Alnylam Receives Notice of Allowance from United States Patent and Trademark Office (USPTO) for New Patents from the Tuschl II

  Alnylam Receives Notice of Allowance from United States Patent and Trademark
  Office (USPTO) for New Patents from the Tuschl II Patent Estate Broadly
  Covering RNAi Therapeutics

 – New Composition of Matter and Method of Use Claims Allowed in Three Patent
                                Applications –

Business Wire

CAMBRIDGE, Mass. -- November 26, 2012

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, announced today that the United States Patent and Trademark Office
(USPTO) has issued Notices of Allowance for three patent applications from the
company’s exclusively held Tuschl II patent estate. The USPTO has allowed
claims in patent applications 12/537,602, 12/683,081 and 12/838,786 which
cover compositions, methods, and uses of small interfering RNA (siRNA) that
are important for the development and commercialization of RNAi therapeutics.
These newly allowed claims are the first to emerge from the U.S. prosecution
of the Tuschl II patent series that include composition of matter for siRNA,
the molecules that mediate RNAi. The Tuschl II patent family is exclusively
licensed to Alnylam for RNAi therapeutics on a worldwide basis through an
agreement with Max Planck Innovation GmbH, the licensing agent for the Max
Planck Society.

“We are very pleased with continued advancement of our Tuschl II patent estate
through the USPTO, which further extends Alnylam’s leadership on intellectual
property for RNAi therapeutics in the world’s largest pharmaceutical market,”
said Laurence Reid, Ph.D., Senior Vice President, Chief Business Officer at
Alnylam. “We believe that our unparalleled intellectual property estate will
protect the innovative medicines we advance to the market as part of our
‘Alnylam 5x15’ product strategy. In the near term, Alnylam continues to
realize value from this estate through our partnering efforts.”

“We are gratified that the USPTO has decided to allow these new composition of
matter, method, and use claims from the Tuschl II patent estate,” said Dr.
Joern Erselius, Managing Director, Max Planck Innovation GmbH. “The continued
successful worldwide examination of the Tuschl II patent series highlights
that this invention is seminal for RNAi, demonstrating the importance of the
work performed by Professor Tuschl and other co-inventors at the Max Planck

Patent applications 12/537,602, 12/683,081 and 12/838,786 include claims
broadly covering compositions, methods, and uses for double-stranded RNAs
having key structural elements that are widely recognized as important for the
therapeutic activity of siRNA, including:

  *two RNA strands with a length of 19-23 or 19-25 nucleotides;
  *at least one strand forms a single-stranded 3’ overhang from 1-3 or 1-5
  *with or without chemical modifications; and
  *the double-stranded RNA molecule cleaves a target mRNA at a single site.

The prosecution of these cases has been handled by the law firm of Lando &
Anastasi, LLP.

Alnylam’s intellectual property position is comprised of fundamental,
chemistry, delivery, and target patents and patent applications that the
company believes are necessary for the development and commercialization of
RNAi therapeutics. In aggregate, Alnylam owns or has in-licensed over 1,800
active patent cases, of which over 700 have issued or been granted worldwide,
and over 300 have issued or been granted in the U.S., Europe, or Japan, the
world’s largest pharmaceutical markets.

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 RNA (siRNA), 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 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-AT3 for the
treatment of hemophilia, ALN-PCS for the treatment of severe
hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and
ALN-TMP for the treatment of hemoglobinopathies. As part of its “Alnylam
5x15^TM” strategy, the company expects to have five RNAi therapeutic products
for genetically defined diseases in clinical development, including programs
in advanced stages, on its own or with a partner by the end of 2015. Alnylam
has additional partnered 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, Cubist, Ascletis, Monsanto, and Genzyme. 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, Sanofi, AstraZeneca and
Biogen Idec. Alnylam has also formed Alnylam Biotherapeutics, a division of
the company focused on the development of RNAi technologies for applications
in biologics manufacturing, including recombinant proteins and monoclonal
antibodies. Alnylam’s VaxiRNA™ 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

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam’s future expectations,
plans and prospects, including, without limitation, statements regarding
Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy,
and Alnylam’s views with regard to the strength, enforceability, and validity
of its intellectual property estate, 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 enforce its
patents against infringers and defend its patent portfolio against challenges
from third parties, 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.
Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and
Corporate Communications
Amanda Sellers, 202-955-6222 x2597
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