Introduction

The goal of Regulatory Science is to support regulatory decision-making based on a scientific understanding of the benefits and risks associated with a product. Regulatory Science can be viewed as an important bridge for product development, connecting regulators who establish regulations, applicants who must comply with them, and the scientific community that researches relevant areas. In the Nanopharmaceutical & Regulatory Science (NPRS) group, we conduct research focused on gaining a deeper understanding of nanotechnology in the regulatory context to inform decision-making.

Our work

Recently, we have directed our attention to the use of composite nanoparticles as active ingredients in medicinal products. Nanomedicines are unique in that all components of the nanoparticle play a role in the efficacy and safety profiles of the final drug product. We are studying how the classification of individual components of a drug product impacts regulatory data requirements and regulatory dossier structure.

Our research is dedicated to advancing the development of the new generation of enzyme replacement therapy for lysosomal storage diseases. To achieve this goal, we are using cutting-edge approaches, such as encapsulating lysosomal enzymes into lipid-based nanoformulations and utilizing carbohydrate-based targeting strategies to enhance specific particle uptake. Through these techniques, we aim to create formulations with improved pharmacokinetic properties and reduced adverse immune reactions compared to current therapies. Our team believes that these novel formulations have the potential to overcome the limitations of current therapies, such as undesired enzyme distribution and immune responses, and offer a more effective treatment option for patients suffering from lysosomal storage diseases.