Tag: <span>standards</span>

The Adenovirus Reference Material (ARM) is a purified and well-characterized wild type adenovirus (Ad5) now available to researchers worldwide. Due to the need for a common reference material, the ARM was produced with the purpose of validating assay methods and internal standards for use in developing recombinant adenovirus for gene therapy. Analysis of ARM by RP-HPLC, however, detected the presence of a contaminant peak with a distinctive A240 local wavelength maximum. The contaminant was found in all of the vials, with some variability in amount between vials. It appears that the contaminant is not associated with the virus and it is unlikely that it will interfere with the use of the ARM as a reference material. The source of the contaminant was probably a leachate or plasticizer from the tubing or containers used during the final processing step…

Viral Reference Materials

Recombinant adeno-associated viral (rAAV) vectors are known to be efficient vehicles for gene transfer in animal models. The attractive feature of this vector system consists primarily of long-term gene expression with little or no associated toxicities following administration to a variety of tissues. Previous and ongoing clinical trials in humans demonstrate a very good overall safety profile, but problems persist due to the lack of any systematic method for normalizing doses administered to animals and humans. To date, most of the work involves AAV serotype 2 vectors, but vector systems based on other AAV serotypes continue to develop rapidly. Administered doses are usually based on titer, but the defective nature of AAV makes determining vector infectious units difficult. Titering methods based on vector genomes (using hybridization, real-time PCR, or spectrophotometry) are more reliable, but give no information as to the infectivity of the vector. Determining infectious titer is critical, as the ratio of infectious virions to vector genome-containing virions helps to determine the dose, potency, and strength of the vector preparation…

Viral Reference Materials Viral Vectors

As you stand on the brink of finalizing your first Investigational New Drug (IND) application for a cellular therapy product, there is always the question looming in the back of your mind. “What did we forget?” Hopefully, the answer is “Nothing.” However, it is always good to undergo a review of the standard systems needed in order to transition from research to clinical manufacturing. This article describes an overview of the basic regulatory guidelines and quality systems necessary to begin clinical trials under the regulations of the Food and Drug Administration. However, this should only be considered a guideline, as it does not necessarily address the standards of other regulatory agencies. The investigational product, the clinical indication, and the manufacturing materials used in the investigational product can also change the regulatory requirements needed to proceed with the initiation of clinical trials…

Cell & Gene Therapy Manufacturing Regulatory