Inventor(s): KiBum Lee, Thanapat Pongkulapa, Brandon Conklin

Awarded: April 2022

Summary:

The rising demand for complex biological therapies has created an urgent need for robust and advanced genome editing techniques. However, these current techniques are still lacking the technology to deliver genetic materials (i.e., CRISPR plasmids) in an efficient, quick, and precise manner, which is preventing them from being used in clinical trials. Current delivery technologies rely on viral vectors, which have technical limitations in cargo loading size. Such a limitation is the bottleneck for a clinical translation of the more sophisticated gene-editing systems to the clinics. To this end, we offer our magnetic nanoparticle-based delivery platform as a solution to effectively deliver genetic materials in a non-viral manner. The platform permits the delivery of multiple plasmids without size constraints, owing to our unique nanoparticle design. Moreover, with our unique composition of magnetic nanoparticles, the magnetic properties can be used for magnetic-assisted cell sorting, resulting in enhanced gene-editing efficiency. In both academic settings and biopharma businesses producing CRISPR-based and other gene therapies, our solution can be integrated and used in drug development pipelines. In summary, our technology provides an effective CRISPR-based gene therapeutic method using the unique magnetic nanoparticle platform for genetic disorders.

Market Applications:

  • Transfection reagents for therapeutic genes and gene editing tools (e.g., CRISPR/Cas9)
  • Magnetic-assisted cell sorting products
Visual description of Magnetic Nanoparticle-Assisted Non-Viral CRISPR-Cas9 for Advanced Genome Editing