Alnylam Pharmaceuticals (NASDAQ: [[ticker:ALNY]]) has spent the last couple of years amassing clinical trial evidence it needs to show that RNA interference just might work against diseases that can’t be treated by conventional pills or protein drugs. And Alnylam’s rising tide is apparently helping lift at least one more RNAi boat.
Watertown, MA-based Dicerna Pharmaceuticals, which has its own proprietary approach to silencing disease-related genes through RNAi, is announcing today it has raised $60 million in a Series C round of venture financing.
The round was led by RA Capital, one of several new names to join Dicerna’s investor group along with Brookside Capital, Deerfield, and Omega Funds—firms better known for their investments in public companies. All five of Dicerna’s previous shareholders contributed to the round as well: Abingworth Management, Domain Associates, Oxford Bioscience Partners, Skyline Ventures, and SR One (the VC arm of GlaxoSmithKline). RA Capital partner Peter Kolchinsky has joined Dicerna’s board of directors as part of the investment.
The cash, which now gives Dicerna $110 million in raised venture funds to date, should give the company enough money to wrap up its preclinical work and bring the first of its RNAi drugs into clinical trials. CEO Doug Fambrough expects Dicerna to file its first investigational drug application around the end of the year, and begin dosing human patients in its first trial in the first quarter of 2014. Dicerna expects to bring “multiple” programs into the clinic with the cash, which Fambrough says will take the company all the way into the second half of 2016.
“It’s a long runway,” he says. “It shows that we have demonstrated, on a preclinical basis, that we have things that are likely to work in people, and that we have been able to address the challenges—particularly the challenges of delivery for RNAi—effectively.”
Like other companies in the RNAi space, Dicerna is creating drugs that are designed to hit a disease at its roots by engineering RNA molecules that shut down the production of specific disease-causing proteins. Dicerna, however, does this in its own differentiated way. Rather than engineering small RNAi molecules in the way that, say, Alnylam does, Dicerna makes them a little bit longer, which enables them to act a step earlier in the gene-silencing process, and thus potentially be more potent and last longer in the body. This, in turn, could mean that Dicerna could give its drugs in lower doses, produce them for less money, and administer them with fewer shots.
Even so, Dicerna has yet to make the big step from promising research-based company to a true clinical-stage biotech. Today’s news represents the first time
