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The Promise of RNAi

Opening new doors for pharmaceutical innovation.

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RNA interference (RNAi) is a biologic process in which certain double-stranded RNA molecules inhibit the expression of disease-causing genes by destroying the messenger RNAs (mRNAs) of those genes. It reflects a new approach in the development of specific and powerful therapies to treat rare diseases, chronic liver diseases, cardiovascular disease, and viral infectious diseases. RNAi has the potential to treat these diseases by silencing some of the most well-validated, yet previously inaccessible drug targets.

Leveraging the Potential of RNAi Technology

RNAi offers the potential to overcome the key limitations of traditional approaches to drug therapy. Rather than targeting and binding to proteins to inhibit their activity, RNAi exerts its effects one step earlier in the gene silencing process by targeting the mRNA, the instruction set that directs the building of the protein. By attaching this instruction set, RNAi can attack any target. This includes disease-causing genes that are expressed exclusively inside cells and which lack good small-molecule binding pockets, putting them beyond the reach of conventional antibody and small-molecule modalities.

Some of these disease-causing genes have been known for decades and are considered highly attractive to drug developers. Additionally, RNAi-based therapeutic approaches hold promise in offering more convenience for patients via infrequent subcutaneous dosing and a very long duration of effect, while reducing the side effects that can occur with small-molecule and antibody therapy.

Through RNAi, Dicerna’s product candidates have the potential to reach important, well-validated targets in liver-targeted diseases – targets that the pharmaceutical industry has not been able to access with the conventional therapies.