Drug trailblazers Moderna, Juno grab forum’s spotlight
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SAN FRANCISCO — New ways to fight diseases are guaranteed to get plenty of attention at the top gathering of health care entrepreneurs and investors. So it's no surprise that Moderna Therapeutics Inc. and Juno Therapeutics Inc., pioneers in potentially game-changing drug discovery technologies, emerged as two of the highest profile companies at this week's J.P. Morgan Healthcare Conference.
Both of the early-stage US companies are run by seasoned European executives, and both are pushing the envelope on approaches that could yield multiple treatments for everything from hard-to-treat cancers to rare genetic disorders.
Each also caught the attention of conference-goers by unveiling significant deals and alliances that could accelerate their research and development efforts.
Four-year-old Moderna, based in Cambridge, Mass., revealed that two Big Pharma companies would be licensing its messenger RNA technology. Merck & Co., the US drug giant, will use that method to develop vaccines against viral diseases.
Anglo-Swedish drug maker AstraZeneca Inc. will collaborate on using mRNA – which makes copies of an individual's healthy genes and deploys them against disease targets – for cancer therapies. Moderna and AstraZeneca struck an earlier deal to work on mRNA drugs to combat cardiovascular, metabolic, and renal diseases.
Moderna, which has about 320 employees at two sites near Kendall Square and is planning to expand to a third site, last year received $450 million from an investment consortium in the largest private financing round ever. The company's founders include Cambridge venture capital firm Flagship Ventures and Massachusetts Institute of Technology professor Robert Langer.
This week, Moderna also revealed an initial grant of $20 million from the Bill and Melinda Gates Foundation. That money will be used to help develop a new drug cocktail based on the mRNA technology to treat the HIV virus.
"Messenger RNA is the software of biology," French-born Moderna chief executive Stephane Bancel said in an interview. "We are sending a different set of instructions from your natural ones, but they're mimicking how biology does it naturally. . . . Our ambitions are very clear. We want to bring dozens and dozens of drugs to market through our technology and through our partnerships."
Moderna scientists are engaged in pre-clinical research on 92 experimental drugs and more advanced development-stage research on seven. The company has one drug in clinical studies to treat infectious diseases and hopes to start trials of four others by the end of this year.
"This is totally unprecedented," Bancel said. "If you look at biotech companies, nobody has this many drugs in the clinic so fast. We have to make this science work because there are so many diseases that it could treat."
For its part, Juno, a three-year-old company based in Seattle, is a leader in the hot field of immunotherapy, which seeks to stimulate the immune system to fight diseases. Specifically, it is working in a promising niche called CAR-T, which engineers the immune system's T cells to kill cancer tumors.
Earlier this week, Juno, which has about 300 employees, said it was paying $125 million in cash and stock to buy Harvard University spinout AbVitro Inc. Juno operates a 15-person division in Waltham, but it will be moving privately held AbVitro and its 18 employees from Boston to Seattle later this year.
In contrast to Moderna, one of the handful of private companies invited to present at the J.P. Morgan conference, Juno went public 13 months ago in an initial offering that raised $264 million and established the Seattle startup as a formidable competitor to Swiss drug maker Novartis AG, which is working on T cells with research pioneer Carl June at the University of Pennsylvania.
Juno chief executive Hans E. Bishop said in an interview that "the competition for us is cancer." T cell immunotherapies "have the potential to become the third pillar of medicine," after pills and biotech drugs, said Bishop, a British native. "How big we can get is a function of how good our medicines are."
The company's technology came from the labs of its six scientific co-founders at Fred Hutchinson Cancer Research Center in Seattle, Seattle Children's Hospital, and Memorial Sloan Kettering Cancer Center in New York.
Researchers there have experimented with injecting new genes into T cells, creating antibody fragments that can bind to cancers and destroy them. Juno has nine drug candidates in clinical trials. Its lead drug, which targets acute lymphoblastic lymphoma, a rare blood cancer, could be on the market as early as next year.
AbVitro uses technology developed at the Harvard lab of geneticist George Church. It enables researchers to scan millions of cells in a few hours and identify the rare but important receptors that bind to cancer cells and kill them. "It's so integral to the rest of our research program that we really need them at our main lab" in Seattle, said Bishop.
He said the Boston area is "the most flourishing scientific hub in the world." He said Juno will continue to scout for smaller research companies in Massachusetts and elsewhere "that have the technology to make us a better company."