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This new class of pharmaceutical agents has the ability to block or silence gene expression at the level of messager RNA (mRNA) and inhibit the production of specific proteins that are implicated in disease processes.

Based on the 2006 Nobel Prize winning work of Andrew Fire and Craig C. Mello, the mechanism of RNAi interference is the focus today of drug discovery and development efforts throughout the biotechnology and pharmaceutical industry.

These therapeutics have potential superiority relative to their target selectivity and specificity, ability to reach heretofore "undruggable targets" and potential to streamline and accelerate drug discovery and development compared to small molecule and other macromolecule approaches. In addition, RNAi therapeutics, also referred to as short interfering RNA or siRNA, have demonstrated broad applicability to many therapeutic areas, including cancer, inflammatory and autoimmune diseases, infectious disease and metabolic disorders.

These promising therapeutics are being held largely in check today by the lack of broadly applicable delivery technology. RNAi therapeutics generally cannot, on their own, penetrate the cell membrane and gain access to the cytoplasm where they can reach the drug targets.

The PhaseRx polymer system overcomes the central stumbling block in the field of RNAi therapeutics by delivering RNAi molecules into the cytoplasm where they can reach and inhibit the desired target of interest.


 
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