Biodistribution Studies

AltheaDx offers comprehensive Biodistribution Studies designed to determine the distribution of cellular and gene therapy products to tissue sites other than the intended therapeutic site. Our custom designed real-time qPCR analysis provides a powerful tool for assessing the safety and efficacy of nucleic acid therapeutics. It addresses both regulatory and scientific concerns by providing information on the safety of the product, the gene it transports, and the expression of the specific transgene.

Working closely with the research investigator who designs and completes the animal studies, AltheaDx will complete the following steps:

• Develop and qualify a signature real-time qPCR assay. Sensitivity of 10 copies of vector in a background of 1µg of genomic DNA is routinely achieved.
• Receive and access each tissue sample.
• Extract nucleic acid from each specimen and prepare appropriate aliquots.
• Perform real-time quantitative PCR using ABI Prism®* 7700 or 7900 Sequence Detection System. Each sample analysis is performed with up to 1 mg of genomic DNA per well, 3 wells per sample (2 unspiked, 1 spiked with a known quantity of the target sequence).
• Perform expression analysis on positive samples using RT-qPCR assays designed to detect low transcript numbers of transgene in a high total RNA background.
• Generate custom, comprehensive reports for both assay development/qualification and biodistribution results.
• Conduct study in compliance with GLP if required.

AltheaDx scientists will apply their extensive expertise in qPCR assay development to develop the most robust, specific and sensitive assay for your product. Additionally, AltheaDx's Study Director will maintain close contact with you during the analysis, and will work with you directly to provide a detailed custom report.