Manoe Janssen
Senior Scientist Translational Models & Therapy Development, Radboudumc
Manoe Janssen obtained her BSc in Molecular Life Sciences and MSc in Molecular Mechanisms of Disease at Radboudumc. She completed her PhD in the Department of Gastroenterology and Hepatology, focusing on the molecular mechanisms underlying polycystic liver disease. For her postdoctoral research, she joined the Stem Cell Institute in Leuven, and later the lab of Prof. Dr. Roos Masereeuw at Utrecht University, where she became Assistant Professor. Her work centered on genetic kidney disease, using patient-derived iPSC kidney organoids for therapy development and nephrotoxicity testing.
In 2024, she joined the Therapy Accelerator for Rare Diseases at Radboudumc as Senior Scientist Translational Models & Therapy Development. This multidisciplinary team aims to accelerate translation of academic discoveries into clinical therapies. Manoe combines her scientific expertise with a strong commitment to translational impact, guiding researchers in model validation, regulatory awareness, and therapeutic strategy.
She is an active board member of the Dutch Society of Pharmacology and contributes to national consortia including RARE-NL and hDMT (Human Disease Model Technologies), where she advocates for improved integration between model development and clinical application.
Presentation: Translating Academic Potential into Patient Impact
Across the Netherlands, many academic groups are making important strides in the preclinical development of therapies for rare diseases: ranging from disease modeling and target discovery to in vitro validation of therapeutic approaches.
Unfortunately, there is a large gap between academic drug development and the pathway to clinical implementation and as a result many promising findings never reach the clinic. The Radboudumc Therapy accelerator for rare diseases, RARE-NL and hDMT (Human Disease Model Technologies) are now joining forces to create a knowledge platform that facilitates therapy development for rare diseases. Topics will range from early attention to model validation to regulatory requirements and stakeholder engagement. We invite all stakeholders within the therapy development pathway to join and share their expertise.
In this presentation, I will highlight current gaps in academic rare disease therapy development and possible steps that can be taken to promote translation of preclinical work—particularly in the rare disease field, where time, resources, and patient populations are limited.
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