SAN DIEGO, Calif. November 15, 2001 AGY Therapeutics, Inc. today announced study results that demonstrate for the first time that RNA interference (RNAi), a technique for silencing genes, was shown to successfully block neuronal gene expression in mammalian cells, providing opportunities for greater analysis of gene function and development of gene-specific therapeutics related to stroke and other central nervous system (CNS) diseases. The study was presented here at the 31th Annual Meeting of the Society of Neuroscience.
While researchers have demonstrated that RNAi can function in mammalian cells, this study is the first to demonstrate successful mediation of gene-specific silencing in mammals.
"These results prove that RNAi in mammalian cells is a viable and very promising approach to discovering CNS disease treatments and warrants further investigation," said Roman Urfer, Ph.D., AGY's Vice President of Drug Discovery and Development. "In addition to confirming the feasibility of RNAi in humans, we have identified several targets, which we plan to further investigate in more extensive preclinical studies in a variety of mammalian models."
RNAi has recently been shown to be a powerful approach to silencing or suppressing genes by blocking messenger RNA in diverse invertebrate systems like fruit flies and nematode worms. However, its application to mammalian cells until recently has had limited efficacy and possible unspecific inhibition.
Utilizing RNAi as part of AGY's imAGYne platform in their target validation, researchers demonstrated that double-stranded RNA with an average size of 500 base pairs mediated gene-specific silencing in a mouse neuroblastoma cell line. RNAi-mediated gene silencing also was shown to rapidly knock down gene expression for functional validation of novel genes involved in CNS diseases, allowing for faster identification and validation of gene-specific therapeutic targets.
About the ImAGYne Platform
AGY Therapeutics developed the imAGYne platform to provide researchers with a comprehensive approach to defining the intra- and extra-cellular signaling pathways underlying the pathological progression of disease, so potential therapeutic targets could be more easily identified and qualified for further investigation. The imAGYne platform begins with animal models that replicate the same disease found in humans. Next, diseased tissue samples are compared to normal tissue samples. After regulated genes are identified, multiple expression profiles are run over a period of time to distinguish genes that are unique to the diseased tissue sample and their special signaling activities. Upon learning the "what, when and where" of the signaling activities, the activities are translated into pathway models that underlie the disease process. While multiple pathways and targets are identified, it is not until after multiple strategies are applied that the highest-quality targets are gleaned for future drug or diagnostic development.
ABOUT AGY THERAPEUTICS, INC.
AGY Therapeutics, Inc. is a privately held biopharmaceutical company focused on developing therapeutic products for the treatment of diseases of the central nervous system. With its proprietary imAGYne, imArrays and imFormatics platforms, AGY has built a unique knowledge base of the neuroprotective capacities and vulnerabilities of the nervous system.
This news release contains forward-looking statements about future research and development efforts. These statements represent our judgment as of the date of this news release and are subject to risks and uncertainties that could cause actual results or events to differ materially from those expressed in such forward-looking statements. In particular, we face risks and uncertainties that further research into these genes may not go forward as planned or that future scientific data on these genes may not provide supportive data for the diseases we are targeting.