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CAG gapmers

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Company Ionis Pharmaceuticals Inc.
DescriptionChimeric antisense oligonucleotides containing modified nucleic acid residues that induce RNase H-mediated degradation of CUG repeats
Molecular Target
Mechanism of Action 
Therapeutic ModalityNucleic acid: Linear RNA: Antisense
Latest Stage of DevelopmentPreclinical
Standard IndicationMyotonic dystrophy type 1 (DM1)
Indication DetailsTreat myotonic dystrophy type 1 (DM1)
Regulatory Designation
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