July 23, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Cambridge, Mass.-based RaNA Therapeutics, Inc. , announced today that it had closed on a $55 million oversubscribed Series B financing round. Co-leading the round was MRL Ventures, the early-stage therapeutics fund of Merck & Co. , and The Baupost Group, LLC.
Also participating were new investors Rock Springs Capital Management, Brookside Capital, Leerink Partners LLC and a blue chip public investment fund whose identity was not disclosed. Joining in were existing investors Atlas Venture, Monsanto, MS Ventures, Omega Funds, Partners Innovation Fund, Pfizer Venture Investments and SR One.
Joshua Resnick, president of MRL Ventures, will join the RaNA board of directors. The original round closed in January 2012 with $20.7 million.
“We are thrilled to have the support of such a distinguished group of investors that are so excited to utilize this investment to devote more resources to our platforms and programs and further build out the capabilities, team and infrastructure necessary to support our evolution towards a clinical organization,” said Ronald Renaud, chief executive officer of RaNAin a statement. “Our proprietary platforms for targeting RNA and enabling the body’s own cells to selectively increase protein expression create multiple opportunities to develop novel medicines to treat a broad range of diseases with little or no treatment options today.”
RaNA’s approach to RNA-based therapies is different than the more typical RNA interference (RNAi) approach. RaNA’s focus is on supercharging RNA transcription for specific desirable proteins. RNAi’s approach, on the other hand, is to turn off the transcription of disease-related proteins.
“I am extremely excited about RaNA and the progress it has made since we seeded it,” said Brian Gallagher, a partner with GlaxoSmithKline ’s SR One venture group, one of the original investors, in a quote to Forbes. “It is extremely innovative technology with enormous potential to treat diseases utilizing their novel gain of function approach.”
In January 2015, Merck inked a licensing and collaboration agreement with Cambridge, Mass.-based Moderna Therapeutics for an upfront payment of $50 million. That deal was for the discovery and development of vaccines and passive immunity treatments against viral diseases using Moderna’s modified messenger RNA (mRNA) technology. The research deal is for three years with a possible one-year extension.
As far back as 2006, Merck has been interested in RNA therapeutics when it acquired Sirna Therapeutics for $1.1 billion. Sirna was focusing on RNAi.
RaNA’s focus is on long non-coding stretches of RNA. For a long time, scientists referred to these sections of RNA and DNA as “junk,” but over the last decade, researchers are gaining more insight into the so-called “dark matter” of DNA and RNA, finding that they often are involved in turning gene expression on and off like a switch.
RaNA is starting by attempting to develop drugs for spinal muscular atrophy, a genetic muscle control disease, and Friedreich’s ataxia, a genetic neurodegenerative disease. They hope to start clinical trials in 2017.
The funds raised in the Series B round will be used to take the company’s two compounds into human trials. There are rumors RaNA might go public, but Renaud won’t commit, telling Xconomy he plans to build the company “brick by brick. … Going public or whatever strategic initiative we want to do corporate-wise down the road will only come as a byproduct of building a great private company.”
