NovoCell
Cellular Engineering
In vivo Gene Transfer
Transitioning from in vitro transfection to in vivo systems represents a significant leap forward in the treatment of genetically based diseases. In vivo delivery systems have the potential to inhibit undesirable gene expression or to synthesize therapeutic proteins. Although several viral-based vectors have been studied for clinical gene therapy due to their high transduction efficiency, they still face serious challenges, including adverse effects and manufacturing.
NovoCell Features
NovoCell is being developed as a safe and effective nonviral gene delivery system that has shown potential for clinical application. Core to the platform’s performance is to protect cargo from enzymatic digestion, effectively traverse barriers (both extracellular and intracellular) and transfect cells in vivo.
Applications of interest include the treatment of neurogenetic disorders, infectious diseases, and monogenic disorders.
