The Zayed Centre for Research into Rare Disease in Children (ZCR) is currently under construction in Guildford Street. Importantly this will include a dedicated facility comprising seven ‘cleanrooms’ where gene and cell therapy medicinal products can be made to treat children compassionately or on a clinical trial.
Currently GOSH has two ‘cleanrooms’ which are licensed by the Medicines and Healthcare products Regulatory Agency (MHRA) as compliant with “Good Manufacturing Practice (GMP)” and one ‘cleanroom’ for routine bone marrow manipulations licensed by the Human Tissue Authority (HTA). However, these rooms are close to maximum capacity at present. As so many new advanced therapy products are being developed to treat an ever wider range of diseases, the new ZCR GMP will provide essential new capacity by 2018, when the facility opens. It is not easy to predict exactly what new treatments will become available, but so far these include: severe combined immunodeficiencies, different types of leukaemia, whole tumours, skin disorders and viral infection post bone marrow transplant.