Shire spins out mRNA group to RaNA, which is hatching plans to tackle cystic fibrosis in the clinic

Over the last few years, the executive crew at Shire has on a few rare occasions spotlighted their work in mRNA, touting its potential for adding to its forecast of billions in new revenue. A little more than two years ago, the research arm of the Cystic Fibrosis Foundation contributed $15 million to the venture. But now, with their big merger with Baxalta still being worked through, Shire is lateraling their mRNA team — more than a dozen staffers and two key programs — to RaNA Therapeutics in exchange for a chunk of equity.

The Cambridge, MA-based RaNA gets two preclinical programs that are poised to enter the clinic in the near future, with a shot at launching clinical programs on cystic fibrosis and urea cycle disorders in H1 2018. And RaNA CEO Ron Renaud is clearly pumped about adding a new platform to the RNAi company at a time that he is halfway through raising a “substantial” amount of cash to back the next stage of development work at the biotech.

“What I was most intrigued by,” Renaud tells me, “is that this initiative was underway in 2008. This is something they have been working on for awhile, building IP, working through all those development issues to where it is today. Having this new platform opens up a vast amount of target space for us.”

Getting the group will increase RaNA’s head count from 48 to more than 60, says the CEO, giving it a makeover that will fundamentally alter its profile.

Renaud declined to say for now how much equity Shire is getting, or how much he plans to raise.

For its part, a Shire spokesperson says the growing company spun out the platform as part of an ongoing assessment of what works best inside, or outside, Shire.

“From a big picture perspective,” she adds, “periodically evaluating and discontinuing programs is necessary to maintain a fully optimized pipeline where achieving innovation/maintaining focus are the goals….Shire will stay close to RaNA and the MRT Platform as Shire receives an equity stake in RaNA and is eligible for future milestones and royalties on products developed with the technology.”

In a short period, RaNA will jump into a position where it can start making a mark in a field that has attracted intense interest. The nearby biotech Moderna has earned the lion’s share of the attention, and $1.9 billion in backing, for their mRNA work — using messenger RNA technology to smuggle in instructions for cells to make specific therapeutic proteins. Moderna plans to unveil more of its pipeline work next week at the big JP Morgan confab, but it’s still in early-stage development with its lead efforts focused on new vaccines. CureVac in Germany has more advanced work on a prostate cancer vaccine.

It is extremely risky work with little by way of solid clinical data to prove that you can actually do what’s intended. On the other hand, RaNA now is close enough behind the leaders that it can position itself in key fields just as the sector is either taking off, or counting casualties.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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