King's/Royal Free/UCL Gene Therapy Innovation Hub

国王/皇家自由/UCL基因治疗创新中心将为英国学术和临床社区生产临床级基因疗法。这将允许对广泛的罕见和常见疾病进行有希望的治疗，以便在患者中进行测试。这些早期临床研究的结果可能会支持更大规模的试验，并最终为患者提供新的治疗方法。提供合适的质量（GMP）基因治疗产品是这一令人兴奋的领域取得进展的关键限制因素。我们的枢纽将通过大幅增加英国的产能直接解决这一问题。这将涵盖所使用的主要类型的基因治疗，腺相关病毒（AAV）和慢病毒，以及γ-逆转录病毒。此外，我们将投资开发新方法，以增加一次（一批）可以生产的基因治疗产品的数量。这将意味着每次试验需要更少的批次，并且需要高剂量的应用（例如，全身给药）成为可能。它还将降低制造成本。我们还将努力提高英国制造基因疗法的整体能力，通过创建和提供各种级别的在线和面对面的专门培训课程，以满足不同群体的不同需求。除此之外，我们还将向其他正在发展的基因治疗创新中心提供我们的专业知识，以帮助他们尽快投入运营。国王，皇家免费和伦敦大学学院提供无与伦比的专业知识在英国的基因治疗制造，我们是理想的位置，以促进英国的成功，改善治疗许多治疗不善的疾病，并产生可持续的经济效益为国家。

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.