Chemically modified antisense oligonucleotides (ASOS) and compositions for RNA editing

Chemically modified ASOs with specific linkages and nucleosides improve A-to-G RNA editing precision and efficiency, addressing the limitations of existing therapeutics for genetic disorders.

WO2026117623A1PCT designated stage Publication Date: 2026-06-04AIRNA CORPORATION
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Patent Information

Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
AIRNA CORPORATION
Filing Date
2025-11-26
Publication Date
2026-06-04

AI Technical Summary

Technical Problem

Existing oligonucleotide therapeutics for RNA editing, particularly A-to-I editing, lack the precision and efficiency needed for effective site-directed RNA editing, especially in targeting adenosine to guanosine conversions, which are crucial for therapeutic applications in genetic disorders.

Method used

Development of chemically modified antisense oligonucleotides (ASOs) with specific modifications, including phosphorothioate linkages and 2'-O-methyl nucleosides, to enhance binding and editing efficiency, utilizing endogenous adenosine deaminase acting on RNA (ADAR) for precise A-to-G conversions.

Benefits of technology

The modified ASOs demonstrate enhanced editing efficacy and safety, enabling targeted RNA editing in cells, with potential therapeutic benefits for diseases associated with gain-of-function or loss-of-function mutations.

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Abstract

The present disclosure relates to a chemically modified oligonucleotide for use in site-directed A-to-l editing of a target RNA inside a cell with endogenous adenosine deaminase acting on RNA (ADAR), the oligonucleotide comprising a sequence capable of binding to a target sequence in a target RNA and a central base triplet (CBT) of 3 nucleotides (5' - N+1 N0 N-1 - 3'), wherein N0 is the central nucleotide directly opposite to a target adenosine in the target RNA that is to be edited, and wherein the oligonucleotide comprises an asymmetry of 25-1-8 in a 5' to 3' direction; and comprises: (i) one or more phosphorothioate (PS) linkages at one or more of position +22, position +21, position +11, and position +4; and / or (ii) a 2'-O-methyl (2'-O-Me) modified nucleosides at position +13 and / or position +9.
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