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RTX0001

Development stage: Preclinical
Lead developer: ReCode Therapeutics
Modality: mRNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration: Inhalation

Overview

RTX0001 is an inhaled mRNA replacement therapy in preclinical development by ReCode Therapeutics for cystic fibrosis and potentially other diseases caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It consists of a chemically optimized CFTR messenger RNA encoding a functional CFTR protein, formulated for delivery to airway epithelial cells, where translation of the exogenous mRNA is intended to restore CFTR channel activity, normalize ion and fluid transport, and improve mucociliary clearance independent of a patient’s specific CFTR mutation.[7][13][15]

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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