By Mark Terry


Ribometrix, based in Durham, NC, closed on a Series A financing worth $30 million. The round was led by M Ventures, with the participation of new investors Amgen Ventures, Pappas Capital and Illumina Ventures. Founding investors also joined the round, including SV Health Investors, AbbVie Ventures, Hatteras Venture Partners, MP Healthcare Venture Management, the Dementia Discovery Fund and Alexandria Venture Investments.

Ribometrix focuses on developing small molecule drugs that directly modulate RNA activity in cancer and neurodegeneration. The company’s particular focus is the shapes RNA molecules form when they fold into themselves. These are the types of structures that small molecule drugs can attach to.

The company is built on technology that came out of the research of Kevin Weeks at the University of North Carolina (UNC). The technology is dubbed SHAPE, which allows scientists to investigate and predict the 3D structure of RNA in cells.

There is a version of SHAPE publicly available, but the version Ribometrix uses is an improved version. Weeks told Xconomy that the company has its own bioinformatics software and other laboratory-based technologies.

In addition to the financing, Hakan Goker, senior investment director of M Ventures, will join Ribometrix’s board of directors as a non-executive director.

“The ability to target RNA directly with small molecules offers the potential to unlock large, previously ‘undruggable,’ target classes that could revolutionize the treatment of a wide variety of diseases including cancer,” said Goker in a statement. “The Ribometrix team has created and harnessed a highly novel platform delivering a strong pipeline of programs for future development.”

Ribometrix isn’t the only company working in the area of RNA structure. Another, based in Waltham, Mass., is Arrakis Therapeutics, which is using the SHAPE techniques. It’s also built its own bioinformatics software. Jennifer Petter, founder and chief scientific officer of Arrakis, told Xconomy, “We are heavy users of SHAPE and related technologies. Kevin [Weeks]’s work [in developing the SHAPE technology] is an important contribution to the RNA field.”

Ribometrix is starting its work in cancer and neurology. One of its targets is c-myc, a well-known cancer gene that is generally viewed as “undruggable.” It is also working on a target in Huntington’s disease.

Ribometrix’ work is on RNA molecules that create “higher-order” 3D structures. Not all RNA molecules do this, Weeks told Xconomy, but the ones that do have so-called “pockets” that small molecule drugs can fit into.

Another RNA-structure company is San Diego-based Expansion Therapeutics. In early January, Expansion raised $55.3 million in Series A co-led by 5AM Ventures, Kleiner Perkins, Novartis Venture Funds and Sanofi Ventures, with participation from RA Capital Management and Alexandria Venture Investments.

Other companies include Skyhawk Therapeutics, Accent Therapeutics and Gotham Therapeutics.

Ribometrix currently employs 15 people but expects to double that number in the next year.

“A huge medical opportunity awaits RNA-targeting small molecules that can be designed in a systematic fashion, analogous to discovery methods currently employed for protein targets,” stated Ribometrix’ founding chief executive officer Michael Solomon. “We welcome the strong support of both new and existing leading venture investors as we pursue this exciting opportunity. With unique expertise around RNA, an expanding drug discovery team and our broad platform of complementary small molecule and RNA target discovery technologies, we are well positioned to advance a broad pipeline of compelling drug programs.”



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