Enhancing the stability of RNA Therapeutics to improve their delivery and efficacy using Deep Eutectic Solvents

使用低共熔溶剂增强 RNA 疗法的稳定性，以改善其递送和功效

• 批准号: 10070430
• 金额: $ 47.89万
• 依托单位: RNASSIST LIMITED
• 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=10070430
• 关键词: Enhancing; stability; RNA; Therapeutics; improve

The speed with which SARS-CoV-2 vaccines were formulated, approved and rolled out to the UK public has been truly remarkable and unprecedented. In particular the development of mRNA-LNPs proved not only very fast but also highly effective compared with protein based vaccines, promising a new era of therapeutic/vaccine development in 'one hundred days or less'. Despite such rapid progress and promise, the development of mRNA-LNP technology has been a compromise between speed and utility; instability of the therapeutic/vaccine has proved to be a serious drawback requiring an ultra low temperature (-80°C) cold-chain which has directly increased the cost and complexity of vaccine rollout as well as limiting vaccination programs in high income countries. Furthermore because of the extreme fragility of mRNA, the therapeutic/vaccine dose varies depending on the integrity of the cold-chain. Based on results to date we believe we can overcome many of the manufacturing bottlenecks such as storage and dispensing, as well as transport at ambient temperature by developing a universal non-toxic stabiliser that protects the mRNA therapeutic/vaccine from the point of manufacturing through to stockpiling and transport to the clinic. We are confident that the use of our molecular stabilisers will radically change the market for RNA therapeutics/vaccines by allowing the manufacture, storage, transport and delivery of consistently intact and highly active mRNA therapeutic/vaccine treatments even in environments that are lacking a functional cold-chain and distribution system.

SARS-CoV-2疫苗的配制、批准和向英国公众推出的速度确实是惊人的，前所未有的。特别地，与基于蛋白质的疫苗相比，mRNA-LNP的开发被证明不仅非常快速而且非常有效，有望在“一百天或更短”内进入治疗/疫苗开发的新时代。尽管有这样的快速进展和前景，mRNA-LNP技术的发展一直是速度和实用性之间的妥协;治疗剂/疫苗的不稳定性已被证明是需要超低温（_80 ° C）冷链的严重缺点，这直接增加了疫苗推广的成本和复杂性，并且限制了高收入国家的疫苗接种计划。此外，由于mRNA的极端脆弱性，治疗/疫苗剂量取决于冷链的完整性。基于迄今为止的结果，我们相信我们可以通过开发一种通用无毒稳定剂来克服许多制造瓶颈，例如储存和分配，以及在环境温度下的运输，该稳定剂从制造点到储存和运输到临床保护mRNA治疗剂/疫苗。我们相信，我们的分子稳定剂的使用将从根本上改变RNA治疗剂/疫苗的市场，即使在缺乏功能性冷链和分销系统的环境中，也可以生产、储存、运输和递送一致完整和高活性的mRNA治疗剂/疫苗治疗剂。