HEAL-PCD
Human models to enable advanced lung therapy for Primary Ciliary Dyskinesia and beyond
Bahar Yetkin-Arik, UMCU Tamara Paff, UMCU Gimano Amatngalim, UMCU Jeffrey Beekman, UMCU Spirovant
Pacira Germany GmbH
This project brings together researches, clinicians, and industry partners to develop new gene therapies for Primary Cilliary Dyskinesia (PCD), a rare inherited lung disease.
PCD is caused by changes in genes that are important for the function of tiny hair-like structures called cilia. When cilia do not work properly, mucus and germs are not cleared from the lungs, leading to repeated infections, breathing problems and, over time, lung damage. Current treatments mainly manage symptoms, and there is no cure yet.
The project aims to develop new therapies that can restore cilia function. Researchers will create airway cell models using cells from people with PCD. These models will be used to test and improve potential gene therapies in the laboratory before they are considered for clinical studies.
The team is also developing a standardized testing platform that can measure how well cilia function is restored. This will make it possible to compare different therapies and identify the most promising approaches.
The project combines the expertise of UMC Utrecht, Spirovant and Pacira Germany in airway biology, patient-derived cell models and gene therapy. This public-private collaboration aims to accelerate the development of new treatments and bring effective gene therapies for PCD closer to patients.
