Gene therapy has the potential to address a myriad of otherwise untreatable genetic disorders, raising hopes for millions and contributing to vastly improved health outcomes for ensuing generations. Standardized and well-characterized viral vector reference materials are crucial for gene therapy development as they enable accurate measurement of the physical and infectious titers essential for regulatory bodies to assess safety. They also allow researchers and manufacturers to perform rigorous quality control checks.
These reference materials are so vital for benchmarking viral vector measurements throughout the development, testing, production, and regulatory approval processes that the supply of the popular Human adenovirus type 5 reference material (ATCC® VR-1516™)—first produced in 2001—actually became depleted. This forced lab managers to switch to Human adenovirus type 5 (ATCC® VR-5™)—a similar, but not equivalent reference material.
ATCC, the premier global biological materials and standards organization, has announced that the Human adenovirus type 5 reference material (ATCC® VR-1516™) is once again available. Because all new stocks of the reference material are produced and authenticated at ATCC instead of an outside working group, there are no more supply limitations on orders. Further, the new stock of ATCC® VR-1516™ comes with genome copy number, infectious titer, and bioinformatics analysis previously unperformed on working group material.
ATCC urges labs that have been using Human adenovirus type 5 (ATCC® VR-5™) in lieu of the Human adenovirus type 5 reference material (ATCC® VR-1516™) to return to the purified reference material. Although ATCC® VR-1516™ was developed from ATCC® VR-5™, the latter is not as purified, extensively characterized, or analyzed for residuals, according to ATCC. Also, the entire DNA sequence of ATCC® VR-1516™ was determined and deposited in GenBank® (AY339865).
ATCC acknowledges that because of the necessary differences in manufacturing between new lots and the original 2001 production, there are a few changes to the new reference material as compared to previous stocks. ATCC has provided an extensive comparison for reference. Both the old and new stocks have the same origin and host cells, but listed differences include particle concentration, infectious titer, and the analysis performed.
In conclusion, standardized and well-characterized viral reference materials, exemplified by ATCC® VR-1516™, play a pivotal role in advancing gene therapy development. These materials enable the normalization of laboratory-specific internal reference standards and the standardization of measurements made by different laboratories to allow for the accurate comparison of vector dose, safety, and efficacy. Taking the lead to ensure the availability of these essential reference materials underscores ATCC's commitment to providing researchers with reliable tools for groundbreaking advancements in the field of gene therapy.