Alnylam Consolidates Intellectual Property for RNA Activation (RNAa), a New Biological...

Alnylam Consolidates Intellectual Property for RNA Activation (RNAa), a New Biological Discovery for Activation of Gene Expression

RNAa May Have Applications in Many Human Diseases, Including
Certain Genetic Disorders and Cancer

Company Completes Exclusive License Agreements with University of
Texas Southwestern Medical Center, University of California San
Francisco, and the Salk Institute for Biological Studies
CAMBRIDGE, Mass.--(Business Wire)--
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, today announced that it has consolidated the key
intellectual property (IP) for RNA activation (RNAa), creating a
dominant IP position in this new area of biology. RNAa is an emerging
biological discovery involving double-stranded RNAs that target
promoter regions in chromosomal DNA resulting in transcriptional
activation of genes. The transcriptional activation, or up-regulation,
of genes results in an increase in mRNA and protein production.
Accordingly, this technology may have applications in a range of human
disorders such as certain genetic diseases and cancer where the
aberrant low expression of certain proteins is known to occur. In
order to consolidate IP in this field, Alnylam has completed exclusive
license agreements with the leading academic institutions working in
this area: the Corey lab at the University of Texas Southwestern
Medical Center (UT Southwestern); the Li lab at the University of
California San Francisco (UCSF); and the Gage lab at the Salk
Institute for Biological Studies (Salk).

"Alnylam is committed to scientific and IP leadership across all
areas of modern biology's RNA revolution, including our ongoing
efforts in RNAi and microRNA therapeutics, and now RNAa," said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. "While there's
more to understand in this emerging biology, RNAa defines a new
application for double-stranded RNAs that could have the potential to
create an entirely new therapeutic platform for Alnylam. Many human
diseases are caused by the abnormally low expression of proteins, and
RNAa could be used to treat these disorders through selective
transcriptional gene activation. Obtaining exclusive access to these
key RNAa patents positions Alnylam to lead in the translational
research of this biology as it advances toward in vivo validation and
provides a solid foundation for business execution, as we've
demonstrated in the past with our RNAi and microRNA platforms."

"We're excited to be working with Alnylam, as they have
demonstrated a commitment to scientific excellence in their
publications and academic collaborations," said David Corey, Ph.D.,
Professor of Pharmacology and Biochemistry at UT Southwestern. "We
look forward to collaborating on the emerging science of RNAa biology
to further explore the breadth of applications for transcriptional
gene activation with agRNAs."

RNAa is mediated by double-stranded RNAs, possibly including
endogenous miRNAs that target the promoter regions of genes in
chromosomal DNA. So-called "anti-gene RNAs," or agRNAs, appear to
increase transcription by complementary base-pairing with
corresponding promoter sequences which enhance recruitment of the
cellular RNA polymerase II and therefore increase the target gene's
expression. In in vitro studies, agRNAs can increase the levels of a
target gene's mRNA by nearly 10-fold, resulting in an increased level
of the target gene's protein. The work by the Corey lab at UT
Southwestern for the activation of the progesterone receptor was
published last year (Janowski et al., Nature Chemical Biology 3,
166-173 (2007)) and a potential mechanism for RNAa is highlighted in a
more recent paper (Schwartz et al., Nature Structural & Molecular
Biology, doi:10.1038 / nsmb.1444(2008)). In addition, studies on RNAa
have been performed by the Dahiya and the Li labs at UCSF (Li et al.,
PNAS, 103, 17337-17342 (2006); Chen et al., Mol Cancer Ther 7, 698-703
(2008); and Place et al., PNAS, 105, 1608-1613 (2008)) with a range of
genes including VEGF, E-cadherin, and p21, as well as by the Gage lab
(Kuwabara et al., Cell, 116, 779-793 (2004)) at the Salk. While
additional work is necessary to understand the breadth of this
biology, its applications in vivo, and its potential as a
pharmacologic strategy, activation of gene expression with RNAa could
offer new approaches for innovative medicines.

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. RNAi therapeutics target the cause of
diseases by potently silencing specific messenger RNAs (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. In addition, the company is
developing RNAi therapeutics for the treatment of a wide range of
disease areas, including hypercholesterolemia, liver cancers, and
Huntington's disease. 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, and Kyowa Hakko Kogyo. To
reflect its outlook for key scientific, clinical, and business
initiatives, Alnylam has established "RNAi 2010" 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 LLC, 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, visit www.alnylam.com.

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including its views with respect to
the potential of RNAa, 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 risks related to: Alnylam's
approach to discover and develop novel drugs, which is unproven and
may never lead to marketable products; obtaining, maintaining and
protecting intellectual property; Alnylam's ability to enforce its
patents against infringers and to defend its patent portfolio against
challenges from third parties; Alnylam's ability to obtain additional
funding to support its business activities; Alnylam's ability to
realize future milestones and royalties as well as co-development and
co-commercialization opportunities; Alnylam's dependence on third
parties for development, manufacture, marketing, sales and
distribution of products; obtaining regulatory approval for products;
competition from others using technology similar to Alnylam's and
others developing products for similar uses; Alnylam's dependence on
collaborators; and Alnylam's short operating history; 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.

Alnylam Pharmaceuticals, Inc.
Investors:
Cynthia Clayton, 617-551-8207
or
Media:
Yates Public Relations
Kathryn Morris, 845-635-9828

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