RaNA banks $55M to get its RNA drugs into the clinic

Cambridge, MA's RaNA Therapeutics has wrapped up a $55 million B round, putting together the cash it needs to move a pair of novel treatments toward Phase I and build out its internal product engine.

The company is at work on drugs designed to elevate the production of certain proteins within the body's cells by interfering with epigenetic signaling. RaNA has developed targeted therapies that home in on RNA and upregulate beneficial proteins, a technology the company believes is ideal for diseases tied to protein deficiencies.

With its latest fundraise, RaNA is pushing forward with two preclinical treatments for the rare genetic disorders spinal muscular atrophy and Friedreich's ataxia. Each ailment is caused by the suppression or degradation of a key protein, and RaNA figures its in-house technology could be key to reversing the course of disease.

The biotech is now in the lead-optimization stage for both programs, planning to kick off preclinical toxicity studies next year and launch human trials as soon as 2017, CEO Ron Renaud said. And RaNA is also working through exploratory research on new genetic targets, hoping to identify a handful within the next 18 months and gradually flesh out its pipeline.

At the same time, the company plans to dedicate some of its latest fundraise into honing its discovery platform, which is devoted to chasing the regulatory protein PRC2 around the genome. PRC2 binds to snippets of RNA and acts as a suppressant, halting gene expression and standing in the way of protein production. RaNA's technology works by blocking that bind, freeing target RNAs from PRC2 suppression and spurring them to produce a specific protein. Thus, whenever RaNA can find a gene held down by PRC2, it's looking at a potential therapeutic target.

Emboldened by its early preclinical success, RaNA is expanding its R&D footprint in hopes of identifying new candidates in oncology, inflammation and other diseases. Earlier this year, the company recruited Regulus Therapeutics ($RGLS) veteran Balkrishen Bhat to come aboard as head of chemistry, and, come September, RaNA is slated to move to a new space that will quadruple its capacity. The plan, Renaud said, is to bring in-house the chemical synthesis and analysis work the company now largely outsources, settling in for the long haul with ambitions to churn out more and more drug candidates.

"We're building RaNA at this point," Renaud said. "It's a process of building toward a platform where can really leverage this technology to develop some potentially important therapeutics here."

The Baupost Group and Merck's ($MRK) MRL Ventures co-led RaNA's latest round, joined by fellow new investors Springs Capital Management, Brookside Capital and Leerink Partners. Previous backers Atlas Venture, Monsanto, MS Ventures, Omega Funds, Partners Innovation Fund, Pfizer Venture Investments and SR One all returned after pitching in on RaNA's $25 million Series A.

- here's the release