Moderna Therapeutics - 2013 Fierce 15

Moderna Therapeutics
Moderna turns cells in patients into drug factories

Based: Cambridge, MA
Founded: 2010
CEO: Stéphane Bancel
Clinical focus: Messenger RNA therapies

The scoop: Moderna Therapeutics wins the 2013 award for the most impressive alliance deal on preclinical science, snapping up $240 million in upfront cash from London-based drug giant AstraZeneca ($AZN) to fuel discovery of messenger RNA therapies. Based on initial research from the lab of Harvard stem cell expert Derrick Rossi and others, Moderna's messenger RNA therapies aim to turn the body's own cells into makers of protein drugs for a wide range of diseases. This could drastically reduce the cost of developing protein therapies and offer new treatment options for many rare disorders and common ailments such as cancer and heart disease. Flagship Ventures looks smart for incubating Moderna, now led by founding CEO Stéphane Bancel, the former chief executive for the French diagnostics power bioMérieux.

What makes Moderna fierce: Moderna aims to revolutionize the way protein therapies are produced and developed, potentially shaving years and millions of dollars off of these difficult endeavors.

Today biologics are made in large bioreactors with hundreds or thousands of liters of volume capacity, and the process of scaling up can take more than a year. Moderna, on the other hand, can churn out enough supply of its modified messenger RNA compounds for a Phase I clinical trial in a half-liter bioreactor. Picture something around the size of a coffee pot.

Bancel, a veteran of biologics manufacturing at Eli Lilly ($LLY), says the process from the half-liter reactor easily scales up to supply medicines for larger trials and eventual commercialization. Moderna makes synthetic messenger RNAs in a recombinant process, without the hassles and complexity of cell-based production methods used to make most biologics.

Rather than making medicines with mammalian cells in huge reactors, Moderna relies on the proteinmaking machinery within the cells, including the ribosomes, to produce therapeutic agents. Turning the human body into its own drug factory, which the startup has not done yet, would be revolutionary. Many top industry people have joined Moderna in some capacity to take part in this journey.  

"I was attracted to that notion that felt very much like the Genzyme feel many years ago," said Henri Termeer, the legendary former Genzyme CEO and chairman, who joined Moderna as a member of its board of directors in April.  

Moderna has followed Termeer's lead with research of rare-disease therapies. Cancer and rare diseases are the two initial focuses of internal R&D at the company, and Joseph Bolen recently joined to head R&D after a stint as chief scientific officer at the cancer drug specialist Millennium Pharmaceuticals.

The startup has opened the platform to partners for many potential therapeutic uses. AstraZeneca, for example, has dibs on all the cardio drug targets from Moderna for 5 years from the major R&D alliance formed between the two companies in March. Pascal Soriot, who became CEO of AstraZeneca last year, endorsed the gamble on Moderna as the former Roche ($RHHBY) executive seeks such innovation to bolster the scientific reputation of the drugmaker.

Moderna took in $240 million in cash from AstraZeneca at the start of the collaboration, which includes exclusive rights to up to 40 candidates in cardiometabolic, metabolic, and kidney diseases and certain cancers for 5 years. And AstraZeneca agreed to pay $180 million more if Moderna reaches three technical milestones.

Moderna scientists this month published the company's first scientific article in Nature Biotechnology, showing that one of the company's mRNA therapies helped regenerate heart tissue in disease models. The company faces years of additional lab work before reporting any such results in humans. Yet Bancel believes his company has the talent and funding to succeed. 

"I'll take my chances on Moderna any day," Bancel says, "because with cash and finance you can do amazing things."

Flagship Ventures was the lead investor in $40 million in financing revealed in December for the startup, which was incubated in Flagship's startup program in Cambridge, MA, before moving to its own labs.

At the time of the AstraZeneca deal in March, Bancel was in charge of a 25-person operation at Moderna. The company had hit 75 staff members as of August, and the CEO expects its workforce to reach 100 by the end of 2013.

Lead investor: Flagship Ventures.

For more:
Biotech legend Termeer joins the board at Moderna
Biotech upstart Moderna nails down $40M for bold RNA idea

-- Ryan McBride

Moderna Therapeutics - 2013 Fierce 15