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CMCC 3094

Nanoparticles targeting gene editing systems to cancer cells

Inventors: Marsha Moses, Peng Guo

Invention Types: Therapeutics

Research Areas: Oncology/Hematology

Keywords: Animal Model (mouse), Composition of Matter, Drug Delivery, Gene expression, Receptor/Ligand, Drug Design, Gene Therapy, Platform

Invention Description:

To date, existing gene editing delivery systems involve viral vectors or cationic materials, which carry a high human safety concern. Dr. Marsha Moses and her team have designed non-viral, non-cationic nanoparticles to efficiently deliver therapies to cancer cells.

Targeted nanolipogel (TNLG) technology combines a liposome with a hydrogel core to enhance encapsulation of the gene editing agent to be delivered. Further, ligands to cell surface proteins of cancer cells are conjugated to the surface, allowing the nanoparticles to target the cancerous cells specifically.

An in vitro triple negative breast cancer (TNBC) model was used in proof-of-principle studies for the platform. TNLGs carrying CRISPR-Cas9 plasmid targeting lipocalin 2 (Lcn2) shows no cytotoxicity to normal human breast cells. Lcn2 knockout was effective in reducing proliferation and migration of TNBC cells.

Applications:

• Targeted gene therapy delivery platform for oncology indications

Competitive Advantages:

• Eliminates viral vectors and cationic particles in delivery platform
• Highly stable nanoparticle, promising for intravenous administration
• No cytotoxicity in vitro and in vivo

Business Opportunity:

License, sponsored research, collaboration

Key Publications: Guo P, Liu D, Subramanyam K, et al. Nanoparticle elasticity directs tumor uptake. Nat Commun. 2018; 9(1):130

Related Publications: https://vector.childrenshospital.org/2018/03/softer-nanoparticle-better-drug-delivery/

IPStatus: Pat. Pend.