The technology developed at research labs in San Diego, California will enable short double-stranded RNA and microRNA to cross cell membranes, a problem which has cost the industry billions of dollars in trying to solve.
Company founder and inventor of the technology, Professor Steven Dowdy, told In-Pharmatechnologist.com the technology “is essentially a covalent RNAi pro-drug that is converted by intracellular enzymes into the active RNAi drug, but only inside of cells.”
The small size of the molecule and the permeability of the pro-drug – called Ribonucleic Neutrals (RNN) – ensure the enzymes are not present outside of the cells enabling RNA molecules to cross membranes of multiple cell types. This differs from previous RNAi development which have used large molecules.
The comments follow Solstice's announcement of an $18 million series A financing last week which will be used primarily for R&D preclinical work to advance the cell-permeable RNAi pro-drug in vivo.
Big Pharma and RNAi
Solstice and its backers hope such technology will respark Big Pharma interest in RNAi.
Advances have been made in RNAi over the last couple of years though setbacks have been plentiful. Last year Alnylam was forced to axe jobs and cut costs in order to bring an RNAi product to market, after Novartis ended an agreement in the licensing of Alnylam’s RNAi platform.
Furthermore, Arrowhead is continuing to advance its progress after purchasing SiRNA assets from Roche in 2010 after Roche lost confidence and withdrew from this area of drug delivery.
Solstice, however, claims its drug delivery technology can recapture the interest of drug makers.
“The only reason that these Big Pharmas got out of the RNAi business,” said Dowdy, “was because of delivery. Therefore, by solving the delivery problem it will likely bring most of them back inside to take a second look.”