Smart biomanufacturing for genomic medicines

这种伙伴关系建立在现有的Pall-UCL卓越合作中心的基础上，该中心正在开展基础研究，以满足生物制造行业的需求。它汇集了Pall（生命科学和工业领域的全球综合分离解决方案供应商）和UCL生化工程（生物工艺研究的世界领导者，创造新的工程解决方案，以支持未来的生物制造业务在一系列部门）在研究合作，以解决制造下一代生物药物的挑战。这些疗法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而成为可能。目前的遗传分子工具箱包括小干扰核糖核酸（siRNA）、信使RNA（mRNA）、自扩增RNA（SAM）、脱氧核糖核酸（DNA）和基因编辑组分，如向导RNA（gRNA）、核糖核蛋白（RNP）和核酸酶。使用包括脂质纳米颗粒、病毒和细胞外囊泡/外来体的多种载体使递送成为可能。在过去的十年中，个性化基因医学领域出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%，用于该行业发展的投资超过100亿英镑。英国是生命科学发现的世界领导者，但传统上这并不转化为英国通过制造的收入。这一代新的更复杂的生物疗法为英国提供了将其在药物发现方面的领先地位转化为制造的机会。将这些疗法推向市场将需要专门的NHS类型资源来证明原理，并且这些新疗法的生产规模特别适合Pall在小规模快速反应单元生产方面的专业化。繁荣伙伴关系旨在帮助完成从生命科学发现到为患者制造治疗的连续统一体，将UCL生物工艺的发现与Pall UK在这些新型复杂个性化生物药物的新型制造单元方面的专业知识结合在一起。该伙伴关系将由Pall-UCL联合管理委员会管理，由John Welsh博士领导Pall和UCL的丹尼尔Bracewell教授领导。该委员会由具有必要经验的高级领导人组成，以在其生命周期内指导伙伴关系，并根据生物加工行业的快速发展性质更新研究重点。

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.