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Moderna, Vertex to collaborate on cystic fibrosis

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Moderna Therapeutics Inc. has emerged as a partner of choice for drug makers eager to supplement their own research.

Vertex Pharmaceuticals Inc. became the latest established biopharma company to team up with Cambridge-based Moderna, announcing a three-year agreement Wednesday to jointly develop new treatments for cystic fibrosis. The deal could be worth up to $315 million to Moderna, and will give Boston-based Vertex a $20 million equity stake in the five-year-old company.

Moderna is working on experimental drugs using a technique called "messenger RNA" that turns the power of patients' genetic information against their diseases. The approach, which can convert copies of DNA into healthy proteins in patients with defective ones, is part of the field of gene therapy that is seeing a resurgence after more than a decade of caution by scientists.


"There's new interest in using genetic methods for therapeutics," said Michelle Hoffmann, senior vice president for Boston consulting firm Back Bay Life Science Advisors. "If you had asked people five or 10 years ago, they would have said this is a long way off. Now they're more willing to take a risk and they're willing to explore different technologies."

Moderna has previously struck research alliances with Merck & Co. on personalized cancer vaccines, Alexion Pharmaceuticals Inc. on drugs for rare liver diseases, and AstraZeneca PLC on treatments to boost heart function in patients who have suffered heart attacks.

"We're playing a very long game," Moderna chief executive Stephane Bancel said in an interview. "Our goal is to bring the best medicines to patients, and we always like to start with a partner who can bring expertise to a new area we're looking at. It's about whatever organ system we can get messenger RNA into. This is important because it brings it into the lungs."

Vertex markets two approved drugs, Orkambi and Kalydeco, to address different genetic mutations that cause cystic fibrosis, an obstructive lung disease. About 27,000 cystic fibrosis patients worldwide are now eligible to take one of the two Vertex medicines, but thousands of others have genetic mutations for which the company has not yet developed treatments.


Collaborating with Moderna is "another step in our long-term commitment to bring new medicines to people with cystic fibrosis," said Vertex spokeswoman Heather Nichols.

The aim of the Moderna-Vertex collaboration is to deploy messenger RNA to enable cells in the lungs of patients with the disease to produce functional copies of a defective protein known as a cystic fibrosis transmembrane conductance regulator.

Bancel said Moderna will lead early-stage research in the Vertex partnership, and turn over to Vertex later-stage development of promising drug candidates. He said early investigation of mRNA-based cystic fibrosis treatments has already been done by a Moderna business unit, called new venture labs. It is too soon to say whether therapies resulting from the collaboration will replace current Vertex drugs or work in combination with them, Bancel said.

Under the deal, Moderna will receive $40 million upfront, including a $20 million cash payment and a $20 million convertible note investment that will allow Vertex to own shares of Moderna when the company secures its next round of financing. The agreement gives Moderna the potential for up to $275 million more in milestone payments if drug candidates meet regulatory milestones as well as royalty payments on sales of approved drugs.


Moderna's other partners have taken equity stakes in the company totalling about $237 million, giving them combined ownership of about 10 percent, Bancel said. He said Vertex's percentage stake in Moderna won't be calculated until Moderna's next financing round.

Founded in 2011, Moderna has nearly 400 employees at two locations in Cambridge and is scouting for a site in the Boston suburbs to build a drug manufacturing plant.

Robert Weisman can be reached at robert.weisman@globe.com. Follow him on Twitter @GlobeRobW.