Thursday, April 20, 2023 Daily Archives

ELEVECTATM — Truly Stable and Inducible Producer Cell Line for Large Scale AAV Manufacturing

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The gene therapy industry has predominantly focused on rare diseases, but recently there has been a shift to prevalent diseases covering a much broader patient population. This is largely in hopes to drive down the cost of therapy as well as minimize the financial risks to therapy developers. With the current clinical trials pipeline being dominated by therapies for larger populations, the industry must recognize that our current manufacturing methods cannot efficiently produce the amount of material needed to treat…

April 20, 2023

Introduction: Cell-Line Development Discussions at Biotech Week Boston

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The genetic sequence of the Chinese hamster ovary (CHO) cell line first was published just over a decade ago (1, 2). As with the Human Genome Project, genomic knowledge of that and other biopharmaceutical production cell lines — both animal and microbial — has expanded greatly alongside dramatic increases in computing power (3–5). Meanwhile new and improved genetic engineering techniques have offered the potential for targeted rather than random integration of transgenes into production cells. And regulators began to emphasize…

April 20, 2023

Cell Lines Are the Foundation: A Methodical Approach to Derisking Outsourced Development and Improving Manufacturability of Novel Biologics

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The most important choice for bringing a novel biologic into clinical development could be the cell line for expressing it. Somewhat determined by that, development of the initial manufacturing process comes in a close second. After an investigational new drug (IND) application, early clinical studies use materials made from the cell line and manufacturing process that were selected at the start of a clinical candidate’s life cycle. The objective for most sponsor companies is to get IND-enabling material produced in…

April 20, 2023

Targeting RNA: How Epitranscriptomics Can Improve Productivity

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Cell-line development traditionally has focused on genetic engineering of chromosomal DNA in cellular nuclei. Combining technological advances such as zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs) with ever-increasing genomic knowledge has enabled scientists to get impressive performance from microbial, plant, and animal cells. But few researchers have considered the potential for manipulating how genes are transcribed as an avenue for increasing productivity — until recently, that is. Niall Barron is a professor of biochemical engineering at Ireland’s National…

April 20, 2023

Quantitative Synthetic Biology for Biologics Production

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Biologics accounted for more new drug approvals than did small molecules for the first time in 2022, marking a significant shift in the pharmaceutical industry (1). Large-molecule pipelines are also moving from standard monoclonal antibodies (MAbs) to more complex and difficult-to-express molecules, which intensifies pressure on the industry to meet biomanufacturing demands. There is a pressing need for innovative Chinese hamster ovary (CHO)–based bioproduction systems to keep pace with this evolving landscape. While multiple areas of cell-line development (CLD) have…

April 20, 2023

April 2023: From the Editor

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The biopharmaceutical industry is continually reinventing itself. It evolved from a science-driven entrepreneurial approach and has worked over five decades to weave its network of business, regulatory, and process-development modes somewhat after the fact. The origin of so many bioprocesses in academic research laboratories prompted some of us in the editorial world to propose article series on “biobusiness 101 for scientists” back even in the early 1990s. Much of the industry’s progress toward its current form depended on adapting processes…

April 20, 2023

Using Regulatory T Cells for Treatment of Type 1 Diabetes, Part 1

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During the January 2023 Advanced Therapies Week in Miami, FL, Leonardo Ferreira, an assistant professor from the Medical University of South Carolina, spoke about his team’s work in developing chimeric antigen receptor (CAR) regulatory T cells (Tregs) as living therapeutics for type 1 diabetes. Ferreira has focused on type 1 diabetes since 2016, when he undertook postdoctoral work at the University of California at San Francisco under Qizhi Tang and Jeff Bluestone before continuing his research at his own laboratory…

April 20, 2023

Leveraging Material-Binding Recombinant Proteins: A Novel Approach to Tissue Regeneration

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In March 2023, I took the opportunity to speak with Luis Alvarez about the founding of Theradaptive. The company specializes in engineering recombinant proteins, with an initial focus on developing therapeutics for regeneration of soft, vascular, and bone tissue. “Theradaptive grew out of my thinking about combat injuries,” Alvarez told me. Before earning a doctoral degree in biomedical engineering from the Massachusetts Institute of Technology and working as cofounding deputy director of the US Department of Defense’s regenerative medicine program,…

April 20, 2023

Overcoming the Digital Divide: Leveraging Intelligent Automation and Informatics Expertise

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Shifting to a digital regulatory environment is forcing pharmaceutical companies to confront knowledge gaps across key research and development (R&D) functions. As health authorities streamline information exchange through data standardization, the separation between regulatory operations and other functions within the pharmaceutical industry begins to blur. By advancing implementation of ISO identification of medicinal product (IDMP) standards, companies are improving interoperability and information sharing among key clinical, pharmacovigilance, quality, manufacturing, and supply-chain–logistics teams. However, a company’s important regulatory and strategic planning…

April 20, 2023

Biopharma 4.0 — the Talent Evolution

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Biopharma 4.0 refers to applications of data and digital technologies to biotherapeutic manufacturing. Technological advances now enable the internet and its embedded systems to serve as a nucleus through which biomanufacturers can integrate production lines and processes across organizational boundaries, thereby forming a networked and agile value chain. Solutions under the industry 4.0 umbrella include • platforms for “smart” manufacturing made possible by the internet of things (IoT) • artificial intelligence (AI) • systems for process automation • technologies for…

April 20, 2023