Cationic Polymer Carriers for Nucleic Acid and Polypeptide Uptake
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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 challenging problem in peptide, protein, and nucleic acid-based technologies.
Innovation Solution
A polymer comprising a structure of Formula 1, where m1 and n1 are integers from 0 to 1000, and m2 and n2 are integers from 0 to 1000 with a sum greater than 5, linked randomly, with specific groups A1, A2, B1, and B2, is used to deliver nucleic acids and polypeptides.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If large molecules such as polypeptides and nucleic acids are delivered to target tissues using conventional methods, then the therapeutic effect can be achieved, but the delivery safety and effectiveness deteriorate due to challenges in targeting and cellular uptake
Solution Approach 1:
The patent uses cationic polymers as intermediary carriers to mediate the delivery of large therapeutic molecules (polypeptides and nucleic acids) to target tissues. The polymer forms complexes with the therapeutic molecules, protecting them from degradation and facilitating their uptake by target cells through endocytosis, thereby improving both safety and effectiveness of delivery
Solution Approach 2:
The patent employs polymers with specific tunable parameters including molecular weight (5-2000 kDa), polydispersity index (1.05-2.5), and charge density to optimize delivery performance. By adjusting these parameters, the polymer can be tailored to match specific therapeutic molecules and target tissues, improving delivery reliability while maintaining safety
2Ease of manufacture
If the polymer structure is simplified for easier manufacture, then the manufacturing precision may deteriorate, affecting the polymer's ability to effectively deliver biomolecules
Solution Approach 1:
The patent defines specific parameter ranges for the polymer structure (molecular weight 5-2000 kDa, polydispersity index 1.05-2.5, nitrogen content 5-20%) that balance ease of synthesis with functional performance. These parameter specifications allow for relatively simple manufacturing processes while ensuring the polymer maintains the precise structural characteristics needed for effective biomolecule delivery
Solution Approach 2:
The patent employs copolymer structures combining different monomer units with specific functional groups (amines, carboxylic acids, hydroxyls) to achieve the desired balance between manufacturability and performance. The composite nature of the polymer allows for modular synthesis approaches that simplify manufacturing while maintaining structural precision through controlled polymerization conditions
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 effectively delivers nucleic acids and polypeptides to cells, enhancing their uptake and functionality, as demonstrated by successful delivery of Cas9 RNP and Cre recombinase in various cell types and tissues.
Implementation Method 1
The polymer effectively delivers nucleic acids and polypeptides to cells, enhancing their uptake and functionality
Data Source
AI summary
Provided is a polymer comprising a structure of Formula (1): (1) and a method of preparing said polymer. Also provided is a composition comprising the polymer and a nucleic acid and/or polypeptide, and a method of delivering a nucleic acid and/or polypeptide to a cell.