Cell/Gene Therapies

Navigating New Options for Commercial-Scale Biopharmaceutical Production

Scalability remains a critically important topic for biopharmaceutical companies. For conventional protein products, the strategy once was straightforward: Drug makers would scale up, beginning with cultures in flasks and roller bottles to grow enough cells to inoculate laboratory-scale (often glass) bioreactors, then again to pilot- and commercial-scale, stainless-steel, stirred-tank bioreactors. At their highest volumes, such reactors can handle tens of thousands of liters of cells and growth media. If a drug developer did not have the requisite equipment to scale…

Boosting Yield and Preserving Quality: Increasing Production of Induced Pluripotent Stem Cells for Cell Therapy and Regenerative Medicine Applications

Induced pluripotent stem cells (iPSCs) are promising starting materials for several clinical and industrial applications in cell therapy production, drug discovery, and in vitro screening. But as I learned from Maxime Feyeux (cofounder and chief science officer of TreeFrog Therapeutics in Bordeaux, France), such cells are a relatively new option in the toolbox of cell therapy developers. Advanced-therapy products can require controlled proliferation and differentiation of millions or even billions of iPSCs depending on the clinical indication, he pointed out,…

Scaling AAV Production: Easing the Transition from Laboratory Scales to Commercial Manufacturing

Adenoassociated virus (AAV) has emerged as the leading vector for gene therapy delivery. Compared with options such as lentivirus and adenovirus, AAV exhibits a strong safety profile because it has low pathogenicity and requires a helper virus to replicate. AAV is also capable of long-term gene expression, and it can infect both dividing and nondividing cells (1–5). Developers of advanced therapies have found such advantages to be quite attractive. As of January 2021, two gene therapy products have gained US…

eBook: Gene Therapies —
Developers Slowly Emerge from a Pandemic

This eBook gauges shifting expectations for the gene therapy industry amid the COVID-related uncertainties and clinical setbacks of the past couple years. BioProcess Insider founding editor Dan Stanton reports on the January 2022 Phacilitate Advanced Therapies Week event, specifically a standing presentation on the 10 most important industry drivers from the past year. Since 2017, advancements in gene therapies have featured prominently in these presentations. In 2021, gene therapies again made the list, but this time for more troubling reasons,…

Realization of Quality By Design and Beyond: The Intelligent Cell Processing System

Cells are used as raw materials, intermediates, and final products in biopharmaceutical and cell therapy manufacturing. Because living cells are always in a dynamic state, their characteristics must be kept within specified ranges throughout bioprocessing to preserve their utility. Cell population expansion without changing the original cell properties is key for obtaining the required number of cells at the next step. However, limited process data are collected during the expansion phase — information that could be used to understand and…

Get High-Throughput, Definitive Identification of Viable Cells and More in Your Cell Therapy

Analyzing for viable cells using traditional methods such as flow cytometry often encounters clogging resulting in the loss of precious cell therapy samples. Not only is it low throughput, but incredibly complicated, which can lead researchers to misidentify cellular and non-cellular material and confuse cell viability results with product-purity issues. Additionally, it is a regulatory requirement for all injectable drug products be characterized for sub visible particles (SVP) and aggregates that may form during a manufacturing process. With Backgrounded Membrane…

Addressing Critical Issues in Cell and Gene Therapy Manufacturing

Recombinant lentivirus (LV) and adeno-associated virus (AAV) are critical components of cell and gene therapies, which show great promise for treatment of diseases from genetic disorders to cancer. Accordingly, there is an unprecedented need for high titer and large-scale viral vector manufacturing processes to support the growing number of researchers developing biotherapeutics for immunotherapy. Download this Special Report, highlighting: Evolutions of gene therapy manufacturing Improvements to GMP raw-material sourcing. Role of transfection reagents like VirusGEN® GMP as a turnkey element…

Reducing Cell and Gene Therapy Development Time and Cost with New Purification Strategies

The past 40 years have ushered in the most advanced medicines the world has ever seen, with tremendous improvements in biomanufacturing technologies to enable their development. Advances in production technology have brought significant improvements in upstream productivity, which then caused bottlenecks in downstream processing. Although many bottlenecks have been resolved for most biologics, new modalities such as gene therapies and mRNA vaccines are driving the need for differentiated purification solutions. Meanwhile, pressures to increase efficiency and reduce costs continue to…

eBook: Cell and Gene Therapies — Optimization Strategies for Processing and Potency

Although relatively new to the biopharmaceutical industry, cell and gene therapy development and manufacturing are advancing rapidly. At Informa Connect’s September 2021 Cell & Gene Manufacturing & Commercialization Conference and Exhibition, held in Boston and online, presentations reviewed concerns that arise when processing complex therapies and highlighted some innovative strategies for surmounting those obstacles. Most of those approaches described during the event used data-driven solutions, with each step building on the information gained from the previous one. High-throughput technology platforms…

Optimizing the AAV Transfection Process in Suspension Cells

Given the potentially curative nature of gene and gene-modified cell therapies and the successful launches of several such products over the past five years, markets and investments are growing significantly in the sector. In the United States alone, the US Food and Drug Administration has granted approval for several genetic therapies, including Strimvelis (autologous CD34+, developed by GlaxoSmithKline and later sold to Orchard Therapeutics) in 2016 Luxturna (voretigene neparvovec, Spark Therapeutics), Yescarta (axicabtagene ciloleucel, Kite Pharma/Gilead), and Kymriah (tisagenlecleucel, Novartis)…