Building a UK IntraCellular Drug Delivery Centre (ICDDC)

核糖核酸（RNA）为基础的治疗是利用RNA为基础的分子来治疗或预防疾病。信使RNA疫苗最近在应对COVID-19大流行方面取得的成功凸显了该技术的潜力，并大大增加了研究和工业兴趣。RNA通过引入mRNA作为疫苗或治疗剂，提供了在人体内产生几乎任何功能性蛋白质或肽的潜力。RNA疗法和疫苗通常需要特定的靶向递送才能成为有效的药物，而脂质纳米颗粒是一个领先的技术平台，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/自我扩增RNA、siRNA、反义寡核苷酸，以及通过CRISPR/Cas9、碱基或引物编辑的基因编辑技术等尖端和潜在的治疗方法。英国有机会“锚定”这些新型疗法的商业规模设计和制造，并成为研究、开发活动和试验的全球中心——但鉴于这项技术处于早期阶段，它需要在早期到中期的研发方面进行投资，以解决科学、制造、临床和商业方面的挑战。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工艺开发和制造；并针对一系列不同的器官和细胞类型来治疗更广泛的疾病。我们建议升级现有设施，以创造最先进的能力，以及一个雄心勃勃的、大规模的研究计划，专注于建立在产品设计、配方、制造和表征领域创造颠覆性创新的专业知识。这包括合理设计下一代LNP的能力，在体外方法领域创造一个台阶，以预测药物/疫苗的人体临床反应，并最终开发一个数字化的LNP制造工艺，这将成为制药行业先进制造的灯塔项目。通过公共投资，我们将创造专业知识和能力，促进英国研发投资的爆炸式增长——增加我们高质量学术基础的翻译，促进工业研发，临床试验，并最终通过公司增长，提高生产力，在岸和供应链发展创造一个新的生态系统。

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.