Gene Editing for Head and Neck Cancer Treatment

Application

Method for delivering gene-editing therapeutic in head and neck cancers.

Key Benefits

  • CRISPR/CAS9-based gene editing mechanism targeting novel disease-modifying gene.
  • Liquid nanoparticle encapsulation permitting in vivo delivery.

Market Summary

Head and neck cancers (HNC) are the 7th most common global cancer, accounting for 3% of cancer diagnoses and 1.5% of cancer-related deaths in the United States. Recent innovations for treating advanced HNC have limited efficacy across patient subsets. With major pharmaceutical companies recently advancing HNC therapeutics into clinical trials, there is evident demand for more effective therapeutics with improved patient response rates.

Technical Summary

To reduce tumor growth in HNC, Emory researchers designed a gene modification system for targeting previously undruggable cancer mechanisms. To enable safe and effective delivery in vivo, researchers encapsulated mRNA encoding Cas9 and the optimized single guide RNA into lipid nanoparticle formulations. When testing this therapeutic in mouse models of oral cancer, researchers demonstrated significantly reduced tumor burden and effective target engagement. This technology is positioned for further testing in highly clinical-relevant models.

Development Stage

This technology has been assembled and tested in mouse cancer models.

Patent Information: