Value Proposition:
· Scalable, efficient, and tunable nanoparticle creation technique that can deliver a variety of payloads
· Polymer-lipid nanoparticles are biodegradable and are being tested for immunogenicity
· Can be used for in vivo gene therapy delivery as well as in vitro cell transfection and research applications
Technology Description
· Researchers at Johns Hopkins have developed combination polymer-lipid-nanoparticles that can be used to encapsulate genetic material (e.g., plasmid DNA, mRNA, siRNA, etc.).
· These nanoparticles are tunable in size and have increased cargo loading capacity over other comparable nanoparticles.
· These nanoparticles can be used as non-viral vectors for gene therapy that increase loading capacity, long-term stability, and targeted delivery, while reducing side effects as compared to current vectors.
Unmet Need
Many diseases can benefit from gene therapy, including genetic disorders, cancers, and cardiovascular diseases [Nayerossadat, 2012]. The four FDA-approved gene therapy products use viral vectors for gene delivery, but many companies and research groups are developing non-viral vectors, including lipid and polymeric nanoparticles. Current vectors are limited by relatively low cargo capacity, immunogenicity, and the potential for serious off-target effects. Therefore, there is a strong need to develop gene therapy vectors with increased loading capacity, long-term stability, and targeted delivery with reduced side effects.
Stage of Development
· Proof of concept is complete, pre-clinical validation in animal models is ongoing
Publication
· Manuscript in process.
