Translation enhancing nucleic acid compounds for PKD1 and uses thereof

WO2026117724A3PCT designated stage Publication Date: 2026-07-09ARNATAR THERAPEUTICS INC

Patent Information

Authority / Receiving Office: WO · WO
Patent Type: Applications
Current Assignee / Owner: ARNATAR THERAPEUTICS INC
Filing Date: 2025-11-26
Publication Date: 2026-07-09

AI Technical Summary

Technical Problem

Current treatments for Autosomal Dominant Polycystic Kidney Disease (ADPKD) focus on symptom management and slowing disease progression, lacking a cure, and existing antisense oligonucleotides (ASOs) face off-target concerns due to non-specific miRNA modulation, necessitating more precise and effective approaches to increase PKD1 protein levels.

Method used

Development of antisense oligonucleotides using ACT-UP1 technology to specifically enhance translation of PKD1 mRNA, increasing PKD1 protein levels, thereby treating ADPKD and Polycystic Liver Disease (PLD).

Benefits of technology

The ACT-UP1 compounds effectively increase PKD1 protein levels in cells and animal models, reducing cyst formation and potentially reversing cyst-related diseases by targeting PKD1 mRNA translation with minimal off-target effects.

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Abstract

Disclosed herein are methods and compounds for enhancing PKD1 protein expression by compounds targeting PKD1 mRNA. Such methods and compounds are useful for increasing expression of in PKD1 mutation patients for the treatment of a haploinsufficiency and / or loss of function disease, disorder and / or condition such as Autosomal Dominant Polycystic Kidney Disease (ADPKD) and / or Polycystic Liver Disease (PLD).

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