Cell and Gene Therapy Facility

The Zayed Centre for Research Good Manufacturing Practice facility supports research evidence generation and the manufacture of cell and gene therapies for children with rare diseases and cancer. It offers investigators and partners a regulated environment, specialist manufacturing and quality expertise, and a structured route from early project scoping through regulatory approval and delivery.

The Cell and Gene Therapy Facility is a manufacturing site for advanced therapy medicinal products (ATMPs) licensed by the Medicines and Healthcare products Regulatory Agency.

It is comprised of 7 bespoke laboratories and provides comprehensive facilities for all stages of manufacture. This includes tissue culture labs, storage, waste treatment and quality control. This work is labour intensive, highly regulated and innovative. The facility also has a simulation suite that is used for education and training.

Cell and Gene Therapy products are manufactured specifically for each patient and often have a shelf-life of only a few hours, therefore manufacture on-site at Great Ormond Street Hospital (GOSH) can be crucial in delivering these treatments to patients.

The laboratories are based at the hospital and The Zayed Centre for Research (ZCR) into Rare Disease in Children. The Centre, located next to GOSH, uniquely brings together pioneering research and clinical care under one roof to help drive forward new treatments and cures for children with rare diseases.

The work of the NIHR GOSH Biomedical Research Centre is also integral to facilitate research and development of cell and gene therapies produced at GOSH.

• Regulatory status: Licensed by the MHRA in January 2023 for ATMP and viral vector manufacture.
• Core infrastructure: 7 cleanroom laboratories, QC and tissue culture, and a process validation suite.
• Specialist storage: Dedicated liquid nitrogen storage for cryopreserved products.
• Capacity: Capacity for up to 150 products per year, including routine cell therapy laboratory activities and viral manufacture.
• Delivery models: Projects can engage through contract manufacturing or through access to the GMP laboratories.
• End-to-end support: Support spans project management, technology transfer, validation, manufacturing, testing, release and regulatory activity.