Alnylam and MIT Collaborators Discover Novel “Core-Shell” Nanoparticles for Systemic Delivery of RNAi Therapeutics
July 25 2011 - 8:00AM
Business Wire
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, together with collaborators at the
Massachusetts Institute of Technology (MIT), announced today the
publication of new paper describing discovery of “core-shell”
nanoparticles for systemic delivery of RNAi therapeutics. The
core-shell nanoparticles were generated using a high-throughput
polymer synthesis strategy and screened for intracellular delivery
applications including siRNA delivery. These findings, published in
the Proceedings of the National Academy of Sciences (Siegwart et
al., PNAS, 2011, doi: 10.1073/pnas.1106379108) allow for the
development of novel nanoparticles that have optimal chemical and
physical properties for effective intracellular delivery of RNAi
therapeutics.
“Continued progress in delivery of RNAi therapeutics requires
broad-based efforts around novel lipids, conjugates, and polymers.
In the current study, core-shell nanoparticles were discovered
using combinatorial approaches to identify novel materials for
siRNA delivery,” said Kevin Fitzgerald, Ph.D., Senior Director of
Research at Alnylam. “These findings further expand our systemic
delivery platform to achieve the broadest applications of RNAi
therapeutics.”
“Together with our collaborators at Alnylam, we continue to make
exciting progress on delivery of RNAi therapeutics,” said Dan
Anderson, Ph.D., of the David H. Koch Institute for Integrative
Research at MIT. “Importantly, these new data on core-shell
nanoparticles highlight non-lipid approaches for siRNA delivery
with opportunities for further optimization.”
Specifically, this study evaluated a library of over 1,500
chemically diverse nanoparticles as drug delivery vehicles, with
precise control over particle size, chemical composition and
architecture. The physical and chemical properties of materials can
have controlling effects on their utility as nanotherapeutics and
the findings revealed that certain chemical functionalities may be
advantageous for polymer-based delivery. Initial in vivo studies on
one of these novel nanoparticles showed silencing of
hepatocyte-specific Factor VII in a pre-clinical model. The ability
to control and modify the chemical nature of the core and shell of
the nanoparticle may afford utility of these materials in a wide
range of drug delivery applications.
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 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-PCS for
the treatment of severe hypercholesterolemia, and ALN-HPN for the
treatment of refractory anemia. As part of its “Alnylam 5x15TM”
strategy, the company expects to have five RNAi therapeutic
products for genetically defined diseases in advanced stages of
clinical development 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, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko
Kirin, and Cubist. 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 and Sanofi. Alnylam has also
formed Alnylam Biotherapeutics, a division of the company focused
on the development of RNAi technologies for application in
biologics manufacturing, including recombinant proteins and
monoclonal antibodies. 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
www.alnylam.com.
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 respect
to the potential for systemic delivery of RNAi therapeutics using
“core-shell” nanoparticles 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
delivery approaches and novel drug candidates, successfully
demonstrate the efficacy and safety of its drug candidates in human
clinical trials and establish and maintain strategic business
alliances and new business initiatives, as well as those risks more
fully discussed in the “Risk Factors” section of its most recent
annual 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.
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