Methods for modulating expression of complex gene networks

By inserting a synthetic miRNA sequence into a donor locus under the control of activating transcriptional elements and disrupting endogenous disease-promoting sequences, the method achieves precise modulation of gene networks, enhancing therapeutic efficacy against diseases like cancer.

WO2026088194A1PCT designated stage Publication Date: 2026-04-30LEPTON PHARM LTD
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Patent Information

Application Number
PCT/IL2025/050941
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-10-25
Filing Date
2025-10-26
Publication Date
2026-04-30

AI Technical Summary

Technical Problem

Existing gene therapy strategies struggle to precisely modulate complex gene networks, often leading to off-target effects, incomplete silencing, or excessive expression, which disrupt cellular homeostasis, and lack effective methods for physiologically relevant miRNA expression control.

Method used

A method involving the insertion of a synthetic sequence encoding a therapeutic genetic element, such as a miRNA, into a donor locus under the control of transcriptional elements that activate in response to internal or environmental signals, while disrupting the expression of endogenous RNA-encoding sequences contributing to disease, using CRISPR/Cas technology to achieve precise regulation.

Benefits of technology

Enhances therapeutic efficacy by precisely modulating gene networks, improving cell therapies by enhancing therapeutic miRNA expression in response to pathological signals, thereby inhibiting disease progression and therapy resistance.

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Abstract

Described herein are methods for modulating the expression of complex gene networks by increasing expression of an RNA-encoding therapeutic genetic element that is normally attenuated in response to an internal or environmental signal while simultaneously disrupting expression of an endogenous RNA-encoding sequence that is normally activated in response to an internal or environmental signal. Methods for enhancing cell therapies and treatment of conditions related to aberrant gene expression are also described.
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