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Alnylam Pharmaceuticals and the Ludwig Maximilian University Munich Publish Data on Short Interfering RNAs and Immune Stimulation Mechanisms.


CAMBRIDGE, Mass. -- Scientific Insights Published in Nature Medicine Advance the Development of RNAi Therapeutics

Alnylam Pharmaceuticals Alnylam Pharmaceuticals Inc. (NYSE: ALNY), a biopharmaceutical company, engages in the development and commercialization of therapeutic products based on RNA interference (RNAi) in the United States. , Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and its collaborators at the Ludwig Maximilian University Munich announced today the online publication of an article in Nature Medicine identifying specific immunostimulatory motifs of short interfering RNAs (siRNAs), the molecules that induce RNA interference RNA interference
n.
A process in which the introduction of double-stranded RNA into a cell inhibits the expression of genes.
. The identification of these motifs enables the design of siRNAs that avoid interferon induction, an important goal for achieving safe RNAi therapeutics. These data have applications for deciphering the biological mechanisms of gene silencing and understanding how to design and optimize siRNA sequences that are both safe and efficacious as therapeutics.

In the paper, immune stimulation, as measured by interferon induction, was found to occur in association with some siRNA sequences but not others. These findings were observed in vivo in vivo /in vi·vo/ (ve´vo) [L.] within the living body.

in vi·vo
adj.
Within a living organism.



in vivo adv.
 in association with the use of liposomal delivery of the siRNAs. In connection with the Nature Medicine publication, Alnylam has licensed certain intellectual property relating to relating to relate prepconcernant

relating to relate prepbezüglich +gen, mit Bezug auf +acc 
 Dr. Gunther Hartmann's work from the Ludwig Maximilian University Munich.

"Understanding the sequence-specific regulation of the immune response immune response
n.
An integrated bodily response to an antigen, especially one mediated by lymphocytes and involving recognition of antigens by specific antibodies or previously sensitized lymphocytes.
 toward short interfering RNA RNA: see nucleic acid.
RNA
 in full ribonucleic acid

One of the two main types of nucleic acid (the other being DNA), which functions in cellular protein synthesis in all living cells and replaces DNA as the carrier of genetic
 is a tremendous advancement in RNAi biology and the development of therapeutics based on RNAi," said Dr. Hartmann, M.D., Division of Clinical Pharmacology Clinical pharmacology is the science of drugs and their clinical use. It is underpinned by the basic science of pharmacology, with added focus on the application of pharmacological principles and methods in the real world. , Ludwig Maximilian University Munich Hospital, Germany.

"Alnylam is committed to developing safe and efficacious RNAi therapeutics," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam Pharmaceuticals. "Elucidating the mechanism of action responsible for siRNA immunostimulation will allow for efficient selection of appropriate siRNA molecules."

About RNA Interference (RNAi)

RNA interference, or RNAi, is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes. 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 10 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 oncology, metabolic, and autoimmune diseases Autoimmune diseases
A group of diseases, like rheumatoid arthritis and systemic lupus erythematosus, in which immune cells turn on the body, attacking various tissues and organs.

Mentioned in: Complement Deficiencies, Premature Menopause
. The company's global headquarters are in Cambridge, Massachusetts This article is about the city of Cambridge in Massachusetts. For the English university town, see Cambridge, England. For other places, see Cambridge (disambiguation).
Cambridge, Massachusetts is a city in the Greater Boston area of Massachusetts, United States.
. For additional information, please visit www.alnylam.com.

Alnylam Forward Looking Statement

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 Safe Harbor

1. A legal provision to reduce or eliminate liability as long as good faith is demonstrated.

2. A form of shark repellent implemented by a target company acquiring a business that is so poorly regulated that the target itself is less attractive.
 provisions under The Private Securities Litigation Reform Act The Private Securities Litigation Reform Act of 1995 (PSLRA) implemented several significant substantive changes affecting certain cases brought under the federal securities laws, including changes related to pleading, discovery, liability, class representation and awards fees and  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 most recent Form 10-Q Form 10-Q

See 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.
COPYRIGHT 2005 Business Wire
No portion of this article can be reproduced without the express written permission from the copyright holder.
Copyright 2005, Gale Group. All rights reserved. Gale Group is a Thomson Corporation Company.

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Publication:Business Wire
Date:Feb 22, 2005
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