Cell/Gene Therapies

Novel Technologies for Advancing Allogeneic Cell Therapies

In many ways, cells are the ultimate therapeutic product. They can integrate and participate in different biologic processes and replace missing biological functions. Cell therapies are dynamic, versatile, and with the appropriate engineering, capable of influencing and correcting disease processes robustly. Cell therapies essentially are living medicines, and their adaptability contrasts with conventional drug modalities that generally have only a single specific target or effect. Because cell therapies are highly complex modalities, their scientific and R&D challenges are different from…

Process Intelligence: Gene Therapy Case Study Shows That the Journey to Improved Capabilities Starts with One Step

The product development team at a gene therapy contract development and manufacturing organization (CDMO) was working on a high-priority drug-substance project for a key client. The material was crucial to that client’s early stage clinical trial, with an immediate value over US$500,000 to both the client and the CDMO. Unfortunately, the bioreactor used in the upstream process — a transfection unit operation for an adenoassociated virus (AAV) vector — had developed an intermittent problem that could force it to shut…

Partnerships Are Crucial to Getting Advanced Treatments to Market

New treatments and approaches for tackling serious diseases are being developed using cell and gene therapy (CGT). CGTs are adding a new dimension to the way patients with such conditions can be treated in modern healthcare. But these advanced treatments require complex research, development, and manufacturing processes. CGTs are related and to a degree interchangeable. However, gene therapies typically use some kind of genetic delivery system, (e.g., virus-based vectors) to treat a disease by replacing a malfunctioning gene or introducing…

Advancing Logistics for ATMP Manufacturing

Advanced therapy medicinal products (ATMPs) hold much potential for improving healthcare. They offer hope for treating or even curing patients. The biopharmaceutical industry has recognized the importance of making such therapies accessible to as many people as possible. To provide personalized ATMPs, biomanufacturers are shifting toward flexible, patient-centered production processes. A Paradigm Shift in ATMP Manufacturing Ex vivo cell and gene therapies are particularly promising approaches to personalized regenerative medicine. Thus, it is no surprise that the numbers of US…

Advanced-Therapy Automation Capabilities Can Increase Efficiencies and Reduce Costs

The cell and gene therapy (CGT) industry has grown out of disparate groups of developers, suppliers, and service providers. Players in the sector must collaborate if we expect to commercialize new therapies at scale. Despite concerted efforts in the field, however, the critical question of how to automate CGT production has yet to be answered. Without automated processes in place, the sector risks spiraling costs and operational inefficiencies that could inhibit patient access to much-needed therapies. Understanding such concerns, several…

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…