Oligonucleotide compositions and methods thereof

WO2026096689A8PCT designated stage Publication Date: 2026-06-11WAVE LIFE SCI LTD +7

Patent Information

Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
WAVE LIFE SCI LTD
Filing Date
2025-10-29
Publication Date
2026-06-11

AI Technical Summary

Technical Problem

Existing oligonucleotide technologies face challenges in efficiently editing nucleic acids, particularly in converting adenosine to inosine, with issues related to stability, selectivity, cellular uptake, immune stimulation, and toxicity, especially when utilizing exogenous components.

Method used

Designing oligonucleotides with specific sugar modifications, nucleobase modifications, and internucleotidic linkages, such as 2'-F and 2'-OR modifications, along with chirally controlled internucleotidic linkages, to enhance editing efficiency and selectivity, stability, and reduce immune stimulation and toxicity.

Benefits of technology

The designed oligonucleotides demonstrate improved stability, selectivity, and reduced toxicity, enabling efficient adenosine-to-inosine conversion in nucleic acids, particularly in mRNA, utilizing endogenous ADAR proteins for targeted editing.

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Abstract

Among other things, the present disclosure provides oligonucleotide compositions and methods thereof. In some embodiments, the present disclosure provides oligonucleotide compositions that can edit an adenosine in a LDLR transcript. In some embodiments, the present disclosure provides methods for upregulating LDLR expression. In some embodiments, the present disclosure provides methods for preventing or treating conditions, disorders or diseases, e.g., those that can benefit from editing of an adenosine in a LDLR transcript or higher LDLR expression.
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