Article
Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double-blind, placebo-controlled phase 1/2 clinical study.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jul 2023
Rowe S M, Zuckerman J B, Dorgan D, Lascano J, McCoy K, Jain M, Schechter M S, Lommatzsch S, Indihar V, Lechtzin N, McBennett K, Callison C, Brown C, Liou T G, MacDonald K D, Nasr S Z, Bodie S, Vaughn M, Meltzer E B, Barbier A J
Abstract excerpt
BACKGROUND: MRT5005, a codon-optimized CFTR mRNA, delivered by aerosol in lipid nanoparticles, was designed as a genotype-agnostic treatment for CF lung disease. METHODS: This was a randomized, double-blind, placebo-controlled Phase 1/2 study performed in the US. Adults with 2 severe class I and/or II CFTR mutations and baseline ppFEV1 values between 50 and 90% were randomized 3:1 (MRT5005: placebo). Six dose...
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