Securities and Exchange
Commission. In addition, any forward-looking statements represent
Alnylam’s views only as of today and should not be relied upon as
representing its views as of any subsequent date. Alnylam does not
assume any obligation to update any forward-looking statements.
