Smart biomanufacturing for genomic medicines

基因组药物的智能生物制造

• 批准号: EP/X025446/1
• 负责人: Daniel Bracewell
• 金额: $ 159.95万
• 依托单位: University College London
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2024
• 资助国家: 英国
• 起止时间: 2024 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=EP%2FX025446%2F1
• 关键词: Smart; biomanufacturing; genomic; medicines

This partnership builds on the existing Pall-UCL Centre of Excellence collaboration which is carrying out fundamental research to address biomanufacturing industry needs. It brings together Pall (a global integrated separations solutions supplier to the Life Sciences and industrial sectors) and UCL Biochemical Engineering (a world leader in bioprocess research creating novel engineering solutions to underpin future biomanufacturing operations across a range of sectors) in a research collaboration to address the challenges of manufacturing next generation biological medicines. These therapies are made possible by the delivery of genetic molecules to the site of disease to control protein production in cells and tissue. The current toolbox of genetic molecules includes small interfering ribonucleic acid (siRNA), messenger RNA (mRNA), self-amplifying RNA (SAM), deoxyribonucleic acid (DNA) and gene editing components such as guide RNA (gRNA), ribonucleoprotein (RNP) and nucleases. Delivery is made possible using a variety of vectors including lipid nanoparticles, virus and extracellular vesicles / exosomes. This field of personalised genetic medicine has seen unprecedented growth in over the last decade. In 2021 the gene therapy global market was estimated at £622M with 25% year on year growth and over £10 billion invested in development of the sector.The UK is a world leader in life science discovery but traditionally this does not translate into revenue generation for the UK via manufacture. This generation of new more complex biological therapies offers the opportunity for the UK to convert its lead in medicines discovery to manufacturing. To take these therapies to market will require specialist NHS-type resource for proof of principle and the scale of manufacture of these new therapies is particularly suited to Pall's specialisation in small-scale rapid response units for manufacture. The Prosperity Partnership is designed to help complete the continuum from life science discovery to manufactured therapies for the patient bringing together UCL bioprocess discoveries and Pall UK's specialism in novel manufacturing units around these new classes of complex personalised biological medicines.The partnership will be managed by a joint Pall-UCL management committee led by Dr John Welsh for Pall and Prof. Daniel Bracewell for UCL. The committee is constituted of senior leaders with the necessary experience to steer the partnership during its lifetime and update the focus of the research with the fast-moving nature of the bioprocessing sector.

这种伙伴关系建立在现有的PALL-UCL卓越合作中心，该中心正在进行基础研究以满足生物制造业需求。它汇集了PALL（全球综合分离解决方案供应商的生命科学和工业领域）和UCL生物化学工程（世界生物过程的世界领导者（世界领域的世界领导者），创造了新的工程解决方案，以支撑着未来生物制造业务的新工程解决方案），以应对制造下一代生物学的挑战，以应对众多领域。通过将遗传分子递送到疾病部位以控制细胞和组织中的蛋白质产生，使这些疗法成为可能。遗传分子的当前工具箱包括小干扰核糖酸（siRNA），Messenger RNA（mRNA），自我扩增的RNA（SAM），脱氧核糖核酸（DNA）和基因编辑成分，例如指南RNA（GRNA），核糖核酸蛋白酶蛋白（RNP）和核心。使用包括脂质纳米颗粒，病毒和细胞外蔬菜 /外泌体在内的多种媒介，可以进行分娩。在过去的十年中，这个个性化的遗传医学领域已经实现了前所未有的增长。 2021年，基因治疗全球市场估计为6.22亿英镑，同比增长25％，超过100亿英镑用于该领域的发展。英国是生命科学发现的世界领导者，但传统上，这并不能够通过制造业转化为英国的收入。这一代新的复杂的生物疗法为英国提供了将其在药品发现中的铅转换为制造业的机会。将这些疗法用于市场将需要专业的NHS型资源来证明原理，并且这些新疗法的制造规模尤其适合Pall在小型快速响应单元中进行制造的专业化。繁荣合作伙伴关系旨在帮助完成从生命科学发现到制造疗法的连续性，用于将UCL BioProcess发现和PALL UK在这些新型复杂的个性化生物学药物的新型制造部门中的专业中汇总在一起。该委员会由高级领导人组成，具有必要的经验，可以在其一生中引导合作伙伴关系，并以生物处理部门的快速发展性质更新研究的重点。