Alnylam Announces United States Patent and Trademark Office (USPTO) Issues Patent Covering RNAi Therapeutics for the Treatment of Hepatitis B Virus (HBV) Infection
Jun 12,2014
- New '277 McSwiggen Patent Includes Broad, Sequence-Independent Claims for Chemically Modified siRNAs Targeting the HBV Genome -
"We are pleased with the USPTO's decision to issue the '277 patent from
our McSwiggen patent family, a key component of the IP estate we
recently obtained through our acquisition of
The McSwiggen patent family includes 23 granted patents around the world, including patents in the U.S.1 and the EU2, and generally describes chemical modifications of siRNA. The '277 patent includes broad, sequence-independent issued claims on compositions of chemically modified siRNA targeting HBV, including:
- siRNA that mediates RNAi against a hepatitis B virus target mRNA wherein each strand is between 18 to 24 nucleotides; and
- sense and antisense strand each comprising 10 or more 2'-deoxy, 2-O-Me, 2'-F or universal base modified nucleotides and 10 or more pyrimidines of the sense and/or antisense strand are 2'-deoxy, 2-O-Me, 2'-F.
Such chemical modifications of siRNA are generally required to achieve in vivo potency and durability for RNAi therapeutics. Additional HBV-directed claims for the McSwiggen patent family are pending in filed patent applications.
Granted claims of the '277 patent, as well as other granted Alnylam owned or licensed patents, are provided on the company's website, and in aggregate broadly cover siRNA 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 those termed "unlocked nucleobase analogs." In addition, Alnylam's owned or licensed patents broadly cover delivery of RNAi therapeutics, including those that employ GalNAc-siRNA conjugate technology. Finally, Alnylam's IP estate also includes patents that broadly cover siRNA toward a wide range of disease targets.
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 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
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,
including ALN-HBV for the treatment of HBV infection, its expectations
with respect to the selection of a Development Candidate and the timing
of regulatory filings to initiate a clinical trial for ALN-HBV, its
expectations regarding its "Alnylam 5x15" product strategy, its plans
regarding commercialization of RNAi therapeutics, including ALN-HBV, 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
1 Issued U.S. patents in the McSwiggen patent family include U.S. Patent Nos: 7,923,547; 7,956,176; 7,989,612; 8,202,979; 8,232,383; 8,236,944; 8,242,257; 8,268,986; 8,273,866; 8,507,661; 8,618,277.
2 Granted EU patents in the McSwiggen patent family include EU Patent Nos: 1423406; 1458741; 1627061; 2278004; 2287306.
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President, Investor Relations and Corporate Communications
or
Media
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