Nickase-retron template-based precision editing system and methods of use

The chimeric gene editing system, integrating prime and retron editor components, addresses the limitations of current gene editing tools by providing enhanced precision and efficiency through the use of modified retron ncRNAs and msDNAs as polymerase templates.

WO2025081042A1 Publication Date: 2025-04-17RENAGADE THERAPEUTICS MANAGEMENT INC
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Patent Information

Application Number
PCT/US2024/051048
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-06-06
Filing Date
2024-10-11
Publication Date
2025-04-17

AI Technical Summary

Technical Problem

Current gene editing tools, despite advancements, lack the precision and efficiency needed for certain genetic alterations, particularly in achieving targeted nucleic acid edits, insertions, replacements, and deletions.

Method used

A chimeric gene editing system combining components of a prime editor and a retron editor, utilizing a nickase component, guide RNA, polymerase component, and a polymerase template sequence provided by a modified retron ncRNA, to enhance editing specificity and efficiency.

Benefits of technology

The chimeric system achieves precise and efficient genome editing by utilizing a templated ncRNA and/or msDNA as a polymerase template, allowing for permanent installation of edits into the genome.

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Abstract

Disclosed herein relates to a chimeric gene editing system comprising a modified retron editing system for precise and efficient gene editing. Additionally, a chimeric gene editing system that comprises one or more components of a prime editing system (or a prime editor-like system) with one or more components of a retron editing system, is disclosed, providing an entirely new gene editing system with distinct properties, capabilities and advantages.
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