Sana Biotechnology, Inc., South San Francisco, California, USA.
Biopreserv Biobank. 2021 Aug;19(4):332-341. doi: 10.1089/bio.2020.0130. Epub 2021 Jan 25.
Cell-based therapeutics promise to transform the treatment of a wide range of diseases, many of which, up to this point, are incurable. During the past decade, an increasing number of cell therapies have been approved by government regulatory agencies in the United States, Europe, and Japan. Thousands of clinical trials based on live cell therapies are now taking place around the world. But most of these live cell therapies face temporal and/or spatial distances between manufacture and administration, posing a risk of degradation in potency. Cryopreservation has become the predominant biobanking approach to maintain the product's safety and efficacy during transportation and storage. However, the necessity of cryogenic shipment and storage could limit patient access to these emerging therapies and increase the costs of logistics. In the (bio)pharmaceutical industries, freeze-drying and desiccation are established preservation procedures for manufacturing small molecule drugs, liposomes, and monoclonal antibodies. Over the past two decades, there has been a growing body of research exploring the freeze-drying or drying of mammalian cells, with varying degrees of success. This article provides an overview of the technologies that were adopted or developed in these pioneering studies, paving the road toward the preservation of cell-based therapeutics in a dry state for biomanufacturing.
基于细胞的疗法有望改变多种疾病的治疗方法，其中许多疾病迄今为止都是无法治愈的。在过去的十年中，越来越多的细胞疗法已获得美国、欧洲和日本政府监管机构的批准。目前，全球范围内正在进行数千项基于活细胞疗法的临床试验。但是，这些活细胞疗法中的大多数都面临着从制造到给药之间的时间和/或空间距离，从而存在效力降低的风险。冷冻保存已成为生物库维持产品在运输和储存过程中安全性和有效性的主要方法。然而，低温运输和储存的必要性可能会限制患者获得这些新兴疗法的机会，并增加物流成本。在（生物）制药行业中，冷冻干燥和干燥是制造小分子药物、脂质体和单克隆抗体的既定保存程序。在过去的二十年中，越来越多的研究探索了哺乳动物细胞的冷冻干燥或干燥，取得了不同程度的成功。本文概述了这些开创性研究中采用或开发的技术，为细胞疗法的干燥状态下的生物制造保存铺平了道路。
