Neural Cell-Derived Nanoparticles for Targeted Delivery to Neural Cells


This invention is an engineered biomimetic platform composed of lipid-based nanoparticles functionalized with human neural proteins, termed neurosomes. The integration of these neural proteins within the nanoparticle allows for a completely customizable platform which can be used for localized drug delivery to neural cells in order to treat a variety of neurological disorders. Various hydrophilic and hydrophobic reagents can be encapsulated into this platform, including but not limited to, siRNA, mmRNA, miRNA, and therapeutic proteins.


Stage of Development


In vitro data: Neurosomes encapsulating siRNA were used to validate protein coating consistency, encapsulation efficiency, stability of encapsulation, reproducibility, toxicity, and various other characterization parameters.

In vivo data: Preliminary biodistribution studies revealed that neurosomes remained localized around the injection site for 24 hours without displaying signs of toxicity.


Competitive Landscape


There are currently no platforms which integrate neural proteins into nanoparticles to aid in the targeted delivery of therapeutic cargo to neural cells. However, the recent FDA approval of Patisiran, the first RNAi lipid nanoparticle formulation for polyneuropathy, suggests that there is a need for better delivery platforms targeting the nervous system. This is especially true in regards to delivery of genetic cargo and drugs which have significant adverse effects.


Competitive Advantages


•       Enhanced neural cell targeting

•       Minimal toxicity

•       Ability to deliver various sized therapeutic cargo (molecule or genetic)

•       Off-the-shelf potential 

•       High yield, rapid, and cost-effective manufacturing process


Patent Information:
Licensing Contact
Paige Glumac

Robert Krencik
Assaf Zinger
Ennio Tasciotti
Caroline Cvetkovic