Biodegradable Polymers for Gene Delivery

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Invention Summary:

In gene therapy, a key challenge is delivering therapeutic genes to target cells, which requires gene delivery vectors. While viral vectors are effective, they pose risks such as immune responses and toxicity. Non-viral vectors, particularly cationic polymers, are preferred for their safety, cost-effectiveness, and high loading capacity, but their high molecular weight, positive charge, and non-degradable nature cause cytotoxicity, limiting their clinical use. 

Researchers at Rutgers and collaborators from Boston College have developed biodegradable cationic polymers (BDPs) for gene delivery to address toxicity issues. This study builds on earlier reports of degradable vinyl random copolymers synthesized via radical ring-opening cascade copolymerization (rROCCP) of macrocyclic allylic sulfones. This chemistry incorporated biodegradable ester groups directly into the polymer backbone, improved biocompatibility, reduced cytotoxicity, and enhanced gene delivery function. Backbone-degradability may enhance disassembly kinetics of the polyelectrolyte complex in the cytosol enabling more efficient payload release.

Market Applications:

• Non-viral vector for gene therapies
  • Genetic disease treatment
  • Cancer gene therapy
  • mRNA-based therapeutics

Advantages:

• Cytotoxicity: The biodegradable polymer backbone lowers toxicity, making gene delivery safer for cells.
• Gene Delivery: Enhanced disassembly in the cytosol allows more efficient release of genetic material into target cells.
• Biocompatibility: Degradation into smaller fragments facilitates easier excretion and reduces long-term accumulation in the body.