Building a UK IntraCellular Drug Delivery Centre (ICDDC)

基于核糖核酸（或RNA）的疗法是使用基于RNA的分子治疗或预防疾病。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这项技术的潜力，并极大地提高了研究和工业兴趣。通过将mRNA作为疫苗或治疗剂引入人体，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治疗和疫苗通常需要特异性的靶向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，沿着替代纳米递送系统。这适用于一系列基于RNA的疗法，如mRNA/自扩增RNA、siRNA、反义寡核苷酸，以及尖端和潜在的治愈性治疗方法，如基因编辑技术，通过CRISPR/Cas9、碱基或引物编辑。有机会在英国“锚”商业规模设计和制造这些新类别的疗法，并作为全球研究中心，开发活动和试验-但鉴于该技术处于早期阶段，它需要投资于早期到中期的研发，以解决科学，制造，临床和商业挑战。需要应对的挑战包括：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;更有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，使治疗更广泛的疾病。我们建议升级现有的设施，以创造最先进的能力，沿着一个雄心勃勃的，大规模的研究计划侧重于建立专门知识，以在产品设计、配方、制造和表征领域进行颠覆性创新。这包括合理设计下一代LNP的能力，在预测药物/疫苗人类临床反应的体外方法领域创造一个台阶式变化，最后，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，我们将创造技术和能力，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转化，促进工业研发，临床试验，并最终通过公司增长，提高生产力，外包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.