Cell/Gene Therapies

eBook: Cell and Gene Therapies —
A 2021 Industry Update

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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…

August 25, 2021

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

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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…

June 18, 2021

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

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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…

June 18, 2021

Four Design Factors Shaping Multimodal Cell and Gene Manufacturing

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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…

June 18, 2021

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

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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…

June 18, 2021

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

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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…

June 18, 2021

Analytical Methods for Cell Therapies: Method Development and Validation Challenges

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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…

June 18, 2021

A Complete Solution for MSC Therapy Workflows: Cell Scale-Up, Cryopreservation, and DMSO Removal

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Mesenchymal stem cells (MSCs) are used frequently for cell therapy applications. As multipotent cells, they can differentiate into other lineages such as adipocytes, osteocytes, and chondrocytes. Additionally, they are known to secrete trophic factors that can play important roles in immunoregulation. Although MSCs can be isolated from several different tissue sources, those derived from bone marrow commonly are studied because they are easy to access in quantities large enough for therapeutic dosing (2 × 106 cells/kg of body weight). Still,…

June 18, 2021

Overcoming Obstacles in AAV Viral Vector Manufacturing

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Rapidly growing interest in gene therapy has led to the need for more cost-effective and scalable viral-vector manufacturing platforms. Adenoassociated virus (AAV) has become a vector of choice because of its safety profile (nonpathogenic infection). In addition, AAV cannot replicate on its own and is not integrated directly into the host genome. AAV vector manufacturing using human embryonic kidney (HEK) cells in either adherent or suspension mode includes several typical processing steps: cell expansion, plasmid transfection, viral-vector production, cell lysis,…

June 18, 2021

Facilities for Novel Therapies: Demystifying Design and Engineering Requirements for Cell and Gene Therapy Production

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Many veterans of the biologics industry presume that emerging therapeutics such as cell and gene therapies (CGTs) require production facilities that differ substantially from those for monoclonal antibodies (MAbs) and other conventional biologics. But experience with designing CGT facilities bears out that far more synergies than differences exist across facilities for conventional and advanced therapies. Herein, I call attention to some of those shared design concerns and demystify facilities and engineering requirements for CGTs. Observing the Synergies Many processes have…

May 21, 2021