Stem cell models for personalized treatment of rare genetic diseases

This project, a collaboration between UMC Utrecht and FAIR Therapeutics, focused on developing patient-derived organoid models to improve drug development for cystic fibrosis (CF) and other rare genetic diseases. Using airway and intestinal organoids grown from patient cells, the team successfully optimized assays to study how different CFTR modulators work, including novel compounds developed by FAIR Therapeutics. 

Comparative studies with patient-matched organoids deepened understanding of CFTR responses and laid a solid foundation for future research in CF and beyond. Importantly, nasal organoids were validated as a non-invasive model to predict drug responses in the lungs, reducing the need for invasive procedures in patients. 

The project also expanded to other rare diseases, optimizing culture conditions for kidney and airway models, which opens new research and treatment possibilities for conditions like primary ciliary dyskinesia and renal cystic diseases. 

These advances support more cost-effective drug development by improving predictability of clinical outcomes, reducing failure rates, and enabling better patient selection. This not only benefits patients through more personalized treatment options and improved health outcomes but also creates new market opportunities for organoid-based assays and therapies, strengthening FAIR Therapeutics’ competitive position.