Silica Nanoparticle Transfection System for Gene Therapy

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Solution Overview

Problem

The high cost and inefficiency of large-scale production of virus-based gene therapy vectors for clinical use, particularly due to toxic envelope proteins that harm producer cells, limit the widespread adoption of gene therapy.

Innovation Solution

A composition comprising inorganic nanoparticles with projections and a transfection agent coating, which effectively binds and delivers vectors, viruses, or plasmids to cells, enhancing transfection and transduction efficiency.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Productivity

If traditional transfection agents (PEI, FuGENE, Lipofectamine) are used to deliver plasmids to producer cells, then transfection efficiency is achieved, but the envelope proteins become toxic to producer cells limiting virus production

Engineering Contradiction:

Improvevirus productionVSAvoidtoxicity to producer cells

Core Design Contradiction:

ProductivityVSObject-affected harmful factors

Solution Approach 1:

The patent uses an inorganic nanoparticle as an intermediary carrier to deliver envelope proteins to producer cells. The nanoparticle binds the envelope protein and facilitates its entry into the cell without the protein directly contacting and toxicizing the cell membrane, thus resolving the toxicity problem while maintaining transfection efficiency

Inventive Principle:

Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent changes the physical form and delivery mechanism of the envelope protein by conjugating it to inorganic nanoparticles. This parameter change in delivery method allows the envelope protein to be introduced into producer cells without exerting toxic effects, enabling sustained virus production

Inventive Principle:

Principle #35Parameter changes

2Reliability

If multiple plasmids are transfected simultaneously to produce recombinant viruses, then complete viral components are delivered, but the complexity of ensuring all plasmids reach the same cell increases

Engineering Contradiction:

Improvecomplete viral component deliveryVSAvoidtransfection system complexity

Core Design Contradiction:

ReliabilityVSDevice complexity

Solution Approach 1:

The patent combines multiple plasmids into a single inorganic nanoparticle complex. The nanoparticle can bind and deliver multiple different plasmids (including envelope protein plasmids and viral genome plasmids) simultaneously to the same producer cell, ensuring complete viral component delivery while simplifying the transfection process

Inventive Principle:

Principle #5Merging (Combining)

Solution Approach 2:

The inorganic nanoparticle serves as a universal delivery vehicle that can carry multiple types of genetic material (different plasmids) with a single transfection step, making the system multi-functional and eliminating the need for separate transfection procedures for each plasmid

Inventive Principle:

Principle #6Universality (Multi-functionality)

3Reliability

If replication-incapable viruses are produced by removing structural genes from the viral genome, then safety is improved, but production costs increase due to complex plasmid construction and transfection requirements

Engineering Contradiction:

Improvesafety of gene therapy vectorsVSAvoidproduction cost

Core Design Contradiction:

ReliabilityVSEase of manufacture

Solution Approach 1:

The inorganic nanoparticle acts as a mediator that simplifies the manufacturing process by enabling efficient delivery of multiple plasmids in a single step. This reduces production complexity and costs while maintaining the safety advantage of replication-incapable virus production

Inventive Principle:

Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent changes the delivery parameter from conventional chemical transfection agents to inorganic nanoparticle-mediated delivery, which improves manufacturing efficiency and reduces costs associated with producing safe, replication-incapable therapeutic viruses

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 nanoparticle-based delivery system improves the efficiency of transfection and transduction, allowing for the simultaneous delivery of multiple components and reducing the toxicity issues associated with traditional transfection agents, thereby making gene therapy more viable and cost-effective.

Implementation Method 1

The nanoparticle is at least partially coated with a transfection agent; wherein the one or more delivery components are selected from: vectors, viral vectors, plasmids, viruses, viroids, prions, virus-like particles, virus-derived components and mixtures thereof

Methodology Applied

Scientific EffectTransfection:

Implementation Method 2

the nanoparticle comprises projections thereon; wherein the nanoparticle has a diameter in the range 50 nm to 3000 nm; wherein the inorganic nanoparticle is at least partially coated with a transfection agent

Methodology Applied

Scientific EffectAdsorption: Adsorption

Data Source

PatentUS20230165971A1Transfection and Transduction System

Publication Date: 2023.06.01 N4 PHARMA UK LTD

AI summary

The invention relates to the fabrication and use of silica organic nanoparticles as delivery vehicles for vims and virus-like species to the body. The nanoparticles typically have a hollow core and a surface morphology that allows effective adhesion of species to the surface for delivery to the body. In particular, the invention is particularly useful for performing transfection and transduction.