– Allowed Claims of Tuschl ’262 Application
Broadly Cover “Dicer Substrate” and Other RNAi Molecules, Including
Those Containing Nucleotide Analogs such as “Unlocked Nucleobase
Analogs (UNAs)” –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, announced today that the United States Patent
and Trademark Office (USPTO) has issued a Notice of Allowance for
claims in the Tuschl et al. patent application 13/725,262. The ’262
patent application includes newly allowed claims directed to
compositions that mediate RNAi, and comprising a double-stranded
molecule with up to 25 base pairs and at least one nucleotide
analogue. Specifically, the newly allowed patent application
broadly covers small interfering RNA (“siRNA”) molecules of various
designs, including so-called “dicer substrate” RNAi triggers
(Amarzguioui et al., Nat Protoc. 2006;1(2):508-17; Rose et al.,
Nucleic Acids Res. 2005 Jul 26;33(13):4140-56) and double-stranded,
RNAi-mediating molecules containing moieties that include “unlocked
nucleobase analogs” (“UNA”) amongst other naturally or
non-naturally occurring nucleotide analogues.
“This notice of allowance for the Tuschl ’262 patent application
recognizes the groundbreaking and fundamental work of Tom Tuschl
and colleagues regarding RNAi trigger molecules. Specifically, the
newly allowed claims broadly cover double-stranded molecules with
up to 25 base pairs that mediate RNAi, and thus include diverse
siRNA trigger approaches, such as dicer substrates or those
utilizing, for example, UNA modifications,” said Laurence Reid,
Ph.D., Senior Vice President and Chief Business Officer of Alnylam.
“We aim to continue to advance claims from the Tuschl patent family
and from other Alnylam-held patent families. Indeed, we are
committed to protecting the innovative discoveries of our founders,
advisors, and employees that laid the cornerstone for current and
future RNAi therapeutics.”
The allowed claims of the Tuschl ’262 patent application, as
well as other granted, Alnylam-owned or -licensed patents, are
provided on the company’s website, and in aggregate broadly cover
compositions, methods, and uses of RNAi therapeutics. Alnylam’s
intellectual property (IP) estate also includes patents that
broadly cover delivery of RNAi therapeutics, such as Alnylam’s
GalNAc-siRNA conjugate technology, and siRNAs directed toward a
wide range of disease targets.
Alnylam will be providing an update on their ALN-AAT program, an
RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the
treatment of liver disease associated with AAT deficiency, in an
online RNAi Roundtable to be held today at 12:30 p.m. ET and can be
accessed by clicking here.
About 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 as genetic
medicines, including programs as part of the company’s “Alnylam
5x15™” product strategy. Alnylam’s genetic medicine programs are
RNAi therapeutics directed toward genetically defined targets for
the treatment of serious, life-threatening diseases with limited
treatment options for patients and their caregivers. These include:
patisiran (ALN-TTR02), an intravenously delivered RNAi therapeutic
targeting transthyretin (TTR) for the treatment of TTR-mediated
amyloidosis (ATTR) in patients with familial amyloidotic
polyneuropathy (FAP); ALN-TTRsc, a subcutaneously delivered RNAi
therapeutic targeting TTR for the treatment of ATTR in patients
with TTR cardiac amyloidosis, including familial amyloidotic
cardiomyopathy (FAC) and senile systemic amyloidosis (SSA);
ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the
treatment of hemophilia and rare bleeding disorders (RBD); ALN-CC5,
an RNAi therapeutic targeting complement component C5 for the
treatment of complement-mediated diseases; ALN-AS1, an RNAi
therapeutic targeting aminolevulinic acid synthase-1 (ALAS-1) for
the treatment of hepatic porphyrias including acute intermittent
porphyria (AIP); ALN-PCS, an RNAi therapeutic targeting PCSK9 for
the treatment of hypercholesterolemia; ALN-AAT, an RNAi therapeutic
targeting alpha-1 antitrypsin (AAT) for the treatment of AAT
deficiency-associated liver disease; ALN-TMP, an RNAi therapeutic
targeting TMPRSS6 for the treatment of beta-thalassemia and
iron-overload disorders; ALN-ANG, an RNAi therapeutic targeting
angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of
mixed hyperlipidemia and severe hypertriglyceridemia; ALN-AC3, an
RNAi therapeutic targeting apolipoprotein C-III (apoCIII) for the
treatment of hypertriglyceridemia; and other programs yet to be
disclosed. As part of its “Alnylam 5x15” strategy, as updated in
early 2014, the company expects to have six to seven genetic
medicine product candidates in clinical development - including at
least two programs in Phase 3 and five to six programs with human
proof of concept - by the end of 2015. Alnylam is also developing
ALN-HBV, an RNAi therapeutic targeting the hepatitis B virus (HBV)
genome for the treatment of HBV infection. The company’s
demonstrated commitment to RNAi therapeutics has enabled it to form
major alliances with leading companies including Merck, Medtronic,
Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist,
GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and
Genzyme, a Sanofi company. In March 2014, Alnylam acquired Sirna
Therapeutics, a wholly owned subsidiary of Merck. In addition,
Alnylam holds an equity position in Regulus Therapeutics Inc., a
company focused on discovery, development, and commercialization of
microRNA therapeutics. Alnylam scientists and collaborators have
published their research on RNAi therapeutics in over 200
peer-reviewed papers, including many in the world’s top scientific
journals such as Nature, Nature Medicine, Nature Biotechnology,
Cell, New England Journal of Medicine, and The Lancet. 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 RNAi
therapeutics, its expectations regarding its “Alnylam 5x15” product
strategy, its plans regarding commercialization of RNAi
therapeutics, and its views with regard to the scope of its IP
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 manage operating expenses, Alnylam’s ability to discover
and develop novel drug candidates and delivery approaches,
successfully demonstrate the efficacy and safety of its drug
candidates, the pre-clinical and clinical results for its product
candidates, which may not support further development of product
candidates, actions of regulatory agencies, which may affect the
initiation, timing and progress of clinical trials, obtaining,
maintaining and protecting intellectual property, Alnylam’s ability
to enforce its patents against infringers and defend its patent
portfolio against challenges from third parties, obtaining
regulatory approval for products, competition from others using
technology similar to Alnylam’s and others developing products for
similar uses, Alnylam’s ability to obtain additional funding to
support its business activities and establish and maintain
strategic business alliances and new business initiatives,
Alnylam’s dependence on third parties for development, manufacture,
marketing, sales and distribution of products, the outcome of
litigation, and unexpected expenditures, as well as those risks
more fully discussed in the “Risk Factors” filed with Alnylam’s
most recent Quarterly Report on Form 10-Q filed with the Securities
and Exchange Commission (SEC) and in other filings that Alnylam
makes with the SEC. 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
explicitly disclaims any obligation to update any forward-looking
statements.
Alnylam Pharmaceuticals, Inc.Cynthia Clayton,
617-551-8207Vice President, Investor Relations andCorporate
Communicationsor(Media)Liz Bryan, 202-955-6222 x2526Spectrum
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