Primary immunodeficiencies (PID) are rare genetic diseases of the immune system, predisposing patients to infections, allergy, autoimmunity and cancer.
This project will utilize novel cell and gene therapy approaches to rebuild the healthy immune system of these patients. CELL-PID researchers will study innovative ways to improve the regeneration of the immune system and will perform rigorous preclinical efficacy and toxicology evaluation of the newly developed therapies.
We will carry out multi-centre clinical trials for different variants of PID, using gene transfer technology to correct patient's hematopoietic stem cell and lymphocytes.
The consortium includes clinical centres, scientists and industrial partners pioneering in the field of advanced therapies and aiming at broad clinical application of cell-based therapeutic products.