Moderna Inc (MRNA)

[axelvento]

Inhaled pulmonary therapeutics

Cystic fibrosis (CF)

Moderna's collaborator, Vertex Pharmaceuticals, announced that the U.S. Food and Drug Administration cleared its Investigational New Drug (IND) application for VX-522, an mRNA targeted at treating the underlying cause of cystic fibrosis for the approximately 5,000 people with CF who cannot benefit from a cystic fibrosis transmembrane conductance regulator (CFTR) modulator
VX-522 is delivered to the lung through inhalation of a CFTR mRNA encapsulated by a lipid nanoparticle. Once delivered to the target lung cells, the mRNA is designed to produce functional copies of the CFTR protein

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