Nov 12, 2009 Press Release for Alnylam
Alnylam Provides Update on RNAi Pipeline, Platform, and Technology at Its R&D Day
Nov 12, 2009
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 12, 2009--
“We are pleased to share the exciting progress we’ve made in harnessing
RNAi to develop novel medicines,” said
ALN-TTR Program
Alnylam is developing ALN-TTR, a systemically delivered RNAi therapeutic targeting the TTR gene for the treatment of ATTR, including familial amyloidotic cardiomyopathy (FAC) and familial amyloidotic polyneuropathy (FAP). ATTR is caused by mutations in the TTR gene, which is expressed in the liver, that result in the accumulation of toxic deposits of the mutant TTR protein in several tissues, including nerves, heart, and the gastrointestinal tract. There are more than 100 mutations that have been identified in the TTR gene; ALN-TTR targets a region of the gene common to wild-type and all known mutant forms of TTR, and therefore, has potential as a therapeutic for all patients with FAC and FAP.
New pre-clinical data demonstrate that ALN-TTR administration is
associated with markedly reduced pathogenic deposition of mutant TTR in
tissues. The new studies were performed in a transgenic mouse model
where the human V30M mutated TTR is over-expressed, and were conducted
by Alnylam scientists in collaboration with Dr.
Alnylam expects to file regulatory applications for ALN-TTR by the end
of 2009 with a goal of initiating a Phase I clinical trial in early
2010. ALN-TTR is being advanced using stable nucleic acid-lipid
particles (SNALP) delivery technology in collaboration with
“Alnylam is advancing an important innovation in medicine to patients in need of new therapies. The progress we are making is reflected across our pipeline efforts, including our recent decision to advance ALN-RSV01 in a Phase IIb trial of adult lung transplant patients infected with RSV,” said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research at Alnylam. “In addition, new data from our TTR program provide a clear validation of our RNAi therapeutics strategy in ATTR, where we block the production of TTR protein and its pathogenic deposition in tissues, thereby creating the potential to attenuate the cause of this devastating genetic disease. We certainly look forward to advancing this important program to patients.”
Delivery
Alnylam also presented new data from its delivery research efforts
showing the discovery of novel lipid materials that can be incorporated
into lipid nanoparticles (LNPs) to achieve markedly improved in vivo
potency for gene silencing with systemically delivered RNAi therapeutic.
The improved potency of LNPs provides the opportunity to widen
therapeutic index, decrease cost of goods, and broaden the number of
tissues and cell types available for systemic RNAi. Alnylam showed new
data on three distinct LNPs that derive from novel lipids discovered as
part of the company’s collaborations with Tekmira,
Alnylam Biotherapeutics
Alnylam is also presenting today new data regarding the application of
RNAi technology to improve the manufacturing processes for biologics, an
approach that has the potential to create new business opportunities and
which the company is advancing in an internal effort called “Alnylam
Biotherapeutics.” This initiative is focused on applying RNAi
technologies to transform the
In particular, Alnylam is advancing RNAi technologies to improve the quantity and quality of biologics manufacturing processes using mammalian cell culture, such as Chinese hamster ovary cells, or “CHO” cells. This RNAi technology can be applied to the improvement of manufacturing processes for existing marketed drugs, new drugs in development, and for the emerging biosimilars market. Alnylam has developed proprietary delivery lipids that enable the efficient transfection of siRNAs into CHO cells when grown in suspension culture. Studies have demonstrated that silencing certain target genes involved in certain CHO cell apoptotic and metabolic pathways resulted in 40 to 60% improved cell viability as compared with untreated cells.
As Alnylam Biotherapeutics advances the technology, it plans to seek partnerships with established biologic manufacturers, selling licenses, products, and services. Alnylam Biotherapeutics is comprised of a focused team of Alnylam employees and the company plans to retain complete ownership of this effort at the present time.
Alnylam’s R&D Day is being held on
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 applying its
therapeutic expertise in RNAi to address significant medical needs, many
of which cannot effectively be addressed with small molecules or
antibodies, the current major classes of drugs. Alnylam is leading the
translation of RNAi as a new class of innovative medicines with
peer-reviewed research efforts published in the world’s top scientific
journals including Nature, Nature Medicine, and Cell.
The company is leveraging these capabilities to build a broad pipeline
of RNAi therapeutics; its most advanced program is in Phase II human
clinical trials for the treatment of respiratory syncytial virus (RSV)
infection and is partnered with Cubist and
Alnylam Forward-Looking Statement
Various statements in this release concerning Alnylam’s future
expectations, plans and prospects, 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 the company's ability to
successfully research and develop products and to successfully prosecute
and enforce its patents around the world, 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.
Investors:
Cynthia
Clayton, 617-551-8207
or
Media:
Yates Public Relations
Kathryn
Morris, 845-635-9828