Alexion Pharmaceuticals and Moderna Therapeutics have reached an exclusive strategic agreement for the discovery and development of messenger RNA Therapeutics to treat rare diseases.

Alexion is a global provider in the development and commercialization of breakthrough therapies for patients with severe and life-threatening rare diseases. Moderna is a pioneer in developing messenger RNA (mRNA) Therapeutics, an innovative treatment modality to enable the in vivoproduction of therapeutic proteins.

Messenger RNA Therapeutics are designed to directly utilize the body’s natural processes to enable the in vivo production of both intracellular proteins, which remain within the cells, and secreted proteins, which are released into the bloodstream and act to restore function elsewhere in the body. As a result, the mRNA Therapeutics platform has the potential to speed the development and manufacture of treatments for many rare diseases that currently are untreatable with existing technologies.

Alexion will make an upfront payment to Moderna of $100 million 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 this broad, long-term strategic agreement, while Moderna will retain responsibility for the design and manufacture of the messenger RNA against selected targets. Moderna will be entitled to drug development and commercial milestone payments, as well as high single- to double-digit royalties on commercial sales. In addition, Alexion has made a $25 million preferred equity investment into Moderna.

“This broad and long-term strategic agreement represents a significant expansion of Alexion’s capabilities for drug discovery, and provides us with the strong potential to further our mission of developing novel and breakthrough therapies for patients with severe and life-threatening rare diseases,” said Leonard Bell, M.D., CEO of Alexion Pharmaceuticals.