Nanopharmaceutical & Regulatory Science

The Nanopharmaceutical & Regulatory Science (NPRS) group investigates important topics in nanopharmaceuticals and their related regulatory aspects and challenges. We develop and characterize novel nano-based formulations with the goal of improving the therapeutic index of active pharmaceutical ingredients. In collaboration with other research institutions, we identify and investigate critical quality attributes of nanopharmaceuticals and nanosimilars, such as mechanisms of action, safety, and practical application issues. We also interface with the pharmaceutical industry and regulatory authorities to establish science-based guidelines on the safe and correct use of nanopharmaceuticals and nanosimilars.

Our team is dedicated to the development of safe and effective therapies for lysosomal storage diseases, a group of serious metabolic disorders caused by mutations in lysosomal hydrolases that can lead to major physical or cognitive impairments. The current standard of care involves the replacement of dysfunctional enzymes with recombinant versions through blood infusions. However, immune reactions can result in the neutralization of these therapeutic enzymes and severely decrease the efficacy of treatment.

We believe that incorporating pharmaceuticals into nanoformulations has the potential to decrease the adverse immune reactions seen with current therapies. Therefore, we focus our research on the development of novel strategies for the encapsulation of lysosomal enzymes into lipid-based nanoformulations and use carbohydrate-based targeting strategies aimed at increasing specific particle uptakes and improving the pharmacokinetic properties of our nanoformulations. The results of this project will help to develop novel formulations and overcome the undesired enzyme distribution as well as the adverse immune response seen with current therapies.