- Alnylam VaxiRNATM Platform Applies RNAi to
Enhance Vaccine Production for Influenza and Other Pathogens -
CAMBRIDGE, Mass., Nov 01, 2011 (BUSINESS WIRE) --
Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, announced today it has formed a collaboration with
GlaxoSmithKline (GSK) focused on Alnylam's VaxiRNATM
technology for certain GSK vaccine products, including influenza.
VaxiRNA is a new RNAi technology for the enhanced production of viruses
used in the manufacture of vaccine products. GSK, a leading vaccine
manufacturer, is the first company to form a collaboration accessing
this new platform.
"At Alnylam, we continue to pioneer innovative opportunities for our
RNAi platform that could have transformative impact in medicine and the
biopharmaceutical industry. This is exemplified with VaxiRNA, where
siRNA technology is being used to increase virus titers in the
production of vaccine products. Since production of vaccines can be
limiting for the scale and speed of global immunization against many
pathogens, including influenza, we believe that VaxiRNA can become an
enabling technology for the broader vaccine industry," said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. "As we advance
our VaxiRNA effort, we are excited to form a new collaboration with GSK,
a global leader and innovator in vaccine development and manufacturing.
With GSK, we aim to apply VaxiRNA technology to enhance influenza
vaccine production in cell culture, where Alnylam will receive research
funding, in addition to potential milestones and payments on unit sales
of commercialized vaccine product."
Alnylam's VaxiRNA platform applies siRNAs for the silencing of specific
genes that limit or prevent efficient growth of viruses in vaccine
manufacturing systems, including those in cell culture and also in eggs.
The VaxiRNA platform stems from work Alnylam has performed as part of
its Alnylam Biotherapeutics efforts, where RNAi technology has been
applied to improve the manufacture of biotherapeutic products, such as
recombinant proteins and monoclonal antibodies. This new platform
addresses the significant unmet commercial need for innovative
technologies that can improve the manufacture of vaccine products,
especially where vaccine production is a limiting factor for the scale
and speed of global immunization needs.
The GSK collaboration is focused initially on influenza vaccine
production in cell culture systems. Under the terms of the agreement,
GSK will provide funding and certain milestone payments to Alnylam. If
successfully applied in the manufacture of commercial product, Alnylam
will receive payments on unit product sales. In addition, GSK has
obtained an option for VaxiRNA applications toward two additional
vaccine products.
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 VaxiRNA(TM)
Alnylam is applying RNAi technology to improve the manufacturing
processes for vaccines in an effort called VaxiRNA. With VaxiRNA,
Alnylam is using siRNAs that silence specific genes in vaccine
production systems, such as cells or eggs, which limit or prevent the
efficient growth of viruses used in the manufacture of vaccine products.
New innovations in vaccine manufacturing are needed to enable the scale
and speed of global immunization for a number of pathogens. In 2011,
Alnylam formed a VaxiRNA partnership with GlaxoSmithKline for influenza
vaccine production.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics
based on RNA interference, or RNAi. The company is leading the
translation of RNAi as a new class of innovative medicines with a core
focus on RNAi therapeutics for the treatment of genetically defined
diseases, including ALN-TTR for the treatment of transthyretin-mediated
amyloidosis (ATTR), ALN-PCS for the treatment of severe
hypercholesterolemia, ALN-HPN for the treatment of refractory anemia,
and ALN-APC for the treatment of hemophilia. As part of its "Alnylam 5x15TM"
strategy, the company expects to have five RNAi therapeutic products for
genetically defined diseases in advanced stages of clinical development
by the end of 2015. Alnylam has additional partner-based programs in
clinical or development stages, including ALN-RSV01 for the treatment of
respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment
of liver cancers, and ALN-HTT for the treatment of Huntington's disease.
The company's leadership position on RNAi therapeutics and intellectual
property have enabled it to form major alliances with leading companies
including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa
Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded
Regulus Therapeutics Inc., a company focused on discovery, development,
and commercialization of microRNA therapeutics; Regulus has formed
partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed
Alnylam Biotherapeutics, a division of the company focused on the
development of RNAi technologies for application in biologics
manufacturing, including recombinant proteins and monoclonal antibodies.
Alnylam's VaxiRNA(TM) platform applies RNAi technology to improve the
manufacturing processes for vaccines; GlaxoSmithKline is a collaborator
in this effort. Alnylam scientists and collaborators have published
their research on RNAi therapeutics in over 100 peer-reviewed papers,
including many in the world's top scientific journals such as Nature,
Nature Medicine, Nature Biotechnology, and Cell.
Founded in 2002, Alnylam maintains headquarters in Cambridge,
Massachusetts. For more information, please visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including without limitation,
Alnylam's views with respect to the potential for VaxiRNA to apply RNAi
technology to improve the manufacturing process for vaccines, its
expectations regarding the receipt of research funding, milestone
payments and payments on unit product sales under its collaboration with
GlaxoSmithKline, as well as Alnylam's expectations with respect to its
"Alnylam 5x15" product strategy, 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, without limitation,
Alnylam's ability to successfully apply RNAi technology to improve the
manufacturing process for vaccines, including in connection with its GSK
collaboration, demonstrate the efficacy and safety of its drug
candidates in pre-clinical and human clinical trials, and establish and
maintain strategic business alliances and new business initiatives, 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.

SOURCE: Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Senior Director, Investor Relations and
Corporate Communications
or
Spectrum
Amanda Sellers (Media), 202-955-6222 x2597