21EBTA Engineering Biology for Cell and Gene Therapy Applications

细胞疗法和基因疗法（cgt）是生物医学研究和治疗的相关领域，旨在治疗、预防和潜在地治愈疾病。细胞疗法的目的是通过恢复或改变某些细胞群或利用细胞在体内进行治疗来治疗疾病。基因疗法旨在通过将基因替换、灭活或导入患者细胞来治疗疾病。细胞疗法和基因疗法的重叠之处在于，它们都需要将新的遗传物质转移到细胞中，以产生可以被认为是“活的药物”。目前最常用的细胞疗法是嵌合抗原受体（CAR） t细胞疗法。这包括对患者自身的T细胞进行基因修饰，以表达针对肿瘤抗原的CAR特异性，然后进行体外细胞扩增并重新输注回患者体内，使工程T细胞能够识别癌细胞并摧毁它们。2020年，全球细胞和基因治疗制造市场规模为131亿美元，预计从2021年（170亿美元）到2028年（574亿美元）将以20.3%的复合年增长率（CAGR）扩大。尽管这些疗法前景光明，但它们对剂量、时间或定位的控制有限，而且往往昂贵得令人望而却步。这些缺点可以通过使用工程生物学来创造下一代细胞和基因疗法来克服。我们将利用我们独特的自动化设备开发新的工程生物学工具，解决生产cgt的瓶颈，并为未来的临床应用提供新的，廉价和安全的治疗方法。该研究将分为四个工程生物学工作包：1。2.遗传控制技术在生物工程中的应用。3.传递机制；4.细胞系标准化；负责任的研究和创新。

Cell therapy and gene therapies (CGTs) are interrelated areas of biomedical research and treatment that aim to treat, prevent, and potentially cure diseases. Cell therapy aims to treat diseases by restoring or altering certain sets of cells or by using cells to carry a therapy through the body. Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into a patient's cells. Both cell and gene therapies overlap in that they necessitate the transfer of new genetic material to cells to produce what could be thought of as "living medicines". The most commonly used cell therapy at present is Chimeric Antigen Receptor (CAR) T-cell therapy for treating blood cancers. This involves genetic modification of patient's own T-cells to express a CAR specific for a tumour antigen, following by ex vivo cell expansion and re-infusion back to the patient enabling to the engineered T Cell to identify cancer cells and destroy them. The global cell and gene therapy manufacturing market size was valued at USD 13.1 billion in 2020 and is expected to expand at a compound annual growth rate (CAGR) of 20.3% from 2021 ($17B) to 2028 ($57.4B). Despite their promise, these therapies are limited providing little control over their dosage, timing, or localization and are often prohibitively expensive. These shortcomings can be overcome by using Engineering Biology to create the next generation of cell and gene therapies. We will use our unique automated facilities to develop new engineering biology tools, and solutions for the bottlenecks in the production CGTs and enable new, inexpensive and safe therapies for future clinical applications. The research will be split into four Engineering Biology Work Packages:1. Genetic Devices for Control in CGTs, 2. Delivery Mechanisms, 3. Standardisation of Cell Lines and 4. Responsible Research and Innovation.