Effigene currently has three intellectual assets: a unique RNAi reporter system, a siRNA research kit and two small molecules with the potential as adjuvant siRNA therapeutics.

Effigene’s functional RNAi reporter system will enable pharmaceutical companies to systematically screen on a genome-wide, high-throughput scale to identify small molecules that can inhibit or enhance siRNA activity, genes that can modulate the RNAi pathway, and small molecules or genes that can modulate the activities of specific endogenous microRNA.

To validate our technology, we have successfully used our reporter system to identify two small molecule compounds, EFI-001 and EFI-007, which can significantly potentiate target mRNA knockdown by siRNA.  Combination of EFI-001 with siRNA has the potential to dramatically increase therapeutic siRNA potency, resulting in less administration volume and/or off-target effect.  In vitro and in vivo animal studies have demonstrated that EFI-001 greatly improved siRNA’s pharmacological activity. Effigene has received favorable feedback from NIH for our research proposal to investigate the adjuvant siRNA therapeutic potential of EFI-001 using various animal disease models.