Building a UK IntraCellular Drug Delivery Centre (ICDDC)

建设英国细胞内药物输送中心（ICDDC）

• 批准号: 10058505
• 金额: $ 1274.2万
• 依托单位: CENTRE FOR PROCESS INNOVATION LIMITED
• 依托单位国家: 英国
• 项目类别: Centres
• 财政年份: 2023
• 资助国家: 英国
• 起止时间: 2023 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=10058505
• 关键词: Building; UK; IntraCellular; Drug; Delivery

Ribonucleic acid (or RNA) based therapy is the treatment or prevention of diseases using RNA-based molecules. The recent sucess of messenger RNA vaccines in response to the COVID-19 pandemic has highlighted the potential of this technology and greatly increased research and industrial interest. RNA offers the potential to produce almost any functional protein or peptide in the human body by introducing mRNA as a vaccine or therapeutic agent.RNA therapeutics and vaccines often require specific, targeted delivery to be effective medicines, and a leading technology platform is Lipid Nanoparticles, along with alternative Nano Delivery Systems. This applies to a range of RNA based therapies such as mRNA/self-amplifying RNA, siRNA, antisense oligos, as well as cutting-edge and potentially curative therapeutic approaches such as gene editing technology, via CRISPR/Cas9, base or prime editing.There is an opportunity to "anchor" commercial scale design and manufacturing of these new classes of therapies in the UK and act as a global hub for research, development activities and trials -- but given the early stage of this technology it requires investment in early to mid-stage R&D to solve scientific, manufacturing, clinical and commercial challenges. Challenges to address include: creating more thermostable medicines; understanding and controlling immunogenicity and adverse reactions, developing better characterisation methods; more efficient and high productivity process development and manufacturing; and targeting a range of different organs and cell types to enable the treatment of a wider range of diseases.We are proposing to upgrade existing facilities to create state of the art capability, along with an ambitious, large scale research programme focused on building the knowhow to create disruptive innovation in the field of product design, formulation, manufacture and characterisation. This includes the ability to rationally design next generation LNPs, create a step change in the area of in vitro methods to predict drug/ vaccine human clinical response, and finally develop a digitally enabled LNP manufacturing process that will act as a lighthouse project for advanced manufacture in Pharma.With public investment we will create the knowhow and capability to catalyse an explosion of R&D investment in the UK - increasing the translation of our high quality academic base, boosting industrial R&D, clinical trials and ultimately creating a new ecosystem via company growth, enhanced productivity, onshoring and supply chain development.

基于核糖核酸(或RNA)的治疗是使用基于RNA的分子来治疗或预防疾病。最近针对新冠肺炎大流行的信使核糖核酸疫苗的成功突显了这项技术的潜力，并极大地提高了研究和工业兴趣。通过引入RNA作为疫苗或治疗剂，RNA提供了在人体内产生几乎任何功能蛋白质或肽的潜力。RNA疗法和疫苗通常需要特定的、有针对性的递送才能成为有效的药物，而一个领先的技术平台是脂质纳米颗粒，以及替代的纳米递送系统。这适用于一系列基于RNA的疗法，如mRNA/自我放大RNA、siRNA、反义寡核苷酸，以及尖端和潜在的治疗方法，如通过CRISPR/Cas9、基础或主要编辑的基因编辑技术。有机会在英国“锚定”这些新型疗法的商业规模设计和制造，并作为全球研究、开发活动和试验的中心--但考虑到这项技术的早期阶段，它需要在早到中期的研发中投资，以解决科学、制造、临床和商业挑战。需要解决的挑战包括：创造更耐热的药物；了解和控制免疫原性和不良反应；开发更好的表征方法；更高效和高生产率的过程开发和制造；以及针对一系列不同的器官和细胞类型，以使更广泛的疾病能够得到治疗。我们正在提议升级现有设施，以创造最先进的能力，以及一个雄心勃勃的大规模研究计划，重点是建立如何在产品设计、配方、制造和表征领域创造颠覆性创新的知识。这包括合理设计下一代LNP的能力，在体外方法预测药物/疫苗人类临床反应方面产生阶段性变化，最终开发数字化LNP制造过程，作为制药先进制造的灯塔项目。通过公共投资，我们将创造技术和能力，催化英国研发投资的爆炸性增长-增加我们高质量学术基础的转化，促进工业研发和临床试验，并最终通过公司增长、提高生产率、支撑和供应链发展创建一个新的生态系统。