Hydrolysable Polymer with Cationic and Hydrophobic Side Chains for Nucleic Acid Delivery
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Solution Overview
Problem
The safe and effective delivery of large molecules such as polypeptides and nucleic acids to target tissues remains a challenge due to limitations in existing technologies.
Innovation Solution
A polymer with a hydrolysable backbone comprising hydrophobic and cationic side chains, including a polyamine with a tertiary amine and a tissue-specific or cell-specific targeting moiety, is developed for efficient delivery of nucleic acids and polypeptides to cells.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If existing delivery technologies are used for large molecules, then delivery to target tissues can be achieved, but safety and effectiveness are compromised
Solution Approach 1:
The patent employs a composite polymer structure combining cationic side chains (for nucleic acid binding via electrostatic interactions) with hydrophobic side chains (for membrane interaction and cellular uptake). This composite design creates a delivery system that is both effective at transporting large molecules and safe for biological applications, resolving the contradiction between delivery effectiveness and safety.
2Productivity
If polyamines with multiple nucleophilic centers are used, then transfection efficiency improves, but toxicity increases
Solution Approach 1:
The patent specifies that polyamines should have only a single nucleophilic center (either a primary or secondary amine) rather than multiple nucleophilic centers. This local quality constraint on the polyamine structure maintains sufficient transfection efficiency while dramatically reducing toxic effects associated with multiple reactive nucleophilic sites that can damage cellular components.
Solution Approach 2:
The patent changes the chemical parameter of the polyamine from having multiple nucleophilic centers to having only a single nucleophilic center. This parameter modification reduces the reactivity and toxicity of the polyamine while preserving its ability to facilitate nucleic acid delivery and achieve transfection.
3Ease of operation
If non-specific delivery is used, then delivery process is simple, but target tissue specificity is poor
Solution Approach 1:
The patent incorporates tissue-specific or cell-specific targeting moieties at specific locations on the polymer structure (such as at the terminus of the polyamine side chain). This local addition of targeting functionality allows the polymer to achieve specific delivery to target tissues while maintaining the overall simplicity of the delivery process, as the targeting is achieved through molecular design rather than complex delivery protocols.
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 polymer enables effective transfection and targeted delivery of therapeutic molecules, enhancing payload functionality and biocompatibility, as demonstrated by improved transfection efficiency and bioluminescence imaging in preclinical studies.
Implementation Method 1
the cationic side chain comprises a polyamine with at least one tertiary amine
Implementation Method 2
monomer units comprising a hydrophobic side chain
Implementation Method 3
a hydrolysable polymer backbone
Data Source
AI summary
Provided is a polymer comprising a hydrolysable polymer backbone, the polymer backbone comprising (i) monomer units comprising a hydrophobic side chain; and (ii) monomer units comprising a cationic side chain; wherein the cationic side chain comprises a polyamine with at least one tertiary amine and only a single nucleophilic center, optionally at the terminus of the polyamine, as well as a method of preparing said polymer, and a method of delivering a nucleic acid and/or polypeptide to a cell using the polymer.