Analytical

Reference Standards for Therapeutic Proteins: Current Regulatory and Scientific Best Practices

Sponsors developing and manufacturing protein therapeutic products use a variety of analytical tests (e.g., cell-based potency and chromatographic assays) to assess quality attributes of their active ingredients and drug products. Those tests are used to assess product quality in a number of activities, including characterization, comparability, lot release, and confirmation product quality and stability. Reference standards play a critical role in calibrating and confirming the suitability of such tests and in helping analysts to draw scientifically sound conclusions from data…

Activatable Immunoconjugates for Target Cancer-Cell–Specific Diagnosis and Therapy

In cancer treatment, early diagnosis and targeted therapies are assumed to yield the highest cure rates. However, most current methods are limited by their low sensitivity to early disease and a lack of specificity for targeted cell killing. Newly developed, activatable immunoconjugates assist in the accurate detection of cancer through in vivo imaging with high target-to-background contrast (1,2). They also provide for the possibility of highly specific, light-mediated treatment with minimal effects on healthy cells surrounding tumors (3). In fact,…

Analytics for Modern Bioprocess Development

Twelve years ago, about the same time the US Food and Drug Administration was putting the finishing touches on its quality by design (QbD) and process analytical technology (PAT) guidelines, I wrote an article about breakthrough pharmaceutical educational programs. That article included the perspectives from a few members in academia of the future essential skills for pharmaceutical students. At the time, bioinformatics and computerized industrial process modeling were relatively new disciplines, but their importance in future manufacturing was clear. Several…

Cell Therapy Manufacturing

Fueled by a recent resurgence in public financing and compelling clinical data for indications as diverse as acute macular degeneration and pancreatic cancer, a growing number of cell therapies are driving toward pivotal clinical studies and commercialization. Although regulatory precedents have been set for various autologous and allogeneic products in the United States, Asia, and the European Union, regulatory guidance continues to evolve for a widening array of cell products. Adult stem cells (e.g., mesenchymal stem cells), embryonic stem cells,…

North, South, East, and West

Electrophoresis is the basis of all blotting methods, and BPI Lab covered it last month (1). Electroblotting is a method for transferring electrophoretically separated proteins or nucleic acids onto a polyvinylidene fluoride (PVDF) or nitrocellulose membrane for permanence using electric current and a transfer buffer solution. This allows for analysts to further study them using probes, ligands, or stains. Capillary blotting is a variation designed to work with capillary electrophoresis. After electrophoresis the following are stacked in cathode-to-anode order: a…

Enabling Technologies

Many technological advancements in recent years have enabled companies to shorten time to market, to better understand their manufacturing processes, and to characterize their products well. In BPI’s December 2013 issue (pages 47–50), I reported on the first half of an informal reader survey about those technologies, with commentary from some survey participants and others. This month concludes with my examination of analytical, formulation/fill–finish, and facilities technologies. Analytical Technologies After writing several installments of our new “BPI Lab” series this…

Ready-to-Use Cryopreserved Primary Cells

Abiological measurement of drug activity is perhaps the most critical step in the series of tests required for product release both for clinical trials and the market. This evaluation plays an important role in the stability assessment of drug candidates. According to the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH) Q6B document, measurements of biological activity can be performed in defined animal models that demonstrate a measurable physiological change in response to…

Nucleic Acid Impurity Reduction in Viral Vaccine Manufacturing

Commercial-scale viral vaccine manufacturing requires production of large quantities of virus as an antigenic source. To deliver those quantities, a number of systems are used for viral replication based on mammalian, avian, or insect cells. To overcome the inherent limitations in production outputs with serial propagation of cells, mammalian cells can be immortalized, which increases the number of times they can divide in culture. Modifications that immortalize cells are typically accomplished through mechanisms similar to those converting normal cells to…

Analysis By Size and Charge

An early BPI Lab article addressed the power of liquid chromatographic separations for biopharmaceutical laboratory use (1). Such techniques separate biomolecules based on a number of different properties: size, solubility, hydrophobicity/-philicity, binding affinity. The next most powerful means of separation — and thus high-resolution identification — of nucleic acids and proteins/peptides is based primarily on electrostatic properties: electrophoresis. Although it doesn’t really work in a process or preparative setting, it is a fundamental technique in modern biopharmaceutical laboratories, where it…

Assay Acceptance Criteria for Multiwell-Plate–Based Biological Potency Assays

For most biopharmaceuticals, potency is assessed in a bioassay by comparing dose–response curves of the test material and a reference standard. As with all analytical techniques, such assays require criteria by which their execution can be judged objectively to be valid, regardless of whether the desired or expected result is obtained for the test sample. PRODUCT FOCUS: BIOLOGICSPROCESSFOCUS: R&D, QCWHO SHOULD READ: PRODUCT AND PROCESS DEVELOPMENT, ANALYTICAL, QCKEYWORDS: IMMUNOASSAYS, POTENCY ASSAYS, PRODUCT RELEASE, REFERENCE STANDARDS, CONTROL SAMPLES, SAMPLE ACCEPTANCELEVEL: ADVANCED…