Author Archives: Brian Gazaille

eBook: Potency Bioassays — Development, Trending,
Transfer, and Automation

Bioassay development is a complex process that must be undertaken with great rigor and attention to detail. Potency testing experts use a range of methods including cell-based and binding assays. Consistency and reliability of results over time are paramount. Well-developed and -characterized methods are the end result of much phase-appropriate development work that goes on in parallel with bioprocess and biotherapeutic product development. This eBook begins with BPI senior technical editor Cheryl Scott’s report from the Biopharmaceutical Emerging Best Practices…

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…

eBook: Sensors — Process Analytics for Modern Biopharmaceutical Workflows

To achieve quality by design in biopharmaceutical production, manufacturers need tools that can ensure the stability of critical process parameters (CPPs) and other performance indicators related to product critical quality attributes (CQAs). Over the past couple of decades, sophisticated process analytical technologies (PATs) have emerged to address such needs. Offerings are now abounding for single-purpose sensors that measure temperature, pressure, pH, glucose, protein concentration, or dissolved oxygen. New in-line formats are enabling such instruments to provide data in real time,…

Spurring on Innovation in Gene Therapy Development

Gene therapies based on adenoassociated virus (AAV) vectors hold promise for treating myriad conditions. Immunogenicity remains a challenge for such products, however. With support from PerkinElmer, Roland W. Herzog (professor of pediatrics and Riley Children’s Foundation professor of immunology at the Indiana University School of Medicine) joined Nagendra Venkata Chemuturi (scientific director of global research for drug metabolism and pharmacokinetics, DMPK, at Takeda Pharmaceuticals) to deliver a BPI “Ask the Expert” presentation exploring strategies for minimizing immune responses to AAV-based…

eBook: CAR-T Cell Therapy — Mitigating Clinical and Bioprocess Limitations

Developers of chimeric antigen receptor (CAR) T-cell therapies are working in a state of tempered optimism. As of September 2021, the US Food and Drug Administration has approved only five such products, two coming this year. Now that those approved products have demonstrated the viability of CAR-based immunotherapies, drug developers are trying to address significant limitations that have come to light with increases in available clinical data and bioprocess knowledge. One shortcoming concerns therapeutic efficacy. Blood cancer patients who have…

eBook: Process-Related Impurities — Emerging Strategies for Detection, Identification, and Management of Host-Cell Proteins

Host-cell proteins (HCPs) represent a major class of process-related impurities (PRIs) that are generated during biopharmaceutical manufacture. Although the vast majority of such proteins are removed from a drug substance during downstream purification, residual HCPs can remain in a finished drug product. Even in minimal concentrations, copurifying HCPs can pose safety risks and compromise protein-product yield, efficacy, and stability. Thus, regulatory agencies consider the presence of HCPs to be a critical quality attribute (CQA). Sufficient clearance of these impurities helps…

Mesenchymal Stromal Stem Cells: Next Steps and Considerations for CGMP Manufacturing

Massimo Dominici is scientific founder of Rigenerand srl, a joint venture between RanD (a biomedical company producing bioreactors for liver support and chemohyperthermic technology for cancer) and experts in cell and gene therapy at the University of Modena and Emilia Region in Italy. Rigenerand develops and manufactures medicinal products for cell-therapy applications (primarily for regenerative medicine and oncology) and three-dimensional (3D) bioreactors as an alternative to animal testing for preclinical investigations. The company also produces its own pipeline of cell…

Raw Materials for Advanced Therapies: When the Process Is the Product, Ingredients Are Key

Scott Burger and Bill Janssen are both established, independent consultants specializing in gene and cell therapies. This past spring, we three discussed several aspects of raw material strategy for advanced therapies as well as the need for trained technicians in the industry. With a bachelor of science degree in biology from Tulane University (New Orleans, LA, 1983) and a medical doctorate from the University of Pennsylvania (Philadelphia, PA, 1988), Scott Burger served his residency training and fellowship at Washington University…

Toward the Point of Care: Flexibility and Decentralization Are Key to Making Autologous Therapies More Readily Available

Part of the advanced therapy medicinal products (ATMPs) class of therapeutics, cell and gene therapies (CGTs) can be either autologous, using the patient’s own cells, or allogeneic, using master banked donor cells. Global biotechnology company Orgenesis focuses on autologous therapies, with processes and systems developed for closed and automated processing that have been validated for regulatory-compliant production at the point of care for patient treatment. This technology could help overcome the limitations of traditionally cost-prohibitive CGT manufacturing methods that do…

Cell Banking in the Spotlight: Advising Biologics Developers About Cell Bank Preparation and Characterization

Living cells are at the heart of biotechnology, and cell lines for production and testing of biopharmaceuticals are highly valuable assets. The process of banking cells generally moves from development of a research cell bank (RCB) based on a clone of interest to establishment of a master cell bank (MCB), from which working cell banks (WCBs) can be produced. Especially for biotechnology startups, preparation of an MCB can involve a significant jump from work performed in standard laboratory conditions to…