Cell Therapies

Planning for Commercial Scale of Cell Therapy and Regenerative Medicine Products, Part 1: Achieving Manufacturability and Managing Cost of Goods

Much mystique and mystery surround the emerging industries of cell therapy and regenerative medicine. As companies progress toward commercial manufacture with potential game changers (e.g., cures for cancer and diabetes) the industry could be on the verge of significant breakthroughs. However, with no real successes to date, the question is raised: What core attributes are required to achieve commercial success? The tale of Dendreon’s struggles highlights how difficult it can be to commercialize even an approved cell therapy product. Since…

Foundation Elements for Cell Therapy Smart Scaling

Cell therapy is the injection of cellular material into patients. The injected cell-therapy product (CTP) usually consists of intact living cells. In recent years, cell therapies have evolved and matured, moving from academia to industry. That maturation is reflected in the number of open clinical trials that include the term cell therapy in their descriptions: To date, there are more than 8,700 open trials listed on the US National Institutes of Health’s online database (clinicaltrials.gov), most of which are in…

Extracellular Vesicles Commercial Potential As Byproducts of Cell Manufacturing for Research and Therapeutic Use

Extracellular vesicles (EVs) are emerging as a potential alternative to some stem-cell–derived therapeutics (1, 2). Sometimes called exosomes, they are small, secreted vesicles that can possess similar therapeutic mechanisms to whole cells, possibly representing the active pharmaceutical ingredient. In the past 15 years, academic and industry interest in EVs has exponentially increased as mounting evidence demonstrates their role in physiology and pathology as well as their therapeutic potential. In light of growing efforts in using EVs for research and therapy,…

Maximizing PMBC Recovery and Viability: A Method to Optimize and Streamline Peripheral Blood Mononuclear Cell Isolation, Cryopreservation, and Thawing

The quality of peripheral blood mononuclear cells (PBMCs) isolated from whole blood has a significant impact on their subsequent analysis. Maximizing recovery, viability, and functionality of isolated PBMCs is essential to the reliability and consistency of downstream applications, particularly within cell therapy manufacturing. The standard method for purification of PBMCs is density-gradient centrifugation. It requires precise layering of whole blood over a density medium (e.g., Ficoll polysaccharide reagent from GE Healthcare), with careful pipetting of the floating cell layer after…

The Potential Application of Real‑Time Release Testing for the Biomanufacture of Autologous Cell‑Based Immunotherapies

Cell-based immunotherapies (iTx) are emerging as a truly transformative therapeutic modality that is both complementary and convergent with existing regenerative medicine approaches, including gene therapy, cell therapy, and tissue engineering (Figure 1). Critically, iTx offer step-change improvements in efficacy compared with current standards of care (1) for a range of clinical indications and unmet therapeutic needs — particularly oncology. The clear efficacy of iTx is in contrast with some previous regenerative medicine approaches, including early mesenchymal stem cell (MSC) therapies…

Are You Ready for a Tech Transfer? Part 1: Challenges and Critical Factors for Success in Cell Therapy Development

Cell therapies offer enormous promise for treatment of a range of conditions by replacing damaged tissue or leveraging the body’s own resources to heal itself. Not surprisingly, the cell therapy industry is growing rapidly and is poised to have a major impact on healthcare and disease treatment. The Alliance for Regenerative Medicine (ARM) has reported on the robust state of the industry, noting that revenue from cell-derived products grew from US$460 million in 2010 to $1.3 billion in 2013 (1).…

Perfusion’s Role in Maintenance of High-Density T-Cell Cultures

T-cell therapy is a rapidly growing field of personalized medicine, attracting the interest of venture capitalists and pharmaceutical companies alike. Such therapies exploit T cells’ innate abilities to protect against pathogens as well as to seek and destroy cancerous cells. Although many different forms of T-cell therapies are currently in clinical trials, they all follow a common protocol: T cells are isolated from a patient, modified and expanded in a laboratory setting, and then infused back into the same patient…

Bioreactor Design for Adherent Cell Culture — The Bolt-On Bioreactor Project, Part 1: Volumetric Productivity

The Bolt-on Bioreactor (BoB) project is an independent initiative aimed at developing and commercializing a bioreactor for efficient, automated culture of adherent cells in production of therapeutic cells and other biopharmaceuticals (1). After conducting thorough research on available culture systems for adherent cells, the BoB team believes that a successful alternative to existing devices must solve four major challenges. The first challenge has to do with volumetric productivity, the second with process automation, the third with containment and sterility, and…

BPI Theater at the 2014 BIO Convention

When we launched the BioProcess Theater series at the Biotechnology Industry Organization’s International Convention in 2007, we hoped that our special programming would fill a need within that event’s exhibit hall. We wanted to bring into the hall the type of technical presentations that are not generally part of the main event’s more executive-level, business-focused programming.It has therefore been especially gratifying to see our attendance growing every year — such that standing-room-only is becoming more the rule than the exception.…

Expansion of Human Mesenchymal Stem Cells: Using Microcarriers and Human Platelet Lysate

Cell therapy holds the promise of delivering the next generation of future medical breakthroughs. In this respect, multipotent progenitor cells such as human mesenchymal stem cells (hMSCs) have attracted high clinical interest because of their ability to differentiate into various cell types and their immunoregulatory properties. Furthermore, hMSCs express only low levels of class I major histocompatibility complex (MHC I) molecules on their surfaces and are therefore invisible to a host’s immune system. Finally, hMSCs can actively suppress the innate…