Methods of delivering multiple targeting oligonucleotides to a cell using cleavable linkers

US9732341B2 · kind B2 · utility

Assignee

• Translate Bio MA, Inc. · US

Inventors

• Eugen Uhlmann · Königstein, DE
• Markus Weber · Langenfeld, DE
• Romesh R. Subramanian · Framingham, US
• Thomas Rockel · Mülheim an der Ruhr, DE
• Arthur M. Krieg · Wellesley, US

Key dates

• Filing date: Apr 28, 2015
• Grant date: Aug 15, 2017
• Expiry date: Apr 28, 2035

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2310/52
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

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.