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Company ProQR Therapeutics N.V.
DescriptionRNA-based oligonucleotide
Molecular Target Cystic fibrosis transmembrane conductance regulator (CFTR)
Mechanism of ActionAntisense therapy
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentPhase I
Standard IndicationCystic fibrosis (CF)
Indication DetailsTreat cystic fibrosis (CF)
Regulatory Designation U.S. - Fast Track (Treat cystic fibrosis (CF));
U.S. - Orphan Drug (Treat cystic fibrosis (CF));
EU - Orphan Drug (Treat cystic fibrosis (CF))

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