CIDEB-Targeting RNAi Agents for Precise Gene Silencing

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Solution Overview

Problem

Current treatments for CIDEB-associated diseases, particularly liver diseases such as metabolic dysfunction-associated steatohepatitis (MASH), lack effective methods to inhibit CIDEB expression and address the underlying pathophysiology.

Innovation Solution

Development of RNAi agents comprising a sense and antisense strand targeting CIDEB, designed to form a double-stranded region with nucleotide sequences that closely match specific CIDEB sequences, allowing for precise inhibition of CIDEB expression.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If current treatments are used for CIDEB-associated diseases, then existing therapeutic options are maintained, but effective inhibition of CIDEB expression is not achieved

Engineering Contradiction:

Improveeffectiveness of treatmentVSAvoidavailability of therapeutic options

Core Design Contradiction:

ReliabilityVSAdaptability or versatility

Solution Approach 1:

The patent employs RNA interference technology to change the molecular parameter of CIDEB gene expression, using double-stranded RNA molecules to trigger degradation of CIDEB mRNA and thereby inhibit protein production at the molecular level

Inventive Principle:

Principle #35Parameter changes

Solution Approach 2:

The patent replaces conventional pharmacological approaches with a nucleic acid-based mechanism, utilizing the cellular RNA interference pathway to achieve targeted gene silencing instead of using small molecule drugs that act on protein targets

Inventive Principle:

Principle #28Mechanics substitution (Replace mechanical system)

2Reliability

If RNAi agents with high sequence complementarity are designed to inhibit CIDEB expression, then inhibition effectiveness is improved, but manufacturing precision requirements increase

Engineering Contradiction:

Improveinhibition effectivenessVSAvoidnucleotide sequence accuracy

Core Design Contradiction:

ReliabilityVSManufacturing precision

Solution Approach 1:

The patent applies local quality by designing RNAi agents with specific nucleotide sequences that have high complementarity to target CIDEB mRNA regions, while allowing variations in non-critical regions to maintain manufacturing feasibility

Inventive Principle:

Principle #3Local quality

Solution Approach 2:

The patent optimizes the nucleotide sequence parameters of RNAi agents to achieve maximum binding affinity to CIDEB mRNA while maintaining stability during manufacturing and storage, balancing sequence precision with practical production requirements

Inventive Principle:

Principle #35Parameter changes

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The RNAi agents effectively inhibit CIDEB expression, providing a therapeutic approach for treating CIDEB-associated diseases, particularly liver diseases like MASH, by targeting and reducing the protein's activity.

Implementation Method 1

the dsRNA agent comprises a sense strand and an antisense strand forming a double stranded region

Methodology Applied

Scientific EffectRNA base pairing:

Data Source

PatentUS20250263702A1Rnai agents targeting cideb and related methods

Publication Date: 2025.08.21 FLAGSHIP PIONEERING INNOVATIONS VII LLC

AI summary

Provided herein are, inter alia, agents (e.g., RNAi agents, dsRNA agents) comprising a sense strand and an antisense strand targeting CIDEB (e.g., hCIDEB); and methods of manufacturing and pharmaceutical compositions comprising the same. Further provided herein are methods of utilizing the agents (e.g., RNAi agents, dsRNA agents) including, e.g., methods of inhibiting or decreasing CIDEB expression (e.g., mRNA expression), methods of treating CIDEB associated diseases, and methods of treating liver diseases (e.g., MASH).