Parkinson's Disease to be Targeted by Novel RNA Interference Drug Development

[Murray Charters <mcharters@xxxxxxxxxxxxxx>]

Parkinson's Disease to be Targeted by Novel RNA Interference Drug Development

CAMBRIDGE, Mass., and ROCHESTER, Minn., October 30, 2003 -Alnylam 
Pharmaceuticals, Inc., the leading therapeutic RNA
interference (RNAi) company, today announced the formation of a research 
collaboration to apply its proprietary RNAi-
based therapeutic technology to a discovery by researchers at Mayo Clinic and 
NIH of a causal pathway in Parkinson's
disease. Through the collaboration, Alnylam will provide RNAi technology and 
fund research at Mayo Clinic to develop a
drug that suppresses the expression of a specific gene, alpha-synuclein, found 
to be over-expressed in Parkinson's
patients. The findings are published in the current issue of Science.

Under the terms of the research collaboration, Alnylam will identify, 
synthesize and provide RNAi-based drug compounds
targeted to the alpha synuclein gene expression. Mayo Clinic will test and 
select the RNAi compounds for their efficacy
in a series of in vitro and in vivo studies.

"This collaboration with Alnylam creates the possibility of previously 
unimagined therapeutic advances for Parkinson's
patients," says Matthew Farrer, Ph.D., Assistant Professor of Neuroscience and 
the director of a neurogenetics
laboratory at Mayo Clinic in Jacksonville, Fla. "RNA interference is a 
promising technology that offers the potential
to halt progression of Parkinson's disease through a targeted mechanism of 
action." Demetrius Maraganore, M.D.,
Professor of Neurology at Mayo Clinic in Rochester, Minn., adds 
"Alpha-synuclein gene over-expression is the 'Rosetta
stone' of Parkinson's disease. We anticipate that therapy to reduce 
alpha-synuclein gene expression will benefit not
only Parkinson's disease patients who carry the rare gene mutation, but also 
persons who carry common susceptibility
variants of the gene, or persons who aggregate the alpha-synuclein protein via 
other genetic and non-genetic
mechanisms."

"The groundbreaking research of Mayo Clinic offers us an exciting platform to 
apply RNA interference to silence a
specific gene pathway in Parkinson's disease," says Thomas Ulich, M.D., Senior 
Vice President of Research and
Development at Alnylam. "For this collaboration we have targeted Parkinson's 
disease. This is one of many diseases in
which we plan to apply RNAi-based therapies, to create a new class of medicines 
for patients."

Alpha Synuclein Mechanism
Alpha synuclein is 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). The 
findings in Science highlight that simple
over-expression of normal, wild-type alpha-synuclein is sufficient to cause 
disease in a family with multiple affected
members. Previous work by the Mayo Clinic group, published in Human Molecular 
Genetics, demonstrated that over-
expression of wild-type alpha-synuclein may confer susceptibility in 
Parkinson's disease patients population-wide.

Parkinson's Disease
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.

People with Parkinson's often experience trembling, muscle rigidity, difficulty 
walking, and problems with balance and
coordination. These symptoms generally develop after age 50, although the 
disease affects a small percentage of younger
people as well.

About Alnylam
Alnylam is the leading company in the emerging field of RNA interference (RNAi) 
whose vision is to Harness a Revolution
in Biology for Human HealthTM. Growing from its foundation as the world's first 
company focused on RNAi-based
therapeutics, the company is built around the leading capabilities of its two 
operating units, Alnylam Pharmaceuticals
of Cambridge, Massachusetts, and Ribopharma of Kulmbach, Germany. Over the last 
several years, RNAi has been identified
as a fundamental cellular mechanism for gene silencing. 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-based therapeutics. The company's focus is to 
discover, develop, and commercialize
therapeutic products based on RNAi for a broad range of therapeutic indications 
including viral, oncologic, metabolic,
CNS and autoimmune diseases. The company's mission is to lead research in the 
field of RNAi, to build the leading
pipeline of RNAi-based therapeutics, and to emerge as business leaders. Its 
global headquarters are in Cambridge, MA.
Additional information is available at www.alnylam.com.

###

CONTACT
Barbara Yates
tel.: 781-862-1967
fax: 781-863-0933
email: yatesoffice@xxxxxxx

SOURCE: Atlas Venture


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