While biopharma executives and investors were wheeling and dealing in San Francisco at the annual J.P. Morgan Healthcare Conference, Moderna Therapeutics struck a potentially lucrative deal with Alexion Pharmaceuticals focused on rare diseases and launched Onkaido Therapeutics to take over its efforts in oncology. Both deals revolve around Moderna’s messenger RNA therapeutics platform to reach previously undruggable targets.

Messenger RNA therapeutics offer patients a new treatment approach, helping their bodies produce a therapeutic protein in vivo. The technique provides a new way to treat a wide range of diseases that cannot be addressed today using existing technologies. Moderna’s proprietary messenger RNA are designed to stimulate the body’s natural ability to produce intra-cellular and secreted therapeutic proteins without triggering an innate immune response. The secreted proteins are released into the bloodstream to potentially restore function elsewhere in the body. Enabling the body to produce a therapeutic progein in vivo, says Moderna, opens up new treatment options for a wide range of diseases that are undruggable using existing technologies. The company says using messenger RNA could also potentially reduce the time and expense associated with creating therapeutic proteins using current recombinant technologies.

“Moderna’s strategy is to aggressively seek to commercialize its mRNA platform for patients in need,” says Stéphane Bancel, president and founding CEO of Moderna. “Given the broad potential of this new drug modality, we wanted to forge a few long-term strategic relationships with best-in-class companies.”

The deal with Alexion Pharmaceuticals is focused on rare disease therapeutics. Under the agreement, Alexion will pay Moderna $100 million upfront to purchase 10 product options to develop and commercialize treatments for rare diseases with Moderna’s mRNA therapeutics platform. Alexion will lead the discovery, development and commercialization of the treatments produced through the broad, long-term strategic agreement, while Moderna will retain responsibility for the design and manufacture of the messenger RNA against selected targets. If Alexion exercises its option to license, Moderna will be eligible for drug development and commercial milestone payments, as well as royalties. Alexion also made a $25 million preferred equity investment in Moderna.

Cambridge, Massachusetts-based Moderna also launched a new company, Onkaido Therapeutics, to focus on developing mRNA-based cancer treatments and is committing $20 million to the new venture. The new company will focus initially on 15 preclinical drug candidates in the areas of apoptosis, central regulatory nodes and immunotherapy. Some of these candidates are already under evaluation in in vivo disease models. Moderna expects that establishing Onkaido as a standalone unit will accelerate the development of mRNA-based cancer drugs.

“The vast potential for messenger RNA Therapeutics will only be tapped by simultaneously industrializing our platform and developing our key programs in the clinic as quickly and safely as possible,” says Bancel.

In March 2013, AstraZeneca paid Moderna $240 million upfront to gaine exclusive access to select any target of its choice in cardio-metabolic diseases, as well as selected targets in oncology, over a period of up to five years for subsequent development of messenger RNA by Moderna with the option to select up to 40 drug products for clinical development.