MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS

The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleot...

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Bibliographische Detailangaben
Hauptverfasser: Uhlmann, Eugen, Weber, Markus, Krieg, Arthur M, Rockel, Thomas Dino, Subramanian, Romesh R
Format: Patent
Sprache:eng
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Zusammenfassung:The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided