Tag: <span>analysis</span>

The analytical characterization of recombinant protein therapeutic drug products has broadened to include the use of more sophisticated technologies. The expansion of technical abilities has translated into increasing the depth and breadth of our knowledge and understanding of the drug product intended for commercialization. With the availability of more precise methods, the regulatory expectations for understanding the characteristics of a protein therapeutic drug product are increasing. A thorough understanding of a therapeutic proteinā€™s biochemical and biophysical characteristics is necessary to support investigational new drug (IND) applications and other drug regulatory filingsā€¦

Regulatory

Manufacturers of biological products have come to accept that it makes sense, from both a business as well as a regulatory perspective, to address GMP compliance issues with bioprocessing methods as early as possible in product development. Logically, this same reasoning would also apply to the associated analytical methods used to characterize the product; however, companies still frequently leave methods optimization and validation until later in the developmental timeline which can expose them to unexpected regulatory challenges. In addition, as therapeutics increase in complexity (e.g., cell therapies, transgenics), it raises the likelihood that product characterization will be assessed by novel and increasingly intricate assaysā€”making it difficult to follow a ā€œone size fits allā€ approach to method selection, development and validationā€¦

Manufacturing Regulatory

Analytical tools for the characterization of protein identity and structure are fundamental to many fields of biochemical research. For the development of protein biopharmaceuticals, it is particularly important to measure modifications of the structure that may affect safety and efficacy. This application requires the analysis of large numbers of samples during process development. Small quantities of modifications must be detected in samples that are in the presence of more abundant native protein. Many kinds of analytical techniques are applied to this problem including peptide mapping, bioassays, liquid chromatography, spectroscopy, and so on. One of the most attractive tools is mass spectrometry, since essentially any change in the molecule is accompanied by a change in molecular weight. With the recent advent of readily accessible mass spectrometers capable of routine exact mass measurement, it is appropriate to consider the detailed requirements for this applicationā€¦

Biologics Production

One of the major aims of modern biotechnology companies that are producing recombinant therapeutic proteins is to focus on timeline reduction of critical cell line selection and process optimisation studies in order to minimise the time and financial constraints of early development products. This ā€œminimalist paradigmā€ of maximising early development throughput with minimal capital/operational outlays is a key driver for implementation of novel analytical technologies which can be applied at-line to process instrumentation. The large-scale production of recombinant therapeutics in the biopharmaceutical industry relies on in-process monitoring of product titre. Traditional titre determination methods, including enzyme-linked immunosorbent assay (ELISA) and protein A high performance liquid (immunoaffinity) chromatography (Protein A HPLC), are time consuming, and often reliant on analytical support from separate specialist teams/departments requiring detailed scientific knowledge and extensive training, with expensive capital outlay utilising large equipment…

Biologics Production

The quantitative and qualitative analysis of proteins and their amino acid sequence composition is a critical operation in many research laboratories and operations. Much protein analysis is performed using high-pressure liquid chromatography (HPLC) with ultraviolet (UV) or fluorescence detection. Although these methods are robust and widely used, certain issues limit their utility. For example, some proteins or peptides may have a poor UV response and can be difficult to detect. Direct comparisons of protein levels when quantitated directly by UV can also be problematic due to differences in extinction coefficients of various proteins…

Research

The approval of a new biological drug for therapeutic use requires supporting data from a variety of studies, including those that demonstrate the suitability of the manufacturing process. The regulatory guidance advocates that one of these studies address the issue of cell substrate stability by testing for consistent production of the product of interest by a characterised cell bank, generally the working cell bank (WCB). The study should evaluate stability during cultivation for production by examining a minimum of two time points ā€” at a minimal number of population doublings and at or beyond the limit of in vitro cell age for production. The guidelines state that, “Evaluation of the cell substrate with respect to the consistent production of the intended product of interest should be the primary subject of concern”…

Biologics Production Manufacturing

Validating the safety of biological preparations requires thorough testing for contamination by adventitious agents. Utilizing mammalian cell cultures to produce recombinant proteins as biopharmaceuticals requires testing for viral contamination. The polymerase chain reaction (PCR) may be employed to specifically detect the presence of viral DNA or RNA with great sensitivity. PCR assays are particularly useful for the qualification of recombinant cell banks. Regulatory agencies recommend that mammalian cell banks be tested for a variety of possible human viral contaminants. In most cases the cells used to produce the cell bank have been previously analyzed for viral contamination. The use of PCR for the detection of viruses in the final banked cells can alleviate the need for difficult, costly, and time-consuming infectivity assays. In some cases the relevant viruses cannot be cultured, eliminating the ability to perform infectivity assays. The PCR assay can provide a sensitive and specific method for detection of viral contamination when standard infectivity assays are unsatisfactory…

Cell & Tissue Banking

The Adenovirus Reference Material Working Group (ARMWG) oversaw development of an adenovirus reference material (ARM) with the intent to provide a way to standardize assay measurements from different laboratories. The ARM, which was manufactured in stages by various organizations including Canji (San Diego, CA) and Introgen Therapeutics (Houston, TX), is available from American Type Culture Collection (Manassas, VA). Upon completion of its manufacture, the characterization phase primarily defined viral particle concentration as well as infectious titer for this product. However, many other concurrent characterization studies were conducted including an assessment of vector purity (e.g., host cell DNA, host cell protein, reversed-phase HPLC), a short-term field use and shipping stability study, and a long-term stability study. Also included in these studies was a coordinated effort to determine the complete DNA sequence of the ARM vector genome…

Nucleic Acids Viral Reference Materials Viral Vectors

Various types of viral vectors are being employed extensively as gene therapeutics to treat cancer and genetic diseases. Among the viruses that have been produced for human clinical trials (i.e. retrovirus, adenovirus, poxvirus, adeno-associated virus, and herpesvirus vectors) adenoviruses exhibit the lowest pathogenicity yet still infect an extensive range of cell types with high efficiency. These key characteristics make recombinant adenoviruses efficient gene-delivery vehicles and excellent research tools. However, the time-consuming and complex processes of generation, amplification, purification, and quality testing associated with production of recombinant adenoviruses make it difficult for many researchers to utilize these vectors. This is particularly true with respect to cell culture optimization and the virus propagation protocols employed in vector production. In this regard, the development of innovative cell culture techniques has become vital for optimizing vector production for gene therapy…

Biologics Production Cell & Gene Therapy Viral Vectors

Foreign substances, such as disease-causing viruses and other infectious agents are recognized by the body’s immune system as invaders. Parts of the spectrum of our defenses are antibodies that bind these antigens and direct their destruction by other elements of the immune system. Antibodies have two very useful characteristics. First, they are extremely specific; that is, each antibody binds to one particular antigen. Second, the body retains the memory of encounters with antigens, enabling it to reproduce an antibody response to a later attack by that that same agent or disease. These properties make it possible to develop vaccines. It is the first trait of antibodies, their specificity, which makes monoclonal antibody (MAb) technology so valuable. Not only can antibodies be used therapeutically, to protect against disease, they also can help diagnose a wide variety of illnesses and can detect the presence of infectious agents (including biological threat agent). The use of MAbs specific for biological warfare agents in fielded assays requires maintaining a high-quality and economical supply of these reagents…

Biologics Production