Cell Therapies

Can CDMOs Provide Leadership in Cell and Gene Therapy?

Cell and gene therapies are poised to usher in a new era of healthcare. As of June 2021, 14 cell and gene therapies have been approved for clinical application in the United States, and over 400 more are in various stages of clinical trials. The hopes of many patients hinge on the success of these various programs, which are designed to deliver a significant advance on existing treatments or even a functional cure. As new cell and gene therapies get…

Autologous CAR T Cell Manufacturing Using a Semiautomatic, Closed, Modular Workflow

Cell-based chimeric antigen receptor (CAR) T cell therapies have rapidly advanced in recent years, with a variety of targets in clinical research and several 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, and cryopreservation are…

Evaluating Current Manufacturing Platforms for Viral Vector Production to Enable Gene Therapy Products

The rapidly growing interest in gene therapy has led to the need for more cost-effective and scalable viral vector manufacturing platforms to deliver these therapies. Adeno-associated virus (AAV) has become the vector of choice as it stands out for its safety profile since infection with the vector is not pathogenic. Also, AAV cannot replicate on its own and is not directly integrated into the host genome. Realizing the full potential of viral vector-based therapies requires a successful manufacturing platform for…

Selecting the Best Transfection Method

When to Use Transfection Reagents, Viral Transduction or Electroporation No single delivery method is ideal for all situations, but researchers may routinely employ a suboptimal approach for the sake of familiarity or to avoid any start-up costs associated with new methods. In this white paper, we will describe three methods (chemical, electroporation and viral transduction) and highlight the Mirus Bio TransIT® and VirusGEN® transfection reagents and Ingenio® EZporator® Electroporation System, which are both easy to use and cost-effective. Additionally, we…

Transfection Reagents for Cell and Gene Therapy

Gene therapy is the transfer of genetically modified materials into a patient’s tissues or cells with the goal of correcting the abnormal gene contributing to a particular disease. In recent years, biotherapeutics have emerged as effective treatments for a broad spectrum of diseases as numerous genes connected to disease and cellular processes have been identified as potential targets for therapeutic approaches. It is estimated that every year hundreds of new biotherapeutic candidates are developed. Currently, one of the most critical…

Manufacturing Pluripotent Cell Therapeutics

Here we review strategies for gaining Food and Drug Administration (FDA) approval of allogeneic, pluripotent cell therapies. The crux of the discussion is that when developing a cell therapeutic, it is critical to look as much as a decade ahead to when FDA approval will be sought to commercialize the product through a biologics license application (BLA). While this discussion focuses on FDA approval of cell therapies, it is important to acknowledge the vast number of cell therapy clinical efforts…

Scalability of Lentiviral Production with the CTS LV-MAX Lentiviral Production System in Bioreactors

The Gibco™ CTS™ LV-MAX™ Lentiviral Production System provides a scalable and high-yield lentiviral vector (LV) production platform. It is based on a high-density suspension culture of HEK293F–derived Viral Production Cells that have been optimized for viral production in chemically defined LV-MAX Production Medium. Scalable LV production of greater than 1 x 10⁸ TU/mL LV (unconcentrated) can be achieved using our proprietary lipid nanoparticle transfection reagent in combination with an LV-specific enhancer and production supplement. All components work synergistically to help…

CMC Testing Support for Gene and Cell Therapy

Eurofins BioPharma Product Testing supports the development of ATMPs both for traditional use as well as for use in personalized medicine. We provide comprehensive GMP-compliant CMC testing support to ensure the identity, potency, purity, and safety of starting materials, intermediate products, vectors, and final drug products as well as support for manufacturing process development and validation. Testing Capabilities include Raw Materials, Cell Banks, Virus Banks, Plasmid Vector, Harvest (Lot Release Testing), and Bulk & Finished Products. We support gene therapies,…

Automated PBMC Isolation and T Cell Wash and Concentration by the CTS Rotea System

Successful processing and manufacturing in cell and gene therapy workflows are essential to the efficacy of the product. Autologous cell and gene therapy workflows involve isolating cells from an individual, engineering the cells, expanding and concentrating them, and infusing them back into the patient. Certain steps in these workflows could benefit from optimized automation to decrease hands-on time and the cost of the cell manufacturing process. In this application note, we present the Gibco™ Cell Therapy Systems™ (CTS™) Rotea™ Counterflow…

Integrated Generation and Characterization of CAR T Cells

Chimeric antigen receptor (CAR) T cell therapies have been approved by the FDA and other global public health agencies to treat B cell leukemias and have seen great clinical success. Autologous CAR T cell manufacturing involves isolating T cells from a patient, activating these cells, introducing an engineered CAR construct, and expanding the cells to a scale appropriate for therapeutic dosing. Patient samples from multiple sources result in inconsistent clinical outcomes and overall product quality. To ensure patient safety and…