Freeze-dryer for lyophilization of polyplexes and proteins

在基因和治疗性蛋白质递送、聚合物科学和再生医学的研究项目过程中，一组研究人员迫切需要获得一种仪器，以帮助设计用于基因和蛋白质递送的治疗性制剂的长期储存方法。用于将溶液转化为固体以使其稳定用于储存和分配的最常见过程之一称为冷冻干燥。该工艺包括三个主要步骤（冷冻、一次干燥和二次干燥），需要仔细优化，以保持制剂的物理化学性质，同时将其残留湿度降至3%以下，以实现产品的良好长期稳定性。该冻干机应用程序是对有助于优化长期存储方法的系统进行协调搜索的结果。有了这种仪器，就可以长期储存治疗制剂，供申请人的研究小组进行体外和体内研究，还可以将这些制剂分发给世界各地的合作者。它还将为扩大制剂制备方法铺平道路，该方法必须与长期储存相结合。迫切需要这一系统，以促进技术开发和向工业转移，最终用于医疗用途。

In the course of their research projects in gene and therapeutic protein delivery, polymer science and regenerative medecine, a group of researchers urgently require access to an instrument that will help design long-term storage methods of therapeutic formulations for gene and protein delivery. One of the most common processes used to convert solutions into solids in order to stabilize them for storage and distribution is called freeze-drying. The process consists of three main steps (freezing, primary drying, and secondary drying) that need careful optimization in order to preserve the physico-chemical properties of the formulations while reducing their residual humidity below 3% to achieve a good long-term stability of the products. This application for a freeze-dryer is the outcome of a concerted search for a system that will help optimizing long-term storage methods. Access to such an instrument will permit long-term storage of therapeutic formulations for in vitro and in vivo studies in the applicants research groups and will also permit distribution of these formulations to collaborators throughout the world. It will also pave the way to a scale-up of the formulations preparation method which must be combined with long-term storage. This system is urgently required to facilitate technology development and transfer to industry for eventual medical applications.