May 2023

May 2023: From the Editor

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While editing and proofreading an issue, we often see statements that suggest other intriguing angles to explore. But deadlines loom, and our notes to follow up on those ideas can get lost in the shuffle. And because we pursue a good portion of our manuscripts (cue image of editor chasing after conference speakers in crowded hallways), those notes have value. Seemingly casual statements in talks and manuscripts can help us focus our acquisition efforts. Working through those mental “loops,” adding…

May 23, 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

Vaccine Research and Development Infrastructure in the European Union: Establishing Support Through Integration

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Developing novel medicinal products involves many processes and requires a number of different technologies and areas of expertise. Although large pharmaceutical companies can support in-house development and fill gaps by hiring contract development and manufacturing organizations (CDMOs), the cost, complexity, and scope of development are prohibitive for most academic or small- and medium-sized enterprises (SMEs). Such difficulties are amplified for vaccine developers, especially those working to treat vulnerable populations in low- and middle-income countries where profit potential is limited. A…

May 23, 2023

Pharmaceutical Manufacturing Quality Assurance Programs: Transitioning from Research and Development to the Clinic

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On average it can take or even exceed US$1 billion to get a pharmaceutical product to market, and nine out of 10 products developed never make it to commercialization (1). As technology advances, more potential therapies and preventatives are being developed and optimized by virtual companies. They are typically small, newly formed organizations that build their momentum on programs for novel products. Because many of the program activities are outsourced, virtual start-up companies developing pharmaceutical products raise concerns about ensuring…

May 23, 2023

Digital Transformation in Biopharmaceutical Operations

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With digital innovations revolutionizing consumer-facing products such as medical devices, questions are arising about whether the biopharmaceutical and broader pharmaceutical industries are embracing digital transformation to drive process improvements and meet changing product demands. Below, Fausto Artico (global head and product director of innovation and data science at GSK) shares his insights about digitalization among pharmaceutical companies that are developing protein-based biologics, vaccines, and advanced therapies. Artico has driven several of GSK’s digitalization initiatives, including work with artificial intelligence (AI)…

May 23, 2023

Cleaning Validation Acceptance Limits for Biological Process Residues: Part 1 — Acceptable Exposure of Degraded Proteins Based on Reference Immunogens

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Over the past decade, human therapeutic proteins (HTPs) have become far more potent, and consequently, their acceptable exposures have decreased substantially. That has led to commensurately lower acceptance limits for biological process residues. Simultaneously, host-cell and other protein concentrations have increased considerably, thereby making process equipment potentially more difficult to clean. These trends have made biopharmaceutical cleaning validation more challenging. For example, the acceptance limits for many HTPs — based on acceptable exposures of active proteins — are on the…

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
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Process Validation: Calculating the Necessary Number of Process Performance Qualification Runs

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The 2011 process validation (PV) guidance document from the US Food and Drug Administration (FDA) states that the number of samples used for PV “should be adequate to provide sufficient statistical confidence of quality both within a batch and between batches. The confidence level selected can be based on risk analysis as it relates to the particular attribute under examination” (1). In alignment with those expectations, I present herein two statistical methodologies for calculating the necessary number of process performance…

May 17, 2023

Building the Next Generation of Cell-Line–Development Platforms

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Speed to market is a critical business driver in the biopharmaceutical industry. However, drug development success also requires building a robust process that maximizes efficiency while limiting the cost of goods. Achieving time and cost savings without compromising product quality is critical. Development of a productive and stable cell line is the foundation of an efficient and high-performing bioprocess. Cell-line development (CLD) represents some of the most resource-intensive steps within a process development pipeline. Bioprocess scientists must find a balance…

May 17, 2023

Advantages of Mixed-Mode Chromatography in Host-Cell Protein Removal

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Monoclonal antibodies (MAbs) are the fastest-growing modality in clinical trials and more than 100 such therapies have been approved to treat diseases. But despite the modality’s success, MAb manufacturing is often contaminated by host-cell protein (HCP) by-products. Tom Valorose, senior product manager at Astrea Bioseparations, discussed how companies can reduce HCPs dramatically during MAb purification. Valorose’s Presentation Removing HCPs is an important part of MAb downstream processing. After cell harvest, scientists perform a primary capture using affinity chromatography with protein…

May 17, 2023