Rare disease-focused firm Alexion Pharmaceuticals has inked a long-term agreement with Moderna Therapeutics toward discovery and development of messenger RNA (mRNA) Therapeutics™ for treating rare diseases.

Alexion has made a $25 million preferred equity investment into Moderna and will, per the agreement, make a $100 million upfront payment to Moderna, purchasing 10 product options to develop and commercialize treatments for rare diseases using the mRNA Therapeutics platform Moderna developed. Alexion says it will lead the discovery, development, and commercialization of any new treatments produced through this agreement. Meanwhile, Moderna will be responsible for designing and manufacturing the messenger RNA against selected targets and will also be entitled to commercial and drug development milestone payments, as well as high single- to double-digit royalties on commercial sales. 

Messenger RNA Therapeutics, according to Moderna, enable 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. The firm believes the mRNA Therapeutics platform could speed up the development and manufacture of treatments for many currently untreatable rare diseases.

“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,” said Alexion’s CEO Leonard Bell, M.D., in a statement.

Back in March, Moderna’s mRNA Therapeutics platform also caught the eye of AstraZeneca, which made a multi-year deal with the firm—one that could net Moderna more than $420 million—to use the technology to develop and commercialize new drugs for cancer and “serious” cardiovascular, metabolic, and renal diseases. DARPA also took an interest in the platform back in October, awarding Moderna up to $25 million to research and develop the platform as a way to make antibody-producing drugs to protect against emerging infectious diseases and engineered biological threats.

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