RAP-IDD - Rapid Development of Intracellular Drug Delivery Innovations

RAP-IDD - 细胞内药物输送创新的快速发展

• 批准号: 10070773
• 金额: $ 95.75万
• 依托单位: MICROPORE TECHNOLOGIES LTD
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 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=10070773
• 关键词: RAP; IDD; Rapid; Development; Intracellular

The RAP-IDD project (Rapid Development of Intracellular Drug Delivery Innovations), led by the UK SME Micropore Technologies (Micropore), supported by SME Labman Automation (Labman) and CPI, aims to develop and validate a new technology platform to encapsulate genomic material (RNA and DNA) in protective nanoparticles and integrate this with high-throughput characterisation. In a game-changing advance over current methods, the platform will be upgraded to continuous production to make it applicable to both high-throughput formulation development and continuous manufacturing - compliant with Good Manufacturing Practice (GMP). If successful, this new platform will make a step-change improvement in the efficiency with which new genomic medicines progress from discovery to real application in disease prevention and treatment.The success of mRNA-based vaccines during the COVID-19 pandemic has resulted in a large increase in interest in other nucleic acid medicines that are delivered to cells via nanoparticle delivery systems. Similar technologies are being researched to enable breakthrough vaccines for other diseases, as well as targeted treatments for cancer, rare diseases and more. However, there remain barriers to successful development and manufacture of nanodelivered intracellular drugs. The encapsulation of the nucleic acids within protective nanoparticles (NPs), such as lipid nanoparticles (LNPs), is perhaps the most critical stage in the manufacturing process. Currently there are two major encapsulation technology approaches used: In research, microfluidic mixing devices are commonly used as they can quickly produce large formulation libraries while minimizing waste. However, these mixers cannot accommodate commercial-scale production volumes. Impingement jet mixing (IJM) technology was chosen as an available means to achieve large scale commercial production during the COVID pandemic, by stacking many units in parallel. However, this approach is less controllable and is wasteful and inefficient for discovery.Micropore is pioneering an alternative and patented micromixing/encapsulation technology called Advanced Crossflow (AXF) that combines the size-control and uniformity advantages of microfluidic approaches with an ability to scale up to commercial volumes, simply by increasing instrument size and material flow. The RAP-IDD project will build on this AXF technology with the aim of achieving the 'holy grail' of intracellular drug production: A single, highly-efficient, but flexible, multi-product technology platform that can span multiple phases of the drug development and production pathway -- from lab scale to commercial scale -- without the need to redevelop and re-optimise processes at different stages. The project will undertake research to de-risk and validate this approach.

由英国中小企业微孔技术（Micropore）领导的RAP-IDD项目（快速开发细胞内药物输送创新），由中小企业Labman Automation（Labman）和CPI支持，旨在开发和验证一个新技术平台，以将基因组材料（RNA和DNA）封装在基因组材料（RNA和DNA）中，并在保护性纳米机构中与该较高的表征相结合。在当前方法上改变游戏规则的进步，该平台将升级到连续生产，以使其适用于高通量配方开发和连续制造 - 符合良好的制造实践（GMP）。如果成功的话，这个新平台将在新的基因组药物从发现到真正的预防疾病和治疗中进展到实际应用的效率的逐步变化。基于MRNA的疫苗在Covid-19大流行期间的成功导致对通过NanAmoparticle递送系统传递到细胞的其他核酸药物的兴趣大大增加。正在研究类似的技术，以实现其他疾病的突破性疫苗，以及针对癌症，稀有疾病等的靶向治疗方法。然而，仍然存在成功开发和生产纳米细胞内药物的障碍。保护性纳米颗粒（NP）（例如脂质纳米颗粒（LNP））内的核酸的封装可能是制造过程中最关键的阶段。当前有两种主要的封装技术方法：在研究中，微流体混合装置通常使用，因为它们可以快速生产大型配方库，同时最大程度地减少废物。但是，这些混合器无法容纳商业规模的生产量。通过将许多单元并联堆叠许多单元，选择了撞击射流混合（IJM）技术，作为在Covid大流行期间实现大规模商业生产的可用手段。但是，这种方法不容易控制，并且浪费且效率低下。Micropore正在开创一种称为Advanced Crossflow（AXF）的替代和专利的微混合/封装技术，该技术将尺寸控制和均匀性的优势结合在一起，可通过扩展到商业尺寸的尺寸和物质尺寸和物质流量。 RAP-IDD项目将基于此AXF技术，目的是实现细胞内药物生产的“圣杯”：一个单一的，高效但灵活的，多生产的技术平台，可以跨越药物开发和生产途径的多个阶段 - 从实验室规模到商业规模 - 无需重新开发，重新开发和重新竞争。该项目将进行研究以降低风险和验证这种方法。