Gene therapy products while a relatively new category with limited approval history nevertheless still requires rigorous characterization and quality control. Potency estimation for gene therapy, especially those using adeno-associated virus (AAV) as a vector to deliver the product is a measure of the therapeutic activity of the drug that also involves measure infection of target cells (DNA, copy number), transcription (RNA measurement), translation (protein expression), protein modifications, proper localization of the protein product (protein function at the targeted location).
The Food and Drug Administration (FDA) describes potency for a gene therapy product as “the specific ability or capacity of the product, as indicated by appropriate laboratory tests or by adequately controlled clinical data obtained through the administration of the product in the manner intended, to effect a given result.” (21 CFR 600.3(s)). Applied StemCell (ASC) offers a preclinical Bioservice platform that support AAV gene therapy and cell therapy development to meet the fast-growing needs in gene and cell therapy space. ASC has extensive experience in AAV gene therapy products using AAV2, AAV5, AAV, 6, AAV8, AAV100M and other serotypes: titer estimation, infectivity, and potency; serotype assessment for cell line and animal models.
ASC will develop assays to determine AAV potency at the molecular level using droplet digital PCR (ddPCR) and qPCR in in vitro and in vivo models to detect viral copy number per microgram of genomic DNA at specified time points; and, set specifications for acceptable range of DNA in conjunction with test vector dose. We will work with you every step of the way to develop the right assay for your gene therapy projects.