The Science of RNAi

Leveraging the body’s naturally occurring ribonucleic acid interference (RNAi) response, Sirnaomics is working to develop therapies that target key disease proteins.

RNAi therapies utilize two short segments of RNA, known as short interfering, or silencing RNA (siRNA), to defeat proteins involved in the progression of certain diseases. The siRNA segments are encapsulated within a histidine-lysine polypeptide (HKP) that ensures their intact delivery to target cells within the body. Once taken up at a cellular level, the siRNA strands are released to activate an enzymatic process driven by the RNA-Induced Silencing Complex (RISC). Through this process, the messenger RNA strands of the disease proteins are targeted and cleaved, ultimately preventing their function to express proteins within a cell.

In addition to the synergistic effect of dual gene/protein targeting, RNAi therapeutics work to prevent the reproduction of harmful proteins within the body, rather than neutralizing existing disease. Additionally, this new class of drugs offers speedier development timelines compared to traditional methods of development involving the screening of small molecules or generating antibodies against a protein. This speedier development along with the potential for earlier treatment offers hope for improving the lives of patients who have been diagnosed with conditions ranging from cancer to fibrotic diseases.