From rxpgnews.com
ALN-RSV01 Significantly Inhibits RSV Replication In Vivo
By Akanksha, Pharmacology Correspondent
Mar 20, 2005, 11:35
Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, has announced the presentation of in vivo efficacy data on its lead candidate RNAi therapeutic for the treatment of respiratory syncytial virus (RSV) infection at The Annual Meeting of the American Academy of Allergy, Asthma and Immunology (AAAAI) in San Antonio, Texas.
Experiments performed by researchers at Alnylam and collaborators from the University of Georgia demonstrated that a single low dose of Alnylam's lead RSV candidate, ALN-RSV01, an optimized siRNA, inhibits RSV replication both as a prophylactic and as a treatment when administered by intranasal delivery in a liquid form.
Alnylam collaborator Dr. Ralph Tripp, Ph.D., Department of Infectious Diseases, University of Georgia presented the results.
"There is a significant need for novel therapies to effectively treat patients infected with RSV, a highly contagious virus that can lead to serious respiratory illnesses and hospitalization in vulnerable patient populations," said Dr. Tripp. "Our research with Alnylam bolsters the potential for RNAi therapeutics to treat patients with RSV infections, who until now, have had few or no therapeutic options."
ALN-RSV01 specifically silences an RSV gene required for viral replication. Pre-clinical studies performed in mice demonstrated that a single low dose of ALN-RSV01 (2 mg/kg) protected against subsequent RSV infection. ALN-RSV01 was also shown to be effective in the treatment of an existing RSV infection.
Additional studies in mice demonstrated that intranasal administration of ALN-RSV01 did not activate an interferon response. Ongoing and future studies are analyzing the topical delivery of ALN-RSV01 to the lung by aerosolization through a nebulizer.
"Alnylam is rapidly progressing toward our goal of developing a safe and efficacious RNAi therapeutic for the treatment of RSV infection," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam Pharmaceuticals. "Based on the encouraging pre-clinical data we have seen to date, we are driving ALN-RSV01 forward toward human clinical trials in the first half of 2006."
RSV is a highly contagious virus that causes repeated infections in both the upper and lower respiratory tract. RSV infects nearly every child at least once by the age of two years and is a major cause of hospitalization due to respiratory infection in children and people with compromised immune systems and others.
RSV infection typically results in cold-like symptoms but can lead to more serious respiratory illness such as croup, pneumonia, bronchiolitis, and, in extreme cases, death. RSV infection in the pediatric population accounts for greater than 100,000 hospitalizations per year in the United States.
The development of childhood asthma is also known to occur after childhood RSV infection. As a result, there is a significant need for novel therapeutics to treat patients who become infected with RSV.
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.
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