Patient Monitoring & Biodistribution Studies
Cellular and gene therapy technology has become an important strategy for the development of products for currently untreatable diseases. Biodistribution studies are essential to address regulatory concerns and provide an important part of the safety evaluation of these novel gene therapy technologies. qPCR is required to be performed on different tissues to determine the distribution of cellular and gene therapy products. Studies are designed to investigate integration or expression of the vector construct in the germ line, the persistence of the vector in the target tissue and dissemination to non-target tissues.

In addition, clinical material from patients exposed to gene therapy transfer medicinal products should be monitored with assays to establish if there is any long term persistency of the viral vector, or reactivation. Real-time qPCR assays are recognised as the most sensitive method for this purpose.

Vitrology’s tissue and clinical material qPCR assay service follows the FDA criteria:

Use a minimum of three samples per tissue. One sample of each tissue should include a spike of control DNA, including a known amount of the vector sequences, in order to assess the adequacy of the qPCR assay reaction. The spike control will determine the specified qPCR assay sensitivity.

Provides a rationale for the number of replicates for testing per tissue, taking into account the size of the sample relative to the tissue you are testing.

Click here for literature references.