Generations of high school kids have been taught that only about 3 percent of the human genome is actually useful—meaning it contains genes that code proteins—and the rest is “junk DNA.” Cambridge, MA-based RaNA Therapeutics was founded on the idea that the so-called junk is actually gold, because it contains a type of RNA that can flip genes on inside cells, potentially offering a new approach to modulating diseases. RaNA is coming out of stealth mode today and announcing a $20.7 million Series A financing led by Atlas Venture, SR One, and agricultural giant Monsanto (NYSE: MON). Partners Innovation Fund also participated in the funding.
Research done in the last several years has shown that most of what was formerly dubbed junk in the DNA is actually transcribed into “long non-coding RNAs,” which regulate genes on a high level and control the development process. When these RNAs are improperly expressed in cells, they can cause disease. RaNA is based on technology developed by scientific founder Jeannie Lee, a Howard Hughes Medical Institute Investigator at Massachusetts General Hospital. Lee discovered that some long non-coding RNAs interact with a specific protein—and that blocking that protein can selectively activate genes.
Arthur Krieg, CEO of RaNA, says RaNA has yet to determine which diseases it’s going to go after, but that the opportunities are vast. Krieg, who joined Atlas in 2011 as an entrepreneur in residence, says part of the process of starting up RaNA involved interviewing doctors about how they’d like to see the technology deployed. “We asked them, ‘If you could turn on any gene in the genome, what would you go for?'” Krieg recalls. That polling yielded more ideas than RaNA can pursue—not surprising, Krieg says, since most drug approaches on the market today involve turning genes off rather than on. “The only approach that comes close to what we’re doing is gene therapy, and that has posed problems,” he says, referring to safety issues that have arisen in some experimental gene-therapy procedures.
Monsanto participated in RaNA’s funding because the startup’s technology might also prove useful in agricultural biotech, says Jean-Francois Formela, a partner in Atlas Ventures’ life sciences group. Monsanto and Atlas formed a strategic alliance last year to search for investments that could be applied to both human health and plant biology, Formela says. The RaNA deal is the first one struck under that alliance. “Monsanto had invested in RNA before. The alliance doesn’t focus solely on that, but it was part of the vision,” Formela says. Monsanto has a board seat with RaNA and an option to collaborate on ag-bio applications going forward, though RaNA is not currently working in that area, Krieg adds.
Atlas, too, has a long-standing interest in RNA. It was an early investor in Alnylam (NASDAQ: ALNY), the Cambridge, MA company that’s working on RNA interference technology. And it invested in Miragen, a Boulder, CO company working on microRNA therapeutics. Krieg had been working on RNA at Pfizer ever since the pharmaceutical giant bought the company he co-founded, Coley Pharmaceuticals, in 2008. When Pfizer eliminated that unit last year, Atlas recruited Krieg to help start RaNA. “We really started it from scratch,” Formela says.
RaNA, which has a staff of about a dozen and is about to move into a new office in Cambridge, is currently picking its targets for human therapeutic development, Krieg says. He hopes to be ready to start human trials by the end of this year. The company is already talking to potential pharma partners and will consider early-stage collaborations with companies that are interested in pursuing RaNA’s new scientific approach, Krieg says. “I’m not aware of other companies working on this,” he says. “I wouldn’t be surprised if we have one or two partners by the end of the year.”
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