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.