RaNA Expands, Joins Messenger RNA Drug Race With Shire Deal

RaNA Therapeutics is branching out. Today, the Cambridge, MA, startup is acquiring some assets from pharmaceutical giant Shire that immediately make it a player in an emerging, yet unproven field of drugmaking—messenger RNA therapeutics.

Cambridge, MA-based RaNA has acquired a small, recently shuttered unit of Shire (NASDAQ: [[ticker:SHPG]]) devoted to making drugs with synthetic mRNA. Financial terms aren’t being disclosed, but Shire gets an unspecified equity stake in RaNA in the deal and would get downstream payments if drugs from the acquisition progress forward successfully.

The deal includes two experimental programs for the rare disease cystic fibrosis and the rare metabolic disorder ornithine transcarbamylase deficiency. CEO Ron Renaud says RaNA plans to file paperwork by the end of 2017 to begin human clinical trials for both programs. RaNA will also bring all of the employees working on Shire’s mRNA drugs at the company’s Lexington, MA, outpost in-house, leaving RaNA with over 60 workers total once the integration is complete.

Renaud says the company is in the middle of a “significant raise” of new cash to support the larger enterprise, but he wouldn’t disclose specifics. RaNA raised a $55 million Series B round in July 2015. The company’s investors include Atlas Venture, Merck Ventures, and SR One.

RaNA (pronounced “Ronna”) was formed in 2011 with a plan to mine “junk DNA”—a bunch of DNA sequences that don’t code for proteins and were thus once deemed useless—for drug targets. That work, which has led to two preclinical programs for the rare diseases spinal muscular atrophy and Friedreich’s ataxia, is continuing, Renaud says. But adding a second RNA-based drugmaking method broadens the scope of the company, adds other programs in different disease types, and gets RaNA much closer to human clinical trials. RaNA aims to use multiple RNA drugmaking methods to address disease targets that small molecules, biologic drugs, or gene therapies can’t get to.

“This was a transformational opportunity for us,” Renaud says of the deal.

The idea with mRNA drugmaking is to deliver synthetic mRNA strands into the body so patients’ cells can produce their own therapeutic proteins. It’s a novel way of making drugs that could in theory give drugmakers a way to produce nearly any protein to combat diseases. Yet the method is unproven in humans.

Still, the prospect of mRNA drugs has attracted several drugmakers. For example, one of the more high-flying private biotechs in recent memory, Cambridge-based Moderna Therapeutics, has used a series of big partnership deals and equity financing to amass a roughly $1 billion war chest, but it has yet to produce any human clinical data. And Moderna isn’t alone; Germany-based companies CureVac and BioNTech, among others, are also in the mix, and those two also have alliances with pharmaceutical companies such as Sanofi and GlaxoSmithKline.

CureVac is working on cancer immunotherapies and vaccines. BioNTech is also focusing on oncology. Moderna’s efforts are much more broad, with programs in cancer, rare diseases, vaccines, and other areas allotted to different wholly owned subsidiaries. Moderna cut a deal with Vertex Pharmaceuticals (NASDAQ: [[ticker:VRTX]]) last year to develop an mRNA drug for cystic fibrosis, like Shire.

Shire, meanwhile, has been working on mRNA drugs in-house since 2008. No programs have moved into human trials as of yet, however. And, while Shire announced development of the CF mRNA treatment two years ago along with a funding deal with the non-profit Cystic Fibrosis Foundation, it hasn’t made any announcements regarding the program since.

Asked why Shire decided to deal off its mRNA technology rather than develop the drugs in-house, Shire spokesperson Lisa Adler said in an e-mail to Xconomy that the company chose to discontinue its mRNA drugmaking efforts earlier last year in a “portfolio optimization exercise.”

“We are confident that, as a company focused on next generation RNA-targeted medicines, RaNA will be able to apply the [technology] across various areas of medicine,” she wrote. “Shire continues to recognize the potential of this platform and is committed to a smooth transition to RaNA of the [experimental drugs] and the individuals who have contributed to their progression.”