Single-Stranded Nucleic Acid Molecule with Stearic Acid Delivery

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Solution Overview

Problem

Existing methods for inhibiting gene expression, such as RNA interference, face challenges in manufacturing efficiency and delivery to target tissues, particularly due to the need for double-stranded RNA molecules and laborious handling conditions, as well as difficulties in synthesizing circular RNA molecules.

Innovation Solution

A single-stranded nucleic acid molecule with a linker region bonded to stearic acid, comprising a region complementary to itself, which serves as an expression inhibitory sequence, allowing for direct delivery to target tissues without a carrier, thereby reducing production and handling complexities.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If double-stranded RNA molecules (siRNA) are used to inhibit gene expression, then gene expression inhibition is achieved, but manufacturing efficiency is low and handling conditions become laborious

Engineering Contradiction:

Improvegene expression inhibitionVSAvoidmanufacturing efficiency

Core Design Contradiction:

ReliabilityVSProductivity

Solution Approach 1:

The invention segments the double-stranded RNA into two separate single-stranded RNA components: a sense strand and an antisense strand. These strands are designed to hybridize within the target cell to form the functional double-stranded structure, eliminating the need for complex external hybridization procedures and improving manufacturing efficiency.

Inventive Principle:

Principle #1Segmentation

Solution Approach 2:

The patent introduces a carrier molecule as an intermediary that facilitates the delivery of single-stranded RNA into target cells. The carrier protects the RNA during administration and enables intracellular release, simplifying handling conditions while maintaining gene expression inhibition efficacy.

Inventive Principle:

Principle #24Intermediary (Mediator)

2Reliability

If double-stranded RNA molecules (siRNA) are administered to cells, then gene expression inhibition is achieved, but laborious handling conditions are required to prevent dissociation to single-stranded RNAs

Engineering Contradiction:

Improvegene expression inhibitionVSAvoidhandling conditions

Core Design Contradiction:

ReliabilityVSEase of operation

Solution Approach 1:

The invention performs preliminary action by pre-complexing the single-stranded RNA with a carrier molecule before administration. This pre-formation of the RNA-carrier complex ensures stability during handling and administration, eliminating the need for laborious conditions to prevent dissociation while maintaining the ability to form functional double-stranded structure within the target cell.

Inventive Principle:

Principle #10Preliminary action

3Reliability

If circular RNA molecules are used to inhibit gene expression, then gene expression inhibition is achieved, but synthesis is difficult

Engineering Contradiction:

Improvegene expression inhibitionVSAvoidsynthesis difficulty

Core Design Contradiction:

ReliabilityVSEase of manufacture

Solution Approach 1:

Instead of attempting to synthesize difficult circular RNA molecules, the invention inverts the approach by using linear single-stranded RNA that forms its own secondary structure or hybridizes with complementary sequences to create the functional double-stranded configuration. This inversion dramatically simplifies synthesis while maintaining gene expression inhibition capability.

Inventive Principle:

Principle #13The other way round (Inversion)

4Ease of operation

If carriers are used for delivery of nucleic acid molecules, then delivery to target tissue is achieved, but toxicity of the carrier must be considered

Engineering Contradiction:

Improvedelivery functionVSAvoidcarrier toxicity

Core Design Contradiction:

Ease of operationVSObject-affected harmful factors

Solution Approach 1:

The patent systematically evaluates and optimizes carrier parameters including chemical composition, structural properties, and physical characteristics to achieve effective delivery while minimizing toxicity. By changing these parameters, the invention identifies carriers that balance delivery efficiency with biocompatibility, reducing harmful effects on target tissues.

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 single-stranded nucleic acid molecule achieves effective delivery and inhibition of target gene expression with improved efficiency and reduced toxicity, eliminating the need for carriers and simplifying production processes.

Implementation Method 1

the single-stranded nucleic acid molecule... allows for direct delivery to target tissues without a carrier

Methodology Applied

Scientific EffectTransfection:

Data Source

PatentEP3276003B1Single-chain nucleic acid molecule having delivery function and gene expression control ability

Publication Date: 2022.07.20 BONAC CORP

Figure 1(A)~1(B)
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Figure 3

AI summary

The present invention provides a single-stranded nucleic acid molecule having a delivery function and capable of inhibiting expression of a target gene. The single-stranded nucleic acid molecule of the present invention is a single-stranded nucleic acid molecule composed of a region (Xc), a linker region (Lx) and a region (X), wherein said region (Xc) is complementary to said region (X), at least one of said region (X) and said region (Xc) contains an expression inhibitory sequence that inhibits expression of the target gene, and a bio-related substance having a delivery function is bonded to at least one selected from the group consisting of the 5'-terminus, the 3'-terminus, and said linker region (Lx).