Biotech upstart Moderna nails down $40M for bold RNA idea

Moderna Therapeutics wants to treat patients with therapeutic proteins made in their own bodies rather than in expensive biomanufacturing plants, with new RNA drugs made to trigger production of therapies in the cells of people. The secretive startup has attracted $40 million from Flagship Ventures and other investors to put the bold plan into action, disclosing the financing and details about its research to the public for the first time this morning.

The Cambridge, MA-based company claims its approach is the first entirely new way of producing protein drugs in more than 30 years. Its drugs are designed to manipulate the natural protein-making machinery in cells to produce therapeutics that either stay in cells to combat disease or slip into the bloodstream to battle ailments.

Today, industrial-scale bioreactors produce protein drugs such as insulin and antibody meds that generate billions of dollars in sales. Those therapeutics and the processes to develop and manufacture them are notoriously expensive. While it's far too early to write off traditional protein production, Moderna's messenger RNA (mRNA)-based drugs could offer a potential alternative. And Moderna CEO Stéphane Bancel left his big job as chief executive of the growing diagnostics company bioMérieux last year to run the startup because of the huge promise he saw in its technology.

"Nobody has been able to make therapeutic proteins in [humans] before," Bancel told FierceBiotech in an interview.

In addition to Bancel, Moderna boasts prolific MIT inventor Robert Langer among its founders. Its technology was built from the early work of Harvard stem cell expert and Moderna co-founder Derrick Rossi, who gained notice in 2010 for using mRNA in a process to reprogram adult cells into induced pluripotent stem (iPS) cells.

Rather than focus on the iPS cell application of the technology, Moderna has spent the past year and a half developing the mRNA-based approach to alter the way protein therapeutics are made and delivered to patients.

Messenger RNA molecules play a key role in the production of proteins in cells. In labs, Moderna crafts mRNA molecules with sequences for specific proteins and injects them into subjects. The injected molecules travel to cells, where ribosomes translate the information from the mRNA with the natural machinery of protein synthesis to generate therapeutics.

Moderna's approach could produce thousands of proteins, Bancel says. The company has chosen to focus on cancer supportive care and treatments for rare inherited diseases, hemophilia and diabetes. So far the approach has been tested in animals as large as monkeys, and Bancel believes that human studies could come "soon" although he isn't saying when exactly. 

Investors are keen on the plan. When I asked him to name hot biotechs in his portfolio earlier this year, Flagship Ventures' CEO Noubar Afeyan highlighted Moderna. He's serving as the young company's chairman and making good on his word to keep funding bold ideas with the potential to impact healthcare.

- here's the release
- see Xconomy's article

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