Gene and Cell Therapy Facility

This facility supports complex cell and gene therapy clinical trials through provision of manufacturing sites of gene and cell therapy medicinal products.

The Gene and Cell Therapy Facility is comprised of two clean rooms for the manufacture of gene and cell therapy medicinal products. The facility supports research by allowing rapid translation of cutting-edge research into clinical treatments as part of phase I/II clinical trials and ‘off-trial’ treatments for children with rare diseases. This work is labour intensive, highly regulated and innovative.

This resource is invaluable, as manufacture is complex and fast admission to the patient is often required to treat our patients.

Gene and cellular therapy products are manufactured specifically for each of our patients and often have a shelf-life of only a few hours. Manufacture on-site at Great Ormond Street Hospital (GOSH) is crucial in delivering these treatments and better care to patients with a wide range of rare inherited and acquired disorders, including immunological disorders, cancers and infectious diseases, as well as supporting new transplantation methods and technologies.

About our work

Recently, the completion of a cell therapy clinical trial involving manufacture of liver stem cells for the treatment of metabolic disease was achieved in collaboration with a leading European biotech company and Birmingham Children’s Hospital. This is improving health outcomes for children with liver diseases. Gene therapy trials for Netherton syndrome using genetically modified skin cells have also recently opened, made possible by the expertise that is supported and developed by our BRC.

The Gene and Cell Therapy Facility provides a quality assured interface with industrial partners that require the manufacture of gene modified cell products for specific gene therapy studies. It also acts as a local and national hub for specialised training and for manufacture of personalised cell and gene therapies.

In early 2014, the BRC committed a further £750,000 to investigate strategies to improve the efficiency and effectiveness of T-cell modification with co-funding from Miltenyi Biotech.

This collaboration will drive investigation and development of an automated or part-automated process for production of gene and cellular therapy products for use in patients. The project will support the evolution of the Gene and Cell Therapy Facility in becoming a major European hub for the delivery of cell and gene therapy technologies