Lipid Nanoparticle Composition for Intracellular Enzyme Delivery
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Solution Overview
Problem
Current enzyme replacement therapies for lysosomal storage disorders face challenges such as unwanted immune responses and inefficient intracellular delivery of enzymes, limiting their therapeutic efficacy.
Innovation Solution
A lipid nanoparticle composition comprising hydrogenated soybean phosphatidylcholine (HSPC), cholesterol, dioleoyl-3-trimethylammonium propane (DOTAP), and 1,2-distearoyl-sn-glycero-3-phosphoethanolamine-N-methoxy (polyethylene glycol) (DSPE-mPEG) is used to encapsulate therapeutic proteins, enabling targeted intracellular delivery.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If enzyme replacement therapy is administered intravenously, then the concentration of harmful substances in cells is reduced, but unwanted immune responses occur and intracellular delivery efficiency is low
Solution Approach 1:
The patent uses lipid nanoparticles as an intermediary carrier to deliver enzyme proteins to target cells. The LNP composition including HSPC, cholesterol, DOTAP, and DSPE-mPEG forms a protective vehicle that facilitates cellular uptake while minimizing immune recognition, thus resolving the contradiction between therapeutic efficiency and immune response
2Reliability
If enzyme replacement therapy is administered intravenously, then the concentration of harmful substances in cells is reduced, but intracellular delivery efficiency is low
Solution Approach 1:
The patent modifies the physical and chemical parameters of the delivery system by using lipid nanoparticles with specific composition ratios (HSPC:cholesterol:DOTAP:DSPE-mPEG). This parameter optimization enables efficient cellular uptake and intracellular delivery of enzyme proteins, resolving the contradiction between therapeutic efficiency and delivery efficiency
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 lipid nanoparticles enhance the delivery of enzymes to cells, improving therapeutic efficacy by minimizing immune responses and ensuring effective intracellular distribution, thereby treating lysosomal storage disorders effectively.
Implementation Method 1
a lipid nanoparticle for intracellular or in vivo delivery of a protein... comprising a lipid mixture including hydrogenated soybean phosphatidylcholine (HSPC); cholesterol; dioleoyl-3-trimethylammonium propane (DOTAP); and 1,2-distearoyl-sn-glycero-3-phosphoethanolamine-N-methoxy (polyethylene glycol) (DSPE-mPEG)
Data Source
AI summary
The present invention relates to lipid nanoparticles for the delivery of proteins and, specifically, to lipid nanoparticles for the intracellular or in vivo delivery of various medicinal proteins such as therapeutic proteins or enzymes for enzyme replacement therapy (ERT). The present invention can be advantageously used for the treatment and prevention of various diseases, including lysosomal storage disorders, by stably and effectively delivering various proteins such as therapeutic proteins or ERT enzymes into cells through lipid nanoparticles.