| Alnylam Receives Notice of Allowance from United States Patent and Trademark Office (USPTO) for New Patent Broadly Covering Second Generation Lipid Nanoparticles (LNP) for Systemic Delivery of RNAi Therapeutics |
– New Patent Allowance Covers Seminal Invention of “MC3” Lipid –
“We are very pleased with the USPTO’s allowance of claims for this
important patent related to our second generation LNPs, thus
acknowledging the novelty of the MC3 lipid for systemic delivery of RNAi
therapeutics,” said
The newly allowed patent application includes 30 claims covering
composition of matter and formulations of MC3, as well as methods of
using these compositions and formulations. The patent application lists
as inventors 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 RNAs (siRNAs), 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 for the treatment of genetically defined
diseases, including ALN-TTR for the treatment of transthyretin-mediated
amyloidosis (ATTR), ALN-PCS for the treatment of severe
hypercholesterolemia, ALN-HPN for the treatment of refractory anemia,
ALN-APC for the treatment of hemophilia, and ALN-TMP for the treatment
of hemoglobinopathies. As part of its “Alnylam 5x15TM”
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 partner-based 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, 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
second-generation LNP delivery platform and its “Alnylam 5x15” product
strategy, 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 discover
and develop novel drug candidates and delivery approaches, successfully
demonstrate the efficacy and safety of its drug candidates, including
ALN-PCS and ALN-TTR02, the pre-clinical and clinical results for its
product candidates, including ALN-PCS and ALN-TTR02, 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,
including its patents covering MC3, 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 access to
intellectual property, and the development, manufacture, marketing,
sales and distribution of products, and the outcome of litigation, 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
Source:
Alnylam Pharmaceuticals, Inc. |