Date: 2016-07-06
Type of information: R&D agreement
Compound: messenger Ribonucleic Acid (mRNA) Therapeutics™
Company: Moderna Therapeutics (USA - MA) Vertex Pharmaceuticals (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: R&D licensing
Action mechanism: Messenger RNA (mRNA). Messenger RNA therapeutics™ are a new treatment approach that enables the body to produce therapeutic protein in vivo, opening up new treatment options for a wide range of diseases that cannot be addressed today using existing technologies.
Disease: cystic fibrosis
Details: * On July 6, 2016, Vertex Pharmaceuticals and Moderna Therapeutics have entered into an exclusive research collaboration and licensing agreement aimed at the discovery and development of messenger Ribonucleic Acid (mRNA) Therapeutics™ for the treatment of cystic fibrosis. The three-year collaboration will focus on the use of mRNA therapies to treat the underlying cause of cystic fibrosis by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is known to be defective in people with cystic fibrosis. Through the collaboration, the companies will explore the potential utilization of pulmonary mRNA delivery.
Under the terms of the collaboration, Vertex and Moderna will conduct exclusive research, development and commercialization activities to advance mRNA Therapeutics that aim to enable cells of people with cystic fibrosis to produce functional CFTR proteins in the lungs. Moderna will lead discovery efforts, leveraging its leading mRNA platform technology and mRNA delivery expertise along with Vertex’s scientific experience in cystic fibrosis biology and the functional understanding of CFTR, as well as the company’s proprietary assay platform that utilizes human bronchial epithelial (HBE) cells of multiple different CF gene mutations from people with CF. Vertex will lead all preclinical, development and commercialization activities associated with the advancement of mRNA Therapeutics that result from this collaboration and will fund all expenses related to the collaboration.
Financial terms: Vertex will pay Moderna $20 million in cash as part of its upfront commitment to the collaboration. Vertex will also make a $20 million investment in Moderna in the form of a convertible note that will convert to equity. The investment will provide Vertex with an ownership stake in Moderna. Vertex will also pay Moderna future development and regulatory milestones of up to $275 million, including $220 million in approval and reimbursement milestones, as well as tiered royalty payments on future sales.
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