Source: PR Newswire
http://www.prnewswire.com/cgi-bin/stories.pl?ACCT=109&STORY=/www/story/10
-27-2004/0002313415&EDATE=
Alnylam Pharmaceuticals Presents Results Showing Reduction Of
Alpha-Synuclein Expression By RNA Interference
Data Reported At Neuroscience Meeting Show Continuing Progress In
Preclinical
Program In Parkinson's Disease With Mayo Clinic
CAMBRIDGE, Mass., Oct. 27 /PRNewswire-FirstCall/-- Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading therapeutic RNA
interference
(RNAi) company, together with its collaborators at the Mayo Clinic, today
presented data from cell culture models showing RNA interference (RNAi)-
mediated reduction in the expression of alpha-synuclein, a protein whose
overexpression is linked to Parkinson's disease, at Neuroscience 2004,
the
Society for Neuroscience's 34th Annual Meeting in San Diego, California.
The
preclinical results showed that the use of small interfering RNAs
(siRNAs) to
target alpha-synuclein gene expression offers hope for the development of
RNAi
therapeutics to treat Parkinson's disease. The data presented at this
meeting
were developed by an Alnylam team led by David Bumcrot, Ph.D., Director
of
Preclinical Research at Alnylam, working collaboratively with the Mayo
Clinic
research laboratories of Matthew Farrer, Ph.D., and Jada Lewis, Ph.D.
In several in vitro studies, select siRNA molecules were shown to
specifically target alpha-synuclein expression, demonstrating effective
and
sustained reduction of this expression. Further, siRNA molecules were
stable,
with a long half-life, in both serum and brain extracts, and chemical
modifications that increase siRNA stability were successfully developed
and
tested. These siRNA molecules are now the subject of evaluation in
animal
models. Mayo Clinic and Alnylam established a collaboration to target
Parkinson's disease with RNAi therapeutics in October 2003. As part of
this
collaboration, researchers at Alnylam developed specific siRNA molecules
targeted to alpha-synuclein gene expression and scientists at Mayo Clinic
tested these custom siRNAs for efficacy.
"Through our collaboration with Mayo Clinic, we have made important
progress in demonstrating how RNAi may be harnessed to treat Parkinson's
disease by blocking a recently-identified mechanism underlying the
disease"
said Vincent Miles, Ph.D., Senior Vice President, Business Development of
Alnylam Pharmaceuticals. "Our encouraging results highlight the
significant
potential of RNAi to create a new class of medicines for important
diseases,
such as Parkinson's and other diseases of the central nervous system."
Alpha-Synuclein In Parkinson's Disease
Alpha-synuclein is a protein found in various body tissues, primarily
in
the brain, where scientists believe it may play a role in synaptic
vesicle
recycling (how nerve cells transmit their signals). Over the last few
years,
it has been found that mutations in alpha-synuclein appear to cause
Parkinson's Disease in a subset of patients. Findings published by Mayo
Clinic researchers in Fall 2003 in Science highlighted that, in a family
with
multiple affected members, simple over-expression of normal
alpha-synuclein
appeared sufficient to cause Parkinson's disease. Previous work by the
Mayo
Clinic group, published in Human Molecular Genetics, demonstrated that
over-
expression of alpha-synuclein may confer susceptibility to Parkinson's
disease
in a broader population.
Parkinson's disease is a serious disorder that affects nerve cells
(neurons) in the part of the brain controlling muscle movement. Nearly
one
million Americans currently live with Parkinson's and approximately
50,000
more receive a diagnosis of the disease every year.
About RNAi
RNA interference, or RNAi, is a naturally occurring mechanism within
cells
for selectively silencing and regulating specific genes that is
potentially
the basis for a new class of therapeutic products. Since many diseases
are
caused by the inappropriate activity of specific genes, the ability to
silence
and regulate such genes selectively through RNAi could provide a means to
treat a wide range of human diseases. The discovery of RNAi has been
heralded
by many as a major breakthrough, and the journal Science named RNAi the
top
scientific achievement of 2002, as well as one of the top ten scientific
advances of 2003.
About Alnylam
Alnylam is a biopharmaceutical company seeking to develop and
commercialize novel therapeutics based on RNA interference, or RNAi.
Growing
from its foundation as the world's first company focused on RNAi
therapeutics,
the company's leadership in the field of RNAi is supported by its
preeminent
founders and advisors and its strengths in fundamental patents,
technology,
and know-how that underlie the commercialization of RNAi therapeutics.
Alnylam
is developing a pipeline of RNAi products using Direct RNAi(TM) to treat
ocular, central nervous system, and respiratory diseases and Systemic
RNAi(TM)
to treat a broad range of diseases, including oncologic, metabolic, and
autoimmune diseases. The company's global headquarters are in Cambridge,
Massachusetts. For additional information, please visit
http://www.alnylam.com.
Forward-Looking Statements
Various statements in this release concerning our future
expectations,
plans, prospects and future operating results 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 risks related to: our
approach
to discover and develop novel drugs, which is unproven and may never lead
to
marketable products; our ability to obtain additional funding to support
our
business activities; our dependence on third parties for development,
manufacture, marketing, sales and distribution of our products; the
successful
development of products, all of which are in early stages of development;
obtaining regulatory approval for products; competition from others using
technology similar to ours and others developing products for similar
uses;
obtaining, maintaining and protecting intellectual property utilized by
our
products; and our short operating history; as well as those risks more
fully
discussed in the "Certain Factors That May Affect Future Results" section
of
our Form 10-Q filed with the Securities and Exchange Commission. In
addition,
any forward-looking statements represent our views only as of today and
should
not be relied upon as representing our views as of any subsequent date.
We do
not assume any obligation to update any forward-looking statements.
Contacts: Alnylam Pharmaceuticals, Inc.
Investor Contact: Media Contact:
Lilian Stern Kathryn Morris
Stern Investor Relations, Inc. KMorrisPR
212-362-1200 845-635-9828
SOURCE Alnylam Pharmaceuticals, Inc.
Web Site: http://www.alnylam.com
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