Building a UK IntraCellular Drug Delivery Centre (ICDDC)

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.