– Allowed Claims of Manoharan ’478 Application
Cover GalNAc-Conjugates Independent of Length, Sequence, and
Disease Target –
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 Manoharan et al. patent application 13/693,478. The
’478 patent application includes newly allowed claims directed to
compositions including those comprising a modified RNA agent linked
to a biantennary or triantennary ligand. Specifically, the allowed
application includes claims that broadly cover single-stranded or
double-stranded, chemically modified RNA therapeutics conjugated
with an N-acetylgalactosamine (GalNAc) ligand independent of
length, sequence, or disease target. The allowance of this patent
application represents a major advance in Alnylam’s intellectual
property (IP) estate, since GalNAc-siRNA conjugates enable potent
and durable silencing of liver-expressed disease genes with
subcutaneous dose administration and a wide therapeutic index.
Importantly, this delivery approach is a key component of the
company’s execution on its “Alnylam 5x15” and broader genetic
medicine product strategy.
“These newly allowed claims from the Manoharan ’478 patent
recognize the pioneering work of Alnylam scientists in the
advancement of conjugates as a delivery platform for RNA
therapeutics. We are gratified that the USPTO has acknowledged
these innovations by allowance of broad claims from this patent
application,” said Laurence Reid, Ph.D., Senior Vice President and
Chief Business Officer of Alnylam. “Our conjugate delivery platform
- specifically utilizing GalNAc-siRNA conjugates - enables
subcutaneous dosing of RNAi therapeutics with potent and durable
effects and a wide therapeutic index. With our Enhanced
Stabilization Chemistry (ESC) conjugates, we believe that
once-monthly and possibly once-quarterly subcutaneous dosing
regimens can be achieved in our clinical pipeline programs. Based
on this progress, we believe that GalNAc-conjugates are now the
industry-leading delivery approach for RNA therapeutics targeting
liver-expressed genes, and this patent allowance will reinforce
Alnylam’s central role in value creation based on therapeutics
linked to this technology.”
The allowed claims of the Manoharan ’478 patent application, as
well as other granted, Alnylam-owned or -licensed patents, are
provided on the company’s website, and in aggregate broadly cover
delivery of RNAi therapeutics, including those that employ
GalNAc-siRNA conjugate technology; additional claims from this and
other patent families are pending. In addition, Alnylam’s owned or
licensed patents broadly cover siRNAs and their use in a wide range
of lengths from 15 to 49 nucleotides, and chemical modifications
with naturally or non-naturally occurring nucleotides, including,
for example, acyclic nucleotides such as “unlocked nucleoside
analogs.” Alnylam’s IP estate also includes patents that broadly
cover siRNAs toward a wide range of disease targets.
Alnylam will be providing an update on advances in the delivery
of RNAi therapeutics with ESC-GalNAc-siRNA conjugates in an RNAi
Roundtable webinar to be held at 11:00 a.m. ET today, and can be
accessed by clicking here.
About RNAiRNAi (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 PharmaceuticalsAlnylam 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 5x15TM" 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, the 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 StatementsVarious 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 and Corporate
CommunicationsorFor Media:SpectrumLiz Bryan, 202-955-6222 x2526
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