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Company Touchstone Innovations plc
DescriptionNebulized plasmid DNA encoding the cystic fibrosis transmembrane conductance regulator (CFTR) gene complexed with a cationic liposome
Molecular Target Cystic fibrosis transmembrane conductance regulator (CFTR)
Mechanism of ActionGene therapy
Therapeutic ModalityGene therapy: Non-viral vector
Latest Stage of DevelopmentPhase II
Standard IndicationCystic fibrosis (CF)
Indication DetailsTreat cystic fibrosis (CF)
Regulatory Designation U.S. - Orphan Drug (Treat cystic fibrosis (CF));
EU - Orphan Drug (Treat cystic fibrosis (CF))

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