Block Copolymer Nanoparticles for Large Nucleic Acid Delivery

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Solution Overview

Problem

Current genetic medicine delivery systems, such as adeno-associated viruses (AAVs) struggle to deliver large genetic payloads or multiple payloads effectively, often triggering immune responses and are not suitable for non-viral delivery due to poor biocompatibility and enzymatic degradation.

Innovation Solution

A composition of block copolymers forming polymer nanoparticles (PNPs) that can complex with nucleic acids, allowing for the in vivo expression of full-length antibodies, including Fab and Fc portions, to target specific tissues and cells, using a block copolymer comprising poly-2-(dimethylamino) ethyl methacrylate (DMAEMA) and poly-monomethyl methacrylate (MMA) or poly-vinyl methacrylate (VMA) or butylmethacrylate (BMA), with a hydrodynamic diameter of 10 nm to 1000 nm.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Quantity of substance

If AAVs are used for genetic medicine delivery, then delivery capability is achieved, but large genetic payloads cannot be delivered and unwanted immune responses are triggered

Engineering Contradiction:

Improvegenetic payload sizeVSAvoidimmune responses

Core Design Contradiction:

Quantity of substanceVSObject-affected harmful factors

Solution Approach 1:

The patent uses polymer nanoparticles as an intermediary non-viral delivery vehicle to transport genetic payloads. These nanoparticles serve as a mediator between the delivery system and the genetic material, enabling large payload delivery without triggering the unwanted immune responses associated with viral vectors like AAVs.

Inventive Principle:

Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent changes the fundamental parameters of the delivery system by switching from viral vectors to synthetic polymer nanoparticles. This parameter change allows for larger payload capacities and eliminates the immune response issues inherent in viral-based systems, while maintaining effective delivery capability.

Inventive Principle:

Principle #35Parameter changes

2Object-affected harmful factors

If non-viral delivery systems such as liposomes are used, then immune responses are avoided, but biocompatibility is poor and enzymatic degradation occurs

Engineering Contradiction:

Improveimmune responsesVSAvoidbiocompatibility and stability

Core Design Contradiction:

Object-affected harmful factorsVSReliability

Solution Approach 1:

The patent employs composite polymer nanoparticles that combine multiple functional characteristics. These composite structures provide both the stability and biocompatibility needed for reliable in vivo delivery, while avoiding the immune responses associated with traditional non-viral systems like liposomes.

Inventive Principle:

Principle #40Composite materials

Solution Approach 2:

The polymer nanoparticles are designed as biodegradable, disposable delivery vehicles that perform their function and then naturally degrade in the body. This approach eliminates the need for long-term stability requirements while maintaining high reliability during the delivery period, and avoids immune responses by using synthetic rather than biological materials.

Inventive Principle:

Principle #27Cheap short-living objects (Disposable)

3Object-affected harmful factors

If current non-viral delivery systems are used, then immune responses are avoided, but multiple large payloads cannot be packaged

Engineering Contradiction:

Improveimmune responsesVSAvoidpayload packaging capacity

Core Design Contradiction:

Object-affected harmful factorsVSAdaptability or versatility

Solution Approach 1:

The polymer nanoparticle platform is designed with universal capability to package multiple different types of genetic payloads, including large plasmids and multiple smaller payloads. This multi-functional delivery system maintains the advantage of avoiding immune responses while achieving the versatility needed for various genetic medicine applications.

Inventive Principle:

Principle #6Universality (Multi-functionality)

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

Enables the endogenous production of antibodies in target tissues, providing sustained expression and improved stability compared to exogenous administration, while avoiding unwanted immune responses.

Implementation Method 1

A composition of block copolymers forming polymer nanoparticles (PNPs) that can complex with nucleic acids

Methodology Applied

Scientific EffectElectrostatic interaction: Electrostatics

Data Source

PatentUS20260034069A1Polymer nanoparticle compositions for in vivo expression of polypeptides

Publication Date: 2026.02.05 BATTELLE MEMORIAL INST

AI summary

The disclosure relates to block copolymer nanoparticles for in vivo therapeutic delivery, and methods therefor. More particularly, the invention relates to polymer nanoparticles, such as reversible addition-fragmentation chain transfer (RAFT) polymer compositions, for delivering nucleotides that encode polypeptides.