Cell/Gene Therapies

eBook: Viral Vectors — Progress Toward Production Platforms

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After decades of research and development (R&D) and despite more than a few clinical setbacks, viral-vector gene therapies finally are making headway into the marketplace. However, cost considerations still reverberate around the biopharmaceutical industry. Scientists recognize that nonoptimal manufacturing is implicated in the high prices for gene-therapy products. For instance, adenoassociated virus (AAV) and other such gene-delivery systems are produced at much lower scales than are monoclonal antibodies (MAbs) and other recombinant proteins — understandably so given the size and…

August 29, 2023

How Emerging Technology Is Improving the Cell Therapy Manufacturing Landscape

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Mercedes Segura Gally, Vice President of Process Development, ElevateBio. Mercedes Segura Gally introduced the audience to ElevateBio, gave a short overview of the state-of-the-field in cell therapies, and discussed key drivers for innovation in the cell therapy space. She highlighted current manufacturing tools and upcoming technical innovations. ElevateBio is based in the Waltham, MA, area. It was founded in 2017 to develop technologies to advance cell and gene therapy products, including offering the chemistry, manufacturing, and controls (CMC) infrastructure to…

August 21, 2023

BioProcess Insider Interview: Luis Berrade, Senior Director, Biologics Testing Solutions, Charles River Laboratories

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Charles River Laboratories (CRL) tests different biopharmaceutical modalities such as antibodies and other recombinant proteins, viral vectors, cell and gene therapy (CGT) products, and plasmids. Berrade discussed the importance of biologic testing. He said such work ensures that products are safe to treat patients. Viral-safety testing is critical, for instance, because it is used to assess the quality of pharmaceutical products and test for mycoplasma and potential viruses that could contaminate a drug. In the past five years, newer modalities…

August 21, 2023

International Differences in Quality and Regulatory Requirements for Cellular Starting Material

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Cell and gene therapy (CGT) applications have increased rapidly over the past few years, with hundreds of candidate treatments entering the clinical pipeline annually. Although North America, Asia, and Europe are driving industry growth, drug sponsors in those areas will not want to limit product distribution to their respective regions; rather, they will want to maximize patient impact. Thus, companies must understand global regulatory differences and their implications for therapy production. However, advancements in the CGT industry often outpace adjustments…

August 17, 2023

Improving AAV Manufacturing Processes: Using Design of Experiments and Fit-for-Purpose Transfection Reagents

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When evaluating a new process for viral vector manufacturing, you may be wondering what the best path forward is to save time and resources while ensuring to produce enough data to support the robustness of your future manufacturing and scale-up. Polyplus® free of charge DoE service is the combination of our expertise in the field of nucleic acid delivery and in DoE methodology to support you through the evaluation of our transfection reagents. As each process is different, this service…

July 20, 2023

Measurement of Particulates in Single-Use Systems for Cell and Gene Therapies Manufacturing — Part 1: Misapplication of USP <788>

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Particulates are mobile, undissolved particles other than gas bubbles that are unintentionally present in an injectable drug product. Patient safety can be compromised by the amounts and types of particulates present in a drug product (1). They differ in nature (e.g., metal, glass, dust, fiber, rubber, polymer, mold, and degradant precipitates) and can be divided into three categories: intrinsic, inherent, and extrinsic particulates. Intrinsic (native) particles are derived from operation of a manufacturing process or its equipment, a product formulation,…

June 26, 2023

Introducing a Stable Producer Cell Line for Large-Scale AAV Manufacturing

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Although gene therapies are associated with treating rare diseases, candidate therapies for prevalent-disease indications have overtaken rare-disease treatments in clinical trials, including phase 3 studies. That indicates a change in momentum for new gene therapies that will bring innovative drug products to large patient populations. In an April 2023 webinar, Dovile Gruzdyte, global product manager for cell-line development at Cytiva, discussed how evolving needs in gene-therapy bioprocessing are driving advancements in cell-line development. Gruzdyte’s Presentation Gene therapies for prevalent indications…

June 26, 2023

eBook: Perspectives on CAR-T Cell Therapy

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Adding chimeric antigen receptors (CARs) to T cells makes them capable of recognizing and binding to target cells, with the first applications inciting patients’ immune systems to destroy malignancies. The speed with which CAR-T technology has progressed from discovery to approved therapies — and from a few academic researchers to hundreds of companies — reflects the power this modality has to revolutionize oncology. In some cases, a single treatment has led to complete (maybe even permanent) remission. And other applications…

June 16, 2023

Using Regulatory T Cells for Treatment of Type 1 Diabetes, Part 2: Drug Development and the Transition to Clinical Trials

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In the April 2023 issue of BPI, Leonardo Ferreira discussed the biology of type 1 diabetes and how he has worked toward developing a cure for that disease at his laboratory at the Medical University of South Carolina using chimeric antigen receptor (CAR) T regulatory cells (Tregs) (1). He spoke about industry-wide advances in developing Treg technology and how lessons learned during preclinical trials can be applied in human trials. He also discussed what the industry needs to develop Treg…

May 23, 2023

Viral Safety for Biotechnology Products, Including Viral Vectors: ICH Q5A Revision 2 Brings Updated and More Comprehensive Guidance

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The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) provides guidance on the testing and evaluation of viral safety of biotechnological products derived from characterized cell lines of human or animal origin through its harmonized guideline ICH Q5A (1). The latest revision, released for consultation in October 2022, maintains the key principles of previous versions while introducing key changes in response to important advances in the field. Those advances are covered in new sections that…

May 17, 2023