Oligonucleotides containing multiple ligands and / or lipids

EP4762069A1Pending Publication Date: 2026-06-24ADARX PHARMACEUTICALS INC
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Patent Information

Authority / Receiving Office
EP · EP
Patent Type
Applications
Current Assignee / Owner
ADARX PHARMACEUTICALS INC
Filing Date
2024-08-16
Publication Date
2026-06-24

AI Technical Summary

Technical Problem

There is a need for new compounds that can efficiently deliver pharmaceutical agents to specific locations within a subject, such as the brain, to enhance therapeutic or prophylactic effects while minimizing unintended consequences like off-target effects.

Method used

The development of oligonucleotides comprising two or more radicals of ligands and/or lipids conjugated at one or more positions on the oligonucleotide strand, allowing for targeted delivery of pharmaceutical agents to specific cells or tissues.

Benefits of technology

The oligonucleotides demonstrate higher potency, efficacy, bioavailability, safety, and subject compliance, with a wider therapeutic window, fewer side effects, and lower toxicity compared to traditional oligonucleotides without ligand or lipid conjugation.

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Abstract

The present disclosure provides oligonucleotides comprising two or more radicals of ligands and / or lipids conjugated at at least one position on the oligonucleotide. The present disclosure also provides pharmaceutical compositions and kits comprising said oligonucleotides, methods of delivering said oligonucleotides, methods of modulating the activity of a protein using such oligonucleotides, and methods of treating, preventing, or diagnosing diseases (e.g., central nervous system diseases, neurodegenerative diseases, and neurocognitive diseases) in a subject in need thereof using said oligonucleotides or pharmaceutical compositions thereof.
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