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July 6, 2016

Vertex, Moderna Launch Cystic Fibrosis Collaboration

  • Vertex Pharmaceuticals and Moderna Therapeutics said today they will work to discover and develop new cystic fibrosis (CF) treatments based on Moderna’s class of messenger Ribonucleic Acid (mRNA) Therapeutics™.

    Moderna could generate up to $315 million through the 3-year collaboration, which will focus on using mRNA therapies to treat the underlying cause of CF by enabling cells in the lungs to produce functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein.

    “Moderna's mRNA Therapeutics represent a promising new approach that could provide functional CFTR proteins to the lungs of people with CF, which would complement our existing approach of using CFTR modulators to treat CF,” David Altshuler, M.D., Ph.D., Vertex's evp, global research and CSO, said in a statement.

    Vertex and Moderna said they agreed to conduct exclusive research, development, and commercialization activities aimed at advancing mRNA Therapeutics desgined to enable cells of people with CF to produce functional CFTR proteins in the lungs.

    “We're particularly excited to explore the potential of pulmonary delivery as a new modality to bring transformative mRNA medicines to patients and address unmet needs,” added Moderna CEO Stéphane Bancel.

    Moderna will lead discovery efforts, using its mRNA platform technology and mRNA delivery expertise along with Vertex's scientific experience in CF biology and the functional understanding of CFTR and the company’s assay platform that uses human bronchial epithelial) cells of multiple different CF gene mutations from people with CF.

    Vertex has agreed to lead all preclinical, development, and commercialization activities associated with the advancement of mRNA Therapeutics resulting from the collaboration, and will fund all expenses related to the collaboration, the companies said.

    Vertex agreed to pay Moderna $20 million cash upfront, and will take an ownership stake in Moderna by investing $20 million through a convertible note that will convert to equity. Vertex will also pay Moderna up to $275 million in payments tied to achieving development and regulatory milestones—including $220 million in approval and reimbursement milestones—as well as tiered royalties on future sales.

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