Arc Capsid RNA Delivery for Targeted Cellular Uptake
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Solution Overview
Problem
Current methods for delivering therapeutic agents, particularly nucleic acids, to cells are inefficient and face challenges in targeting specific sites within the cell, leading to low delivery efficacy.
Innovation Solution
Development of recombinant Arc and endogenous Gag polypeptides to form capsids that can encapsulate therapeutic agents, such as RNA, and deliver them to specific cellular locations, utilizing engineered polypeptides with high sequence identity to SEQ ID NOs for targeted delivery.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current delivery methods are used to deliver nucleic acids to cells, then delivery can be achieved, but immunogenicity occurs and off-target effects are produced
Solution Approach 1:
The patent uses Arc capsids as intermediary vehicles to deliver nucleic acid therapeutics. These capsids are engineered to replace immunogenic viral vectors while maintaining delivery functionality. The Arc capsid structure serves as a biocompatible mediator that protects the therapeutic cargo and facilitates cellular uptake without triggering immune responses or causing off-target effects.
Solution Approach 2:
The patent modifies the physical and chemical parameters of the delivery system by using recombinant Arc polypeptides with specific amino acid sequences (at least 80% identity to SEQ ID NO: 1). These parameter changes in capsid composition and structure enable controlled delivery while reducing immunogenicity compared to traditional viral vectors.
2Reliability
If current delivery methods are used, then nucleic acids can be delivered, but payload capacity is limited
Solution Approach 1:
The patent employs a nested structure where therapeutic nucleic acid payloads are encapsulated within Arc capsids. The capsid forms a protective shell that houses the cargo, enabling increased payload capacity while maintaining delivery efficacy. This nested architecture allows for substantial therapeutic cargo loading compared to traditional delivery systems.
3Reliability
If current delivery methods are used, then therapeutic agents can be delivered, but bio-distribution is poor
Solution Approach 1:
The patent engineers Arc capsids with specific local properties including surface charge characteristics and structural features that enhance cellular uptake and tissue penetration. The capsid design incorporates specific amino acid sequences and structural motifs that improve bio-distribution to target tissues while maintaining stable circulation in the bloodstream.
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
Enhances the delivery efficiency and specificity of therapeutic agents, such as RNA, to cells, including eukaryotic, vertebrate, and mammalian cells, particularly human cells, by using engineered Arc and endogenous Gag polypeptides to form capsids that facilitate targeted cargo delivery.
Implementation Method 1
recombinant and engineered Arc and endogenous Gag polypeptides to form capsids that can encapsulate therapeutic agents
Implementation Method 2
deliver them to specific cells by assembling into capsids for targeted cargo delivery
Data Source
AI summary
Disclosed herein, in certain embodiments, are recombinant Arc and endogenous Gag polypeptides, and methods of using recombinant Arc and endogenous Gag polypeptides.