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Moderna highlights partnership with Vertex on inhaled mRNA therapy for CF

According to Moderna Therapeutics, the company used its annual “Science and Technology Day” to highlight its work on lipid nanoparticle (LNP) mRNA formulations, and CEO Stéphane Bancel made special note of the company’s advances in pulmonary delivery, including a partnership with Vertex on development of an inhaled mRNA formulation that could give CF patients the ability to make functioning cystic fibrosis transmembrane conductance regulator (CFTR) protein.

The company said, “Moderna researchers have developed a new LNP formulation that addresses many of the challenges associated with lung delivery of mRNA. This pulmonary LNP formulation is delivered by inhalation, uses multiple lipids, and is able to target specific areas of the lung. Moderna’s optimization of pulmonary delivery of mRNA provides opportunities to address a range of unmet medical needs.”

According to the Vertex web site, the mRNA formulation is intended for the 10% of CF patients who make no CFTR protein at all and therefore are not helped by CFTR modulators such as ivacaftor. Moderna said that IND studies have been completed successfully and that Vertex expects to submit an IND for the inhaled formulation later this year.

Read the Moderna Therapeutics press release.

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published on May 19, 2022

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