Moderna and Vertex Establish New Collaboration to Treat Cystic Fibrosis Using Gene Editing

Moderna, Inc. (Nasdaq:MRNA) and Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) announced today a new strategic research collaboration and licensing agreement aimed at the discovery and development of lipid nanoparticles (LNPs) and mRNAs for the delivery of gene-editing therapies for the treatment of cystic fibrosis (CF). The three-year research collaboration initially will focus on the discovery and optimization of novel LNPs and mRNAs that can deliver gene-editing therapies to cells in the lungs, enabling functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to be produced.

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“We are pleased to enter into this second collaboration with Vertex aimed at delivering potentially novel treatments for patients with cystic fibrosis using gene editing,” said Stéphane Bancel, Chief Executive Officer of Moderna. “Our first collaboration with Vertex to deliver mRNA coding for cystic fibrosis protein in lung cells is advancing well and this second collaboration aims at using Moderna’s technologies to explore the use of gene editing in lung cells.”

"Vertex’s CFTR modulator therapies have the potential to treat the vast majority of CF patients and address the underlying cause of their disease. However, approximately 10 percent of patients do not produce any CFTR protein and so are unlikely to benefit from our existing medicines. Over the past 5 years, we have made important progress in our research efforts aimed at the creation of genetic therapies for CF, with the delivery of such therapies remaining the most significant technological and scientific challenge,” said David Altshuler, M.D., Ph.D., Vertex's Executive Vice President, Global Research and Chief Scientific Officer. “The combination of Moderna’s unique expertise in the discovery and manufacturing of novel LNP delivery systems and mRNA technologies, combined with Vertex’s scientific, clinical and regulatory capabilities in CF, will accelerate the development of groundbreaking genetic therapies for people with CF and supports our commitment to developing therapies for all people living with CF.”