Biological Performance and Clinical Trial — WP3

Professor of Metabolic Diseases and Innovative Therapies – UMC Utrecht, Wilhelmina Children’s Hospital

Short bio:

Dr. Sabine Fuchs is a Professor at UMC Utrecht, specializing in pediatric metabolic diseases. After completing her pharmacy and medical degrees with distinction (cum laude) at Utrecht University, she pursued a PhD in the Department of Metabolic Diseases at UMC Utrecht. Her clinical and scientific work focuses on metabolic and genetic (liver) diseases, particularly in protein metabolism, stem cell research, organoids, and genetic therapies.

Dr. Fuchs is renowned for her innovative research, using prime editing and liver organoid models to investigate disease mechanisms and develop novel therapeutic strategies. These approaches are particularly crucial for patients with rare genetic diseases, where conventional treatments are often inadequate. She has received prestigious fellowships and grants, including the ZonMW VICI, ERC Starting Grant (StG), and ERC Proof of Concept (PoC). Her pioneering contributions to gene editing, including the use of prime editing for genetic correction in patient-derived models, have positioned her at the forefront of the field.

Beyond her research, Dr. Fuchs is a leader in education and has contributed to programs such as the EUREKA Translational Medicine Summer School and TULIPS (Training Upcoming Leaders in Pediatric Science). She is an active member of several advisory boards and committees and has secured multiple competitive grants, including funding from Metakids and ZonMW.

With over 70 peer-reviewed publications and more than 4,500 citations, Dr. Fuchs continues to push the boundaries of scientific research while ensuring the rapid translation of her findings into clinical care.

Professor of nanomedicine – UMC Utrecht

Short bio:

Professor Raymond Schiffelers is the coordinator of the NANOSPRESSO project, a key initiative in the field of nanomedicine. He currently serves as Professor of Nanomedicine at the University Medical Center Utrecht (UMC Utrecht), where his research focuses on bio-inspired and synthetic nanomedicines. His work covers a wide range of topics, including liposomal targeting, siRNA delivery, extracellular vesicles, and personalized medicine.

Schiffelers obtained his PhD in 2001 from Erasmus University Rotterdam, where he specialized in liposomal targeting of antimicrobial agents in bacterial infections. Following this, he worked at Intradigm Co. in Washington, DC, focusing on siRNA delivery using nanoparticles. Upon returning to the Netherlands, he established a nanomedicine research group at Utrecht University, and in 2012, he joined UMC Utrecht. In 2015, he was appointed Professor of Nanomedicine.

Professor Schiffelers has successfully coordinated several major European research projects, including B-SMART and EXPERT under the H2020 framework. His work is highly collaborative, with a strong emphasis on public-private partnerships.

In addition to his academic and research roles, Professor Schiffelers serves as Vice President of Preclinical R&D at Nanocell Therapeutics, bridging the gap between academia and industry. He is also the Chair of the European Technology Platform on Nanomedicine (ETPN) and an editor for several high-impact journals, reflecting his leadership in the field.