Hoang Duc M, Nguyen Quyen T, Phan Trang T K, Ngo Anh T L, Pham Phuong T, Bach Trung Q, Le Phuong T T, Bui Hoa T P, Thanh Liem Nguyen
Vinmec Research Institute of Stem Cell and Gene Technology, Vinmec Healthcare System, 458 Minh Khai, Hanoi 11622, Viet Nam.
Vinmec High Tech Center, Vinmec Healthcare System, 458 Minh Khai, Hanoi 11622, Viet Nam.
Heliyon. 2023 May 3;9(5):e15946. doi: 10.1016/j.heliyon.2023.e15946. eCollection 2023 May.
Mesenchymal stem/stromal cells (MSCs) are multipotent stem cells that can be isolated from bone marrow, adipose tissue, the umbilical cord, dental pulp, etc. These cells have unique properties that give them excellent therapeutic potential, including immunoregulation, immunomodulation, and tissue regeneration functions. MSC-based products are considered advanced therapy medicinal products (ATMPs) under European regulations (1394/2007); thus, they must be manufactured under good manufacturing practices and via effective manufacturing methods. The former can be achieved via a proper laboratory design and compliance with manufacturing protocols, whereas the latter requires an approach that ensures that the quality of the products is consistent regardless of the manufacturing procedure. To meet these daunting requirements, this study proposes an exchangeable approach that combines optimized and equivalent manufacturing processes under the Quality by Design (QbD) principle, allowing investigators to convert from small laboratory-scale to large-scale manufacturing of MSC-based products for clinical applications without altering the quality and quantity of the cell-based products.
间充质干/基质细胞(MSCs)是多能干细胞,可从骨髓、脂肪组织、脐带、牙髓等中分离得到。这些细胞具有独特的特性,赋予它们优异的治疗潜力,包括免疫调节、免疫调控和组织再生功能。根据欧洲法规(1394/2007),基于MSCs的产品被视为高级治疗用医药产品(ATMPs);因此,它们必须按照良好生产规范并通过有效的生产方法进行生产。前者可通过适当的实验室设计和遵守生产规程来实现,而后者则需要一种确保无论生产过程如何,产品质量都保持一致的方法。为了满足这些艰巨的要求,本研究提出了一种可互换的方法,该方法在质量源于设计(QbD)原则下结合了优化且等效的生产工艺,使研究人员能够从基于MSCs产品的小实验室规模生产转换为大规模生产以供临床应用,而不会改变基于细胞产品的质量和数量。