NHSBT Innovation Hub for Gene Therapies

NHSBT 基因治疗创新中心

• 批准号: MR/V030175/1
• 负责人: Jon Smythe
• 金额: $ 601.07万
• 依托单位: NHS Blood and Transplant
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2021
• 资助国家: 英国
• 起止时间: 2021 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=MR%2FV030175%2F1
• 关键词: NHSBT; Innovation; Hub; Gene; Therapies

Many diseases, including cancer, heart disease and diabetes, are caused by the body's cells and tissues malfunctioning. The behaviour of any cell is 'programmed' by its genes and researchers have found that cell behaviour can be altered by changing DNA, RNA and other genetic material in the cell. Gene therapies work by inserting new genetic material into malfunctioning cells, and 'reprogramming' them to function more normally, or by inserting genetic material into a normal cell to produce a new protein (e.g. for a vaccine). Gene therapy offers hope to patients with diseases that have been, up to now, incurable. The UK has excellent teams of researchers who are exploring this exciting topic.Unfortunately, some of the essential materials for gene research are in short supply in the UK, as are skilled technicians to work on the research. There is a particular shortage of viral vectors - special viruses that can be 'loaded' with genetic material and inserted into malfunctioning cells, where their 'payload' replaces genes causing the malfunction. The UK's scientists are held back by these shortages and forced to rely on overseas suppliers; or research is delayed due to difficulties in recruiting staff. This leads to delays in the discovery of new treatments for desperately ill patients.Another important challenge for UK researchers is that achieving great results in the lab is only half the story - before a new treatment can be offered to patients, it must be approved: shown to be safe, effective and affordable. These new treatments are great opportunities for new companies, new jobs and for the UK economy, so academics need support in patenting their discoveries, in undertaking clinical trials and in setting up companies to make new therapies.The MRC and LifeArc have recognised these important issues and are funding a network of hubs around the UK to generate the vital components for gene therapies and to train the skilled personnel needed. The NHSBT Innovation Hub for Gene Therapies will link with the other new hubs around the UK to address these needs. We will work with academic teams to produce the gene therapy components needed, we will train technicians and scientists in producing these at the quality needed for use in clinical trials, and we will support academics in taking their results from the lab to the hospital and the market. Our plan is as follows:1. To agree our role in the network with the other hubs, MRC, LifeArc and Cell and Gene Therapy Catapult (C>C). We are particularly well placed for flexible viral vector production, quality control and training, and we expect to contribute this to the network, while also collaborating with C>C on production, with LifeArc on commercialisation and patenting, and with other hubs, as requested by the network coordinators..2. To install a new viral vector production platform, already selected by C>C as being particularly suitable to meet the needs of UK academic teams. We'll then work with the C>C to ensure it's certified for quality assurance (and so ready to produce clinical grade components). This new platform will produce a specific type of viral vector - adeno-associated-viral vectors (AAVs); this will complement our other platform, producing lentiviral vectors, as well as our existing platform for plasmids (another important gene therapy ingredient).3. To be assigned academic clients and their projects by the coordinating committee, and then to work with these teams to put in place a production line and training programme that meets their needs. A flexible and customised approach is important, as every academic project will be different.4. To build a reputation and customer base that makes the NHSBT Hub a 'go to' destination for viral vector services. As a not-for-profit NHS organisation, our aim is to be sustainable, provide excellent value for the benefit of the UK gene therapy community and, ultimately, to save and improve more lives.

许多疾病，包括癌症、心脏病和糖尿病，都是由身体细胞和组织功能失调引起的。任何细胞的行为都是由其基因“编程”的，研究人员发现，可以通过改变细胞中的 DNA、RNA 和其他遗传物质来改变细胞行为。基因疗法的工作原理是将新的遗传物质插入到故障细胞中，并对它们进行“重新编程”以使其功能更正常，或者将遗传物质插入到正常细胞中以产生新的蛋白质（例如用于疫苗）。基因疗法为迄今为止无法治愈的疾病患者带来了希望。英国拥有优秀的研究团队正在探索这个令人兴奋的课题。不幸的是，英国缺乏基因研究的一些必需材料，以及从事这项研究的熟练技术人员。病毒载体尤其短缺，这种特殊病毒可以“装载”遗传物质并插入到故障细胞中，其“有效负载”取代导致故障的基因。英国科学家因这些短缺而受阻，被迫依赖海外供应商；或由于招聘人员困难而导致研究被推迟。这导致了为重症患者发现新疗法的延迟。英国研究人员面临的另一个重要挑战是，在实验室取得出色成果只是故事的一半——在向患者提供新疗法之前，必须获得批准：被证明是安全、有效和负担得起的。这些新疗法对于新公司、新就业机会和英国经济来说都是巨大的机遇，因此学术界需要支持为其发现申请专利、进行临床试验以及成立公司来生产新疗法。MRC 和 LifeArc 已经认识到这些重要问题并正在资助英国各地的中心网络，以生产基因疗法的重要组成部分并培训所需的技术人员。 NHSBT 基因治疗创新中心将与英国各地的其他新中心建立联系，以满足这些需求。我们将与学术团队合作生产所需的基因治疗成分，我们将培训技术人员和科学家以临床试验所需的质量生产这些成分，我们将支持学术界将他们的结果从实验室带到医院和医院。市场。我们的计划如下： 1．与其他中心、MRC、LifeArc 和 Cell and Gene Therapy Catapult (C>C) 就我们在网络中的角色达成一致。我们在灵活的病毒载体生产、质量控制和培训方面处于特别有利的地位，我们希望将其贡献给网络，同时还与 C>C 进行生产合作，与 LifeArc 进行商业化和专利合作，并根据要求与其他中心合作由网络协调员..2。安装新的病毒载体生产平台，该平台已被 C>C 选择为特别适合满足英国学术团队的需求。然后，我们将与 C>C 合作，确保其获得质量保证认证（以便准备生产临床级组件）。这个新平台将生产一种特定类型的病毒载体——腺相关病毒载体（AAV）；这将补充我们生产慢病毒载体的其他平台，以及我们现有的质粒平台（另一种重要的基因治疗成分）。3。由协调委员会分配学术客户及其项目，然后与这些团队合作建立满足他们需求的生产线和培训计划。灵活和定制的方法很重要，因为每个学术项目都会有所不同。4。建立声誉和客户群，使 NHSBT 中心成为病毒载体服务的“首选”目的地。作为一家非营利性 NHS 组织，我们的目标是可持续发展，为英国基因治疗界的利益提供卓越的价值，并最终拯救和改善更多的生命。