Correcting a genetic defect in the patient's blood stem cells can mean a lifelong cure for a number of rare genetic diseases. Unfortunately, this kind of fully curative gene therapy often doesn't reach patients, and not because the therapy wouldn't work. This consortium will develop a platform infrastructure that can be used for many different diseases, instead of focusing on one approach that is uniquely suited to a single disease. At the same time, they address challenges related to the regulation, reimbursement and public acceptance of these therapies in a knowledge center for dialogue with society. This program can have a significant and stimulating impact on the gene therapy research that is performed in the CCB department.
The program is coordinated by Prof. Dr. F.J.T. (Frank) Staal of the Immunology department of the Leiden University Medical Center (LUMC). Many groups will act in concert in the consortium: Leiden University Medical Center, Erasmus MC, VU University Amsterdam, Utrecht University, Leiden University, Avans University of Applied Sciences, University Medical Center Utrecht, Radboud University Medical Center, Radboud University Nijmegen, Batavia Biosciences, ISHEO, Netherlands Muscle Diseases, Princess Beatrix Muscle Fund, Miltenyi , Oscar Nederland, Foundation for Defense Disorders (SAS), VKS, Foundation Alexander, Zorginstituut Nederland, IPOPI - International Patient Organization for Primary Immunodeficiencies. The total amount allocated for the program is 5.0 million euros.