Smart and flexible end-to-end cGMP mRNA manufacture on demand

RNA疗法将改变许多疾病的护理标准，从罕见病转变为慢性病，并为数百万患者实现个性化医疗。RNA的产生，纯化和递送方面的进展意味着药物可以具有成本效益并靶向新的途径。作为一种颠覆性的治疗技术，小型研究小组和生物技术公司可以快速开发新的RNA构建体。在多种RNA治疗模式中，对灵活制造的需求将增加，以加快其向患者提供的途径并提高其经济性。然而，现有的全球RNA生产利用生物制剂并不优化RNA的生化制造。灵活的制造必须适应不同的生产量，过程控制策略，加快交货时间和安全的供应链，同时提高产量，经济性和可持续性。这是一个雄心勃勃的项目，由两家开拓英国中小企业。BiologIC开发了基于IVT的mRNA小批量生产（InnovateUK10025959）和在cGMP就绪环境中的连续抗体生产（InnovateUK93825）。DeepMirror使用人工智能和专有数据集来寻找针对治疗价值优化的RNA序列。该项目的数字化方面将得到英伟达的支持。该系统将提高生产率，增加全长RNA的产量，并使用过程强化来减少足迹，提高可持续性。该系统将通过灵活的RNA制造进入400亿美元的全球市场，改善患者生活，并将英国打造成使用数字化技术制造RNA的全球领导者。

RNA therapeutics will change the standard of care for many diseases, transitioning from rare to chronic diseases, and actualize personalized medicine for millions of patients.Advances in generating, purifying and delivery of RNA mean drugs can be cost-effective and target new pathways. As a disruptive therapeutic technology, small research groups and biotechs can rapidly develop new RNA constructs.Demand for flexible manufacturing will increase across multiple RNA-therapy modalities to accelerate their route-to-patient and improve their economics. However, existing global RNA manufacturing leveraged from biologics is not optimised to the biochemical manufacturing of RNA.Flexible manufacturing must accommodate different production volumes, process control strategies, accelerated lead times and secure supply chains, while improving yields, economics and sustainability.This is an ambitious project from two pioneering UK SMEs. BiologIC has developed IVT-based small-batch manufacturing of mRNA (InnovateUK10025959) and continuous antibody manufacturing in a cGMP-ready environment (InnovateUK93825). DeepMirror uses AI and proprietary datasets to find RNA sequences optimised for therapeutic value. Digitilisation aspects of the project will be supported by Nvidia.The system will improve productivity, increase yields of full-length RNA and use process intensification to reduce footprint, improving sustainability.The system will access the $40bn global market through flexible RNA manufacturing, improve patient lives and establish the UK as a global leader in the manufacture of RNA using digitilisation technologies.