Our Company

Intellectual Property

We will continue to invest in expanding and improving our intellectual property protection for our GalXCTM RNAi platform technology, including any innovations and improvements. We seek to obtain worldwide patent coverage for novel molecules, composition of matter, pharmaceutical formulations and methods of use, including treatment of disease, methods of manufacture and other novel uses for the molecules originating from our research and development efforts. We believe we have a robust patent portfolio covering our proprietary GalXC RNAi platform and other RNAi technologies. As of March 29, 2017, our patent estate, not including the patents and patent applications we have licensed, included over 20 issued patents or allowed patent applications and over 100 pending patent applications supporting commercial development of our RNAi molecules and delivery technologies.

Our activities focus on two areas:

RNAi Triggers

The City of Hope (COH), a leading academic and research hospital, has granted us an exclusive worldwide license for RNA structures having a 25 to 30 nucleotide sense strand, a blunt end at the 3’ end of the sense strand and a one to four nucleotide overhang at the 3’ end of the antisense strand. This approach is referred to as Dicer substrate short interfering RNA, or DsiRNA, and is covered by several issued US patents, including US patents no. 8,084,599 and 8,796,444, titled, “Methods and Compositions for the Specific Inhibition of Gene Expression by Double-Stranded RNA.”

GalXC RNAi Platform

We have also pioneered an RNAi molecule technology that adds multiple functional features to the RNAi triggers, which is in part facilitated by their extra length, and we refer to these as extended Dicer substrate short interfering RNAs, or DsiRNA-EX. This technology is particularly suited to the development of GalXC compounds, which comprise DsiRNAs that possess a chemically attached targeting component, making them highly efficient in accessing targeted tissue and suitable for subcutaneous injection. GalXC compounds are molecules wherein the delivery/targeting vehicle is directly attached, or conjugated, to the RNAi molecule. The extra length of the DsiRNA-EX molecules in GalXC compounds provides multiple avenues to favorably perform such conjugation. We are using our proprietary GalXC platform to advance the development of next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. This technology is broadly covered by several patents and patent applications, including US patents no. 8,349,809, 8,513,207, and 8,927,705.