Biodistribution Studies, designed to determine the distribution of cellular and gene therapy products to tissue sites other than the intended therapeutic site.
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.
Both regulatory and scientific concerns are addressed by providing supporting data on the safety of the product, the gene it transports, and the expression of the specific transgene with studies conducted in GLP mantained laboratory environment.
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 scientific staff will maintain close contact with you during the analysis, and will work with you directly to provide a detailed custom report.
AltheaDx will work closely with the research investigator, and will take into account the design and key details of the animal study, to complete the following steps:
| • | Develop and qualify a signature real-time qPCR assay. Sensitivity of 10 copies of vector in a background of 1 µg of genomic DNA is routinely achieved |
| • | Receive and assess 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 µg 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 |
DNA INTEGRATION STUDIES
AltheaDx is a leader in providing quantitative real-time PCR methods to evaluate plasmid DNA vaccine integration into host cell genomic DNA.
When a plasmid construct is selected for clinical development, the FDA requires that a study be conducted to confirm that plasmid DNA integration is either absent or at an insignificant level. A key factor in this determination is the limit of detection of the assay used. AltheaDx’s qPCR integration assay has the capability to measure down to 5 copies of plasmid DNA per µg of genomic DNA. Due to this sensitivity, and the specificity of an assay that utilizes primers and a probe designed specifically for your plasmid, qPCR is ideally suited to integration studies.
Our scientists will work closely with your research investigators to design a signature assay, customized to your specific study needs. Once AltheaDx has received tissue from the site of injection, our scientists will:
| • | Separate high molecular weight genomic DNA from low molecular weight extrachromosomal plasmid DNA |
| • | Purify high molecular weight DNA |
| • | Analyze the purified DNA with a custom real-time qPCR assay that utilizes the primers and a probe designed for your specific plasmid DNA product |
| • | Conduct study in compliance with GLP if required |
| • | Provide a comprehensive integration study report |
