Nanoparticles Targeting Gene Editing Systems to Cancer Cells

Targeted gene therapy delivery platform for oncology indications with stable nanoparticles and no cytotoxicity

Background

To date, existing gene editing delivery systems involve viral vectors or cationic materials, which carry a high human safety concern. Researchers at Boston Children’s Hospital have designed non-viral, non-cationic nanoparticles to efficiently deliver therapies to cancer cells.

Technology Overview

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.

Further Details

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

Benefits

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

Applications

Targeted gene therapy delivery platform for oncology indications

IP Status

• Patent application submitted