Tag: <span>quality control</span>

The United States Pharmacopeia (USP) is a 184-year-old organization that has been in the forefront of technology since its inception. From publishing a manual about how to prepare therapeutic potions, USP has evolved into a compendium of standards and information on manufactured pharmaceutical products, with more than 4,000 monographs covering drug substances and biologics, and their dosage forms, excipients, and nutritional supplements. It is not surprising that the USP initiative in cell and gene therapy and tissue engineering has closely followed the emergence of these technologies…

Cell & Gene Therapy Regulatory Risk Analysis and Management

There is an increasing emphasis in clinical and translational research on the discovery and development of biomarkers that are indicative of a disease state. While biomarkers are not exclusively proteins, the emergence of new mass spectrometry platforms combined with the human genome databases has rejuvenated the search for biomarker proteins, especially in readily available body fluids such as blood. There is currently a tremendous need for an improved ability to “mine” the full depth of the proteome in a high throughput manner. To advance clinical proteomics, methodologies are needed that can accommodate higher throughput while facilitating the ability to observe large numbers of protein events…

Biologics Production Research

Parvoviruses are one of the most prevalent infectious agents in the laboratory rodent. Their effect on research can range from immune dysfunction that may mislead researchers when interpreting results to lethal effects on animals. Until recently parvovirus infection in mice was thought to be caused by minute mouse virus (MMV) and in rats by rat viral agents in the KRV or H-1 serogroups. Relatively newly discovered viruses in these groups are mouse (MPV) and rat parvoviruses (RPV-1 and 2). Parvoviruses are 15–20 nm in diameter and are single-stranded DNA viruses of about 5,000 nucleotides, which replicate through a double-stranded DNA intermediate. The protein composition consists of three structural or capsid proteins providing the viral coat (VP-1, VP-2, and VP-3) and two non-structural proteins involved in viral replication (NS-1 and NS-2). Among the capsid proteins,VP-2 is the major protein…

Biologics Production Research

The first HIV-based lentiviral vector to be used in humans, VRX496, is currently being tested in Phase I clinical trials (initiated in January 2003). With each new therapeutic comes the need to establish quality control (QC) testing designed specifically for that product. The QC testing for VRX496 was developed in accordance with the Code of Federal Regulations (CFR) 21 for pharmaceutical and bulk chemical GMPs, Points to Consider in the Characterization of Cell Lines Used to Produce Biologicals (1993) from the Center for Biologics Evaluation and Research (CBER) at FDA, and the United States Pharmacopeia (USP) 1046 for Cell and Gene Therapy Products. This report describes the QC testing of lot VRX496V02-009 of our clinical grade vector, which is being used in an ongoing clinical trial evaluating the first lentiviral gene therapy vector in humans. All assays are performed according to established standard operating procedures (SOPs) and in accordance with the principles of cGMP regulations…

Cell & Gene Therapy Viral Vectors

With the advent of whole cell-based therapeutics has come a growing standardized quality control and quality assurance of the processes employed for developing and manufacturing cellular materials, similar to the controls over traditional drugs and biologicals. Cellular therapeutics present unique process and quality control challenges due to the innate complexities of living cells, making it important to use whole cell assays to provide detailed pictures of the status and consistency of cell preparations that will be used to treat patients. This article illustrates how a cellular assay from Guava Technologies addresses these issues…

Biologics Production Cell & Gene Therapy