A new gene therapy manufacturing hub in London will produce clinical-grade treatments for UK researchers, turning promising lab discoveries into medicines that can be tested in patients. The problem is straightforward: many academic gene therapy projects stall because researchers cannot get enough high-quality virus particles to run a clinical trial. The hub will make adeno-associated virus (AAV), lentivirus, and gamma-retrovirus—the three main delivery vehicles for gene therapies—under strict manufacturing standards. It will also invest in methods to produce larger batches, which matters because some diseases require high doses that current batch sizes cannot supply. If the hub succeeds, it will remove a major bottleneck in the UK’s gene therapy pipeline. More early-stage trials can proceed, generating the data needed to attract investment for larger studies and eventual regulatory approval. The hub will also train new manufacturing staff and help other UK innovation hubs get started, building national capacity that outlasts any single project. The economic benefit is indirect but real: a self-sustaining gene therapy sector that can develop treatments for rare and common diseases without relying on overseas manufacturers.
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The Kings/Royal Free/UCL Gene Therapy Innovation Hub will manufacture clinical-grade gene therapies for the UK academic and clinical community. This will allow promising treatments for a wide range of rare and common diseases to be tested in patients. The results from these early clinical studies may then support larger-scale trials and ultimately new therapies for patients. Provision of suitable quality (GMP) gene therapy product is a key limiting factor for progress in this exciting field. Our Hub will address this directly through a major increase in UK capacity. This will cover the major types of gene therapy used, adeno-associated virus (AAV) and lentivirus, as well as gamma-retrovirus. In addition, we will invest in developing new approaches to increase the amount of gene therapy product that can be made at one time (in one batch). This will mean that each trial needs fewer batches, and that applications needing high doses (for example, administration systemically) become possible. It will also reduce costs of manufacture. We will also work to increase the UK's overall capabilities to manufacture gene therapies, through creation and provision of dedicated training courses, both online and in person, at a variety of levels, to suits varying needs of different groups. In addition to this, we will lend our expertise to other developing Gene Therapy Innovation Hubs, to help them to become operational as quickly as possible. King's, Royal Free and UCL offer unmatched expertise in the UK in gene therapy manufacturing, and we are ideally-placed to contribute to the UK's success in improving therapies for many poorly-treated diseases, and generating sustainable economic benefit for the country.
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