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

国王/皇家自由/伦敦大学学院基因治疗创新中心将为英国学术和临床社区制造临床级基因疗法。这将使有希望的治疗多种罕见和常见疾病的方法能够在患者身上进行测试。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。提供合适质量的基因治疗产品是限制这一激动人心的领域取得进展的关键因素。我们的中心将通过大幅增加英国的运力来直接解决这个问题。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，我们将投资开发新的方法，以增加一次（批量）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的批次更少，并且需要高剂量的应用（例如系统给药）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，通过创建和提供专门的培训课程，包括在线和面对面的，在不同的层次，以满足不同群体的不同需求。除此之外，我们还将把我们的专业知识借给其他正在发展的基因治疗创新中心，帮助它们尽快投入运营。国王学院、皇家自由学院和伦敦大学学院在英国基因治疗制造方面提供了无与伦比的专业知识，我们处于理想的位置，可以为英国在改善许多治疗不善的疾病的治疗方面取得成功做出贡献，并为国家带来可持续的经济效益。

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.