Engineered Genetic Control Systems for Advanced Therapeutics

本申请涉及生物医学工程生物学，特别是基因治疗（GT）领域。细胞和基因疗法（CGT）有可能彻底改变医疗保健，并且由于最近的进展和未来的可能性，可以说是生物技术中最令人兴奋的领域。基因治疗是一种通过改变一个人的基因来治疗或治愈疾病的技术。基因疗法可以通过几种机制起作用：用健康的基因拷贝取代致病基因，使功能不正常的致病基因失活，或者引入新的或修饰的基因来帮助治疗疾病。GT的主要递送机制是病毒载体，包括AAV和慢病毒，其中病毒已被修饰以去除它们引起感染性疾病的能力，并在它们的基因组中释放空间以插入遗传"货物"，从而使治疗基因能够被携带到人类细胞中。CGT的出现在重塑生物制药行业方面发挥了重要作用，并改变了一系列生命的治疗模式。威胁性和罕见的疾病。2022年，全球基因治疗市场价值为63. 6亿美元，这是由于地球仪上遗传疾病的识别和流行率不断增加。市场规模预计将在2023 - 2031年期间以22.8%的复合年增长率增长，到2031年实现403.9亿美元的价值。为了使基因治疗有效和安全，它们需要在正确的组织中以正确的水平表达转基因，持续正确的时间，并有效地传递到正确的组织。工程生物学是解决这些挑战的完美技术，我们的使命是利用工程生物学开发一系列用于基因治疗的工程遗传控制系统，既用于控制治疗性转基因的表达，也用于开发增强型递送系统。该中心将汇集来自爱丁堡大学，牛津大学，伦敦帝国理工学院伦敦和Beatson癌症研究所的多学科团队，开发一套新的工程生物学工具，用于控制转基因表达，交付到细胞中，并在三个应用领域进行测试-肿瘤学，心血管疾病和罕见疾病。

This application addresses engineering biology for biomedicine specifically in the area of gene therapy (GT). Cell and gene therapies (CGTs) have the potential to revolutionise healthcare and are arguably the most exciting areas of biotechnology both due to recent progress and future possibilities. Gene therapy is a technique that modifies a person's genes to treat or cure disease. Gene therapies can work by several mechanisms: Replacing a disease-causing gene with a healthy copy of the gene, inactivating a disease-causing gene that is not functioning correctly or introducing a new or modified gene to help treat a disease. The main delivery mechanisms for GTs are viral vectors including AAV and lentivirus where the virus has been modified to remove their ability to cause infectious disease and free up space in their genome for insertion of genetic "cargo" enabling therapeutic genes to be carried into human cells.The emergence of CGTs has played a major role in reshaping the biopharmaceutical industry and has transformed the treatment paradigm of a range of life-threatening and rare diseases. The global gene therapy market value was USD 6.36 billion in 2022 driven by the increasing identification and prevalence of genetic disorders across the globe. The market size is anticipated to grow at a CAGR of 22.8% during 2023-2031 to achieve a value of USD 40.39 billion by 2031. For gene therapies to be effective and safe they need to express the transgene in the right tissue, at the right level, for the right amount of time and to be delivered efficiently to the correct tissues. Engineering Biology is the perfect technology to address these challenges and our mission is to use Engineering Biology to develop a series of engineered genetic control systems for use in Gene Therapies both in the control of expression of the therapeutic transgene but also in the development of enhanced delivery systems. This Hub will bring together a multidisciplinary team from the Universities of Edinburgh, Oxford, Imperial College London and the Beatson Cancer Research Institute to develop a new suite of engineering biology tools for control of transgene expression, delivery into cells and test them in three application spaces - oncology, cardiovascular disease and rare diseases.