Cell/Gene Therapies

Corning X-WASH System: DMSO Reduction of Cryopreserved Human Mesenchymal Stem Cells

Cryopreservation is a necessary part of workflows for both autologous and allogeneic therapies. The ability to cryopreserve cells for cell therapy increases the range of administration, shelf life, and time for safety testing. Cryoprotectants such as dimethyl sulphoxide (DMSO) often are added to freezing media to increase post-thaw cell survival. However, DMSO itself can be cytotoxic, so reducing its final concentration can be necessary. Below, I demonstrate how the Corning X-WASH system can reduce the amount of DMSO used in…

Accelerating the Development of Cell and Gene Therapies: From Discovery to Biomanufacture

Oxgene, a WuXi Advanced Therapies (WuXi ATU) company, provides cell and gene therapy pioneers around the world with technologies, automation platforms, and service solutions to advance their preclinical research and accelerate their timelines to good manufacturing practice (GMP) manufacturing. WuXi ATU, a global contract testing, development, and manufacturing organization (CTDMO), offers world-class, integrated GMP manufacturing and testing platforms to reduce time to market, while maintaining high titers, high levels of quality assurance, and full regulatory compliance. Together, our complete end-to-end…

Autologous CAR T Cell Manufacturing Using a Semiautomatic, Closed, Modular Workflow: Seamless Transition from Discovery to Clinical Manufacturing

Chimeric antigen receptor (CAR) T cell therapies have advanced rapidly in recent years, with a number of targets in clinical research and several US Food and Drug Administration (FDA)-approved products already on the market. There has been tremendous effort to make CAR T cells more effective, safe, and persistent when treating patients. On the manufacturing side, however, errors, lot-to-lot variation, and contamination can be associated with open processes and manual handling of CAR T cells. Cell isolation, gene editing, expansion,…

eBook: Cell and Gene Therapies —
A 2021 Industry Update

The US Food and Drug Administration (FDA) reports that as of June 2021, 22 advanced therapy products have received regulatory approval in the United States. The first such product gained regulatory approval in 2010. Since then, hundreds of cell and gene therapies have advanced to clinical evaluation, but few products have reached commercial stages — and those that have done so have been hindered by manufacturing problems. In this eBook, writers from the BioProcess Insider and Project Farma analyze trends…

Mesenchymal Stromal Stem Cells: Next Steps and Considerations for CGMP Manufacturing

Massimo Dominici is scientific founder of Rigenerand srl, a joint venture between RanD (a biomedical company producing bioreactors for liver support and chemohyperthermic technology for cancer) and experts in cell and gene therapy at the University of Modena and Emilia Region in Italy. Rigenerand develops and manufactures medicinal products for cell-therapy applications (primarily for regenerative medicine and oncology) and three-dimensional (3D) bioreactors as an alternative to animal testing for preclinical investigations. The company also produces its own pipeline of cell…

Raw Materials for Advanced Therapies: When the Process Is the Product, Ingredients Are Key

Scott Burger and Bill Janssen are both established, independent consultants specializing in gene and cell therapies. This past spring, we three discussed several aspects of raw material strategy for advanced therapies as well as the need for trained technicians in the industry. With a bachelor of science degree in biology from Tulane University (New Orleans, LA, 1983) and a medical doctorate from the University of Pennsylvania (Philadelphia, PA, 1988), Scott Burger served his residency training and fellowship at Washington University…

Four Design Factors Shaping Multimodal Cell and Gene Manufacturing

Cell and gene therapy manufacturing is about to hit a breaking point. The tension lies between increasingly diverse research pipelines and a tradition of dedicated facilities built for single-product, large-scale manufacturing. That incompatibility is widening as more cell and gene therapy products progress toward commercial production, forcing manufacturers to make a choice: either invest in major facility modifications and complex technology transfers to keep up or break from tradition and explore the potential of multimodal manufacturing. More than half of…

Toward the Point of Care: Flexibility and Decentralization Are Key to Making Autologous Therapies More Readily Available

Part of the advanced therapy medicinal products (ATMPs) class of therapeutics, cell and gene therapies (CGTs) can be either autologous, using the patient’s own cells, or allogeneic, using master banked donor cells. Global biotechnology company Orgenesis focuses on autologous therapies, with processes and systems developed for closed and automated processing that have been validated for regulatory-compliant production at the point of care for patient treatment. This technology could help overcome the limitations of traditionally cost-prohibitive CGT manufacturing methods that do…

Measuring Viral Titer in AAV-Mediated Gene Therapy Using a PCR Technique for Absolute Quantitation

Gene therapies have reemerged as promising treatments to a number of genetic illnesses. Nearly 400 gene therapy clinical trials are recruiting or ongoing in the United States for diseases such as hemophilia and spinal muscular atrophy (1). The primary vehicles used today to deliver such therapies to patients’ cells are viral vectors such as adenoassociated viruses (AAVs), but producing biologically active vectors for gene therapy can be problematic. One difficulty is generating vectors at the correct concentration to yield a…

Analytical Methods for Cell Therapies: Method Development and Validation Challenges

Advanced-therapy medicinal product (ATMP) characterization and analysis play important roles in providing chemistry, manufacturing, and controls (CMC) information for regulatory applications as well as in supporting product-release and stability studies. Each type of advanced therapy presents different analytical development challenges, so each requires specific characterization, potency, purity and identity assays. Variability in cells and among patients, multiple and complex mechanisms of action (MoAs), a general lack of readily available reference materials, and complicated analytical methods and instruments underlie the major…