When I go to a therapeutic protein meeting, it’s very hard for me to hear these products referred to as “drugs.” But since they are being regulated by CDER, it’s a little easier to swallow. However, calling viral gene vectors and cellular therapies “drugs” is a bridge too far. My advice to my good friends at CBER is to keep calling their products “biologics,” or they may lose all of their regulatory responsibilities to CDER…
Tag: <span>cellular therapies</span>
The manufacturing of cell-based therapies requires harmonized processing protocols to ensure consistent quality. Expanded adipose-derived mesenchymal stromal cells (ASC) are among the most promising candidates for such therapies due to their regenerative and immunomodulatory properties. However, transitioning these therapies to large-scale production presents challenges related to cell recovery, formulation, and in-process cell counting (IPC). The Gibco CTS™ Rotea™ multipurpose Counterflow Centrifugation System enables multiple workflow operations—including cell separation, concentration, washing, and buffer exchange—and is designed to operate within a GMP environment. From a cell concentration perspective, the Rotea system can also formulate concentrated cells in different types of media, including fetal bovine serum…
The number of cellular therapies in clinical trials and on the market has continued to rise significantly in recent years—and so does the need to maintain strict control over all manufacturing steps in order to reduce batch-to-batch variability. One potential source of product variability is the manual thawing of cryopreserved cells in a water bath, which can differ between operators. Additionally, water baths pose a significant contamination risk, making them less suitable for GMP environments. To overcome these challenges, several companies have developed water-free thawing devices that offer better control of the thawing process. However, these devices either accommodate only one vial at a time or lack U.S.
FDA 21 CFR 11 compliance in producing a computer-generated audit trail. Hence, we have developed a novel, water-free and dry-heat-based, fully programmable thawing device that is capable of thawing up to ten vials simultaneously and complies with 21 CFR11 requirements…
The cell therapy industry is positioned to make major changes in healthcare and disease treatment. The Alliance for Regenerative Medicine (ARM) recently reported on the robust state of the industry and identified that revenue from cell therapy products grew from $460 million in 2010 to $1.3 billion in 2013. There are currently more than 40 commercially available cell therapy products with indications ranging from cardiovascular to cancer and non-healing wounds. The pipeline for these therapies is also expanding. ARM reports nearly 270 trials underway (Phase 1 through Phase 3). Another 58 projects are in the research stage and 245 in pre-clinical. Adding to this total, there are 77 industry-sponsored cell-based immunotherapy trials. Cell therapy represents a very different approach to treatment when compared to small molecules or many biologics. As such, regulatory authorities are evolving and adapting their approach to help ensure patient safety and efficacy of these innovative and complex therapeutics. A recent decision by regulatory authorities in Japan allows for an accelerated pathway for approval. This presents a tremendous opportunity for the industry, but at the same time, exerts tremendous pressure on developers to rapidly and efficiently characterize their products and processes in order to take advantage of such accelerated pathways. This article provides an overview of current regulations for cell-based therapies in the United States (US), European Union (EU), and Japan, and considerations for working successfully within these frameworks. It also describes a structured approach to process development that can help achieve accelerated timelines…
Cell therapy products derived from adipose tissue have some unique processing issues with regard to obtaining accurate cell counts. This is because processing methods may not only show us the nucleated stromal vascular fraction (SVF) cells but also the micellular and microvesicle particles. This is true for both veterinary and human clinical products, and poses special concerns for in-clinic processing where the cell therapy dose is correlated with cell numbers and other QC data is not especially useful. In this study, multiple cell counting methods were compared for SVF cell preparation that were derived from canine adipose tissue using commercially-available processing kits. The data clearly showed that many non-nucleated particles appear cell-like by size and shape, and can lead to counting errors with automated counters. In addition, certain reagents important to processing can have properties wherein the reagents alone (e.g., lecithin) may be counted as cells. The most accurate cell numbers were from hemocytometer-counting of cells stained with 4´,6-diamidino-2-phenylindole (DAPI) which shows the nuclei in concert with a viability stain such as trypan blue. The data clearly showed that care must be taken when counting cells used as a therapeutic dose…