Vertex and Moderna collaborate to develop mRNA Therapeutics for cystic fibrosis

Moderna will receive $40 million upfront, with potential for an additional $275m in milestones and royalty payments

Vertex Pharmaceuticals, a US developer of treatments for rare diseases based in Boston, has entered into an exclusive research collaboration with Moderna Therapeutics aimed at the discovery and development of messenger Ribonucleic Acid (mRNA) therapeutics for the treatment of cystic fibrosis (CF).

Vertex will pay Moderna $20 million in cash upfront and will also make a $20m investment in the Cambridge, MA-based company in the form of a convertible note that will convert to equity. With this investment Vertex will have an ownership stake in Moderna.

Vertex will also pay Moderna future development and regulatory milestones of up to $275m, including $220m in approval and reimbursement milestones, as well as tiered royalty payments on future sales.

The three-year collaboration will focus on the use of 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, which is known to be defective in people with CF. Through the collaboration, the companies will explore the potential use of pulmonary mRNA delivery.

Moderna's messenger RNA Therapeutics represent a promising new approach to treating cystic fibrosis

'We are excited to begin this collaboration with Moderna to further broaden our pipeline and support our goal of treating the underlying cause of CF for all people with this disease,' said David Altshuler, Vertex's Executive Vice President, Global Research and Chief Scientific Officer.

'The collaboration underscores our commitment to invest in and pioneer multiple innovative approaches to expand and enhance the future treatment of CF.'

He added: 'Moderna's messenger RNA 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.'

'Vertex's deep scientific expertise and commitment to creating the best treatment regimens for cystic fibrosis patients make them an ideal partner to advance mRNA Therapeutics for this disease,' said St├ęphane Bancel, Chief Executive of Moderna.

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

Vertex will lead all preclinical, development and commercialisation activities associated with the advancement of mRNA therapeutics that result from this collaboration and will fund all expenses related to the collaboration.

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