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To develop therapeutics for rare diseases
January 13, 2014
By: Kristin Brooks
Managing Editor, Contract Pharma
Alexion Pharmaceuticals and Moderna Therapeutics have entered an exclusive strategic agreement for the discovery and development of messenger RNA Therapeutics to treat rare diseases. Messenger RNA Therapeutics are designed to use the body’s natural processes to enable the in vivo production of both intracellular proteins and secreted proteins, which act to restore function elsewhere in the body. The mRNA Therapeutics platform has the potential to speed the development and manufacture of treatments for many rare diseases, where existing technologies fail. Alexion will make an upfront payment of $100 million to Moderna to purchase 10 product options to develop and commercialize treatments with Moderna’s mRNA Therapeutics platform. Alexion will lead the discovery, development and commercialization of any treatments produced and Moderna will retain responsibility for the design and manufacture of the messenger RNA against selected targets. If Alexion exercises its options, Moderna will be entitled to development and commercial milestone payments, as well as royalties on sales. Additionally, Alexion has made a $25 million investment in 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., chief executive officer of Alexion Pharmaceuticals. “We consider Moderna’s mRNA Therapeutics to be an especially exciting drug discovery platform, and we look forward to combining Moderna’s deep expertise in mRNA with Alexion’s significant global resources and expertise in drug discovery, clinical studies, regulatory affairs and commercialization for the benefit of patients worldwide.” “Moderna’s strategy is to aggressively seek to commercialize its mRNA platform for patients in need,” said Stéphane Bancel, president and chief executive officer 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. As we were looking for a global leader with a proven track record in the discovery, development and global commercialization of treatments for patients with rare diseases, entering into a strategic agreement with Alexion became an obvious choice for us. We are very pleased and honored to work with such a dedicated company to bring new medicines to patients.”
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