Lipid Nanoparticle Stem Cell Targeting via Antibody Moieties

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Solution Overview

Problem

Current RNA-based therapeutic delivery systems face challenges in targeted delivery to specific cell types, particularly for organs other than the liver, due to inefficient targeting mechanisms.

Innovation Solution

A composition comprising a therapeutic agent and a delivery vehicle with a targeting moiety specific for binding to target stem cells, such as hematopoietic or mesenchymal stem cells, is used for targeted delivery. The delivery vehicle can be a lipid nanoparticle encapsulating nucleoside-modified RNA, with targeting moieties like CD34, CD117, or CD105 for specific stem cell types.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If lipid nanoparticles are used for delivering RNA-based therapeutics, then protection and delivery to the site of action is improved, but targeted delivery to specific cell types remains inefficient

Engineering Contradiction:

Improvedelivery protectionVSAvoidtargeting specificity

Core Design Contradiction:

ReliabilityVSManufacturing precision

Solution Approach 1:

The patent employs antibodies or antibody fragments as intermediary molecules that mediate between the lipid nanoparticle delivery vehicle and target stem cells. These antibodies serve as targeting moieties that specifically bind to stem cell surface markers (CD34, CD117, CD133, CD105, ABCG2, BMPR, CD44, Sca-1, Thy-1, CD146, GD2, SSEA-3, SSEA-4, Stro-1, Stro-4, MSCA-1, CD56, CD200, PODXL, CD13, CD29, CD44, or CD10), enabling the delivery vehicle to specifically target and deliver therapeutic agents to stem cells while maintaining the protective function of the lipid nanoparticle structure.

Inventive Principle:

Principle #24Intermediary (Mediator)

2Device complexity

If non-targeted delivery systems are used, then device complexity is reduced, but delivery efficiency to specific stem cell populations is insufficient

Engineering Contradiction:

Improvedelivery system simplicityVSAvoiddelivery efficiency

Core Design Contradiction:

Device complexityVSProductivity

Solution Approach 1:

The patent creates a universal targeting platform where a single lipid nanoparticle delivery vehicle can be configured with different antibody-based targeting moieties to deliver the same therapeutic agent to different stem cell populations. This multi-functional approach allows the delivery system to maintain relative simplicity in its core structure while achieving high delivery efficiency to various specific stem cell types through interchangeable targeting components.

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

This approach enables efficient and targeted delivery of therapeutic agents to specific stem cell populations, potentially enhancing the efficacy and specificity of RNA-based therapies for various diseases.

Implementation Method 1

the delivery vehicle comprises a targeting moiety specific for binding to a target stem cell

Methodology Applied

Scientific EffectMolecular recognition:

Implementation Method 2

Drug delivery systems (DDS) including lipid nanoparticles (LNPs) are employed to pack RNA and protect cargo en route to the site of action

Methodology Applied

Scientific EffectEncapsulation: Physical Containment

Data Source

PatentUS20250041436A1Compositions and Methods for Targeting Lipid Nanoparticle Therapeutics to Stem Cells

Publication Date: 2025.02.06 THE TRUSTEES OF THE UNIV OF PENNSYLVANIA

AI summary

The present invention relates to compositions and methods for effective delivery of an agent to a stem cell using a delivery vehicle comprising a stem cell targeting domain.