GOSH receives new MHRA authorisation to manufacture viral vectors

Cell and gene therapies (CGT) are at the cutting-edge of treatment options for cancer, immunodeficiencies, some blood disorders, metabolic disorders and more. For cancer, they hold promise to offer personalised treatments, including for rare forms that sadly affect children and young people.

Viral vectors are harmless viruses that we can use to ‘trick’ cells in to accepting new genes and are commonly used by CGT innovators to genetically modify human cells to create novel treatments. They are highly complex to make, and their availability and effectiveness dictates whether a clinical trial of gene or cell therapy will work. An industry-wide bottleneck, both in the UK and globally, has resulted in a shortage of viral vector supply, which is believed to be slowing the progress of bringing novel medicines to patients.

In 2021 GOSH announced a partnership with ViroCell Biologics (ViroCell), an innovation-driven Contract Development and Manufacturing Organisation (CDMO) who focus exclusively on the design and GMP manufacture of viral vectors and gene modified cells for clinical trials. ViroCell is focused where the viral vector design and Good Manufacturing Practice (GMP) manufacturing bottleneck is most acute: the zone between pre-clinical concept and pivotal clinical trials. ViroCell is therefore filling the gap between 'small volume' academic core labs and 'large volume' CDMOs.

Granted by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) to GOSH, the new licence allows ViroCell to leverage GOSH's state-of-the-art manufacturing facility to manufacture and globally export viral vectors for use in clinical trials. The production of the vectors will take place in four of the specialist clean rooms at GOSH’s Zayed Centre for Research which was established in 2019 as the UK’s largest single academic GMP accredited unit for manufacturing CGTs. This begins the unclogging the industry-wide bottleneck and acceleration of CGT clinical trials both in the UK and globally, increasing the new specialised treatments that we can offer to our patients here at GOSH.

Thank you to all our staff at the GOSH GMP Cell and Gene Therapy Service whose hard work continues to position GOSH and the UK as a hub for life science innovation.

“We are delighted that GOSH has been granted MHRA licensing for the manufacture of viral vectors. With our state-of-the-art facility and ViroCell’s international network of collaborators, vectors for both UK and global projects can be manufactured at the Zayed Centre for Research, unclogging the industry-wide bottleneck, accelerating cell and gene therapy clinical trials and expanding the novel treatments that we can offer to our patients.”

Professor Claire Booth, Mahboubian Professor in gene therapy and paediatric immunology at GOSH and UCL GOS ICH, and Clinical Academic Lead for the Cell and Gene Therapy Service at GOSH

“Addressing the global viral vector supply/demand imbalance is a top priority for Team ViroCell as the scarcity of high-quality viral vectors has constrained the ability of innovators to manufacture their novel cell and gene therapies. This approval is an exciting milestone for ViroCell and GOSH, which should catalyse our next phase of growth as we unlock ViroCell’s full potential as the partner of choice for cell and gene therapy companies demanding precision-engineered viral vectors produced to GMP standards.

"We highly value the MHRA’s collaboration and look forward to strengthening our partnership with GOSH as we work to help accelerate clinical trials for advanced therapy patient candidates around the world."

John W. Hadden II, CEO at ViroCell

The Cell and Gene Therapy Facility at GOSH is a manufacturing site licenced by the Medicines and Healthcare products Regulatory Agency (MHRA) for manufacture of advanced therapy medicinal products (ATMPs).

It is comprised of seven 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.

In January 2023, GOSH opened additional facilities for manufacturing gene and cell therapies, having received MHRA licensing for the rooms based at the Zayed Centre for Research. These facilities are also known as Good Manufacturing Practice (GMP) facilities. It is the largest single academic GMP accredited unit in the UK and one of the largest in the world for manufacturing of ATMPs, with seven clean rooms and a simulation centre for practice and education.