Vertex, Moderna Collaborate to Discover, Develop mRNA Therapeutics for CF

Daniela Semedo, PhD avatar

by Daniela Semedo, PhD |

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CF research, discovery collaboration

Vertex Pharmaceuticals and Moderna Therapeutics recently entered a three-year research partnership and licensing agreement to discover and develop the messenger ribonucleic acid, trademarked as (mRNA) Therapeutics, for the treatment of cystic fibrosis (CF).

The alliance between the two companies will center on usimg mRNA drugs to treat the CF  at its core, by enabling lung cells to generate functional copies of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is defective in patients with CF. Through the partnership, the two companies will investigate the possible use of pulmonary mRNA delivery.

“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. The collaboration with Moderna underscores our commitment to invest in and pioneer multiple innovative approaches to expand and enhance the future treatment of CF,” said Dr. David Altshuler, Vertex’s executive vice president of Global Research and chief scientific officer in a press release. “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.”

Stéphane Bancel, chief executive officer of Moderna said Vertex’s deep scientific expertise and commitment to creating the best treatment regimens for cystic fibrosis patients make them an ideal partner.

“This collaboration is illustrative of our consistent strategy to partner with world class therapeutic experts in order to maximize the potential of leveraging mRNA as a novel therapeutic approach across a spectrum of serious diseases. 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,” Bancel said.

Under the terms of the alliance, both companies will conduct research, development and commercialization actions to advance mRNA Therapeutics for patients with CF.

Moderna will be responsible for the discovery of molecules leveraging its mRNA platform and mRNA delivery know-how with Vertex’s scientific knowledge in CF biology and the functional understanding of CFTR, and its proprietary platform of human bronchial epithelial (HBE) cells of multiple different CF gene mutations from patients with the disease.

Vertex will conduct preclinical trials, as well as development and commercialization actions related to the advance of mRNA Therapeutics through the partnership. The company is funding all expenses.

Moderna will receive $20 million upfront from Vertex, and a $20 million convertible note investment that will convert to equity. Vertex will also pay up to $275 million to Moderna for future development and regulatory milestones. The deal includes $220 million to Moderna in approval, reimbursement milestones, and tiered royalty expenditures on future sales.