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Pharma & Biotech

Gene therapy

 

Gene therapy has the potential to substantially impact treatment of monogenic disorders, and perhaps even lead to cures, by the transfer of genetic material into the appropriate cells of the body. Gene therapy may be performed using plasmids, but is often performed using viral-mediated gene transfer with Adeno-Associated Virus (AAV) or lentivirus.

AAV vectors are often preferred due to a smaller packaging size (~5 kb) that is useful for the delivery of small genes. Plus they are non-integrating and hence have relatively low systemic toxicity and cannot propagate without a helper virus, which makes them safe for clinical use.

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AAV viral titer quantification

Accurate quantitation and characterization of AAV are critical for precise patient dosing. qPCR is a widely used method for AAV titer quantitation. However, the technique is limited by the presence of PCR inhibitors that can hinder amplification and introduce variability, plus it requires a standard curve.

Digital PCR, conversely, due to partitioning and end-point measurement, demonstrates improved amplification efficiency and higher tolerance to inhibitors than qPCR without the need for a standard curve.

We have developed a new viral vector genome quantification workflow from capsid lysis to titer determination, including singleplex and multiplex assays for quantifying and qualifying AAVs using the QIAcuity Digital PCR System.

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Access custom digital PCR assay design services

We’ve partnered with Niba Labs to develop new custom simplex or multiplex assays for the quantification of nucleic acids and to optimize existing qPCR assays for the use in digital PCR. Niba Labs will evaluate the performance of the assays and validate them within Good Manufacturing Practice (GMP).

With our combined products and services, QIAGEN and Niba Labs can help you to overcome potential resource constraints and meet demanding project timelines.

Your knowledge portal to dPCR and cell and gene therapy
Gain insights through comprehensive articles, publications, videos, webinars and posters.
Interested in a personalized package to meet your company’s needs?
We understand that one solution doesn’t fit all. We offer a range of products, technologies and services for gene therapy research. Contact us and together we can develop a package tailored for you.

Residual host-cell DNA (HCD) carryover poses major safety concerns when manufacturing protein and vaccine therapeutics. Risks include oncogenicity, infectivity and immunogenicity caused by your cell or gene therapy product. Therefore, levels of HCD must not exceed those established by regulatory agencies such as the U.S. Food and Drug Administration and the World Health Organization.

In comparison to qPCR, digital PCR provides higher sensitivity of detection at a lower template input range, enabling more robust applications. A multicopy target assay ensures that results are not affected by the fragmentation level of residual DNA (resDNA), a problem with qPCR.

Moreover, contaminating DNA in your PCR reagents can be a significant source of background and false positives. Ultra Clean Production enhances the specificity and efficiency of probe-based digital PCR to provide accurate, singleplex, or up to 5-plex analysis. Dedicated processes are implemented to enable Ultra Clean Production of the master mix, which minimizes contaminating DNA background and GC bias and gives you absolute certainty in your PCR reagents, making it an ideal choice for contamination-free, microbial applications as well as quality control applications, like residual DNA testing.

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Accelerate your data-driven gene therapies

From data analysis to databases, from gene expression to genome assembly, from interpretation to integration – QIAGEN Digital Insights has the breadth and quality of bioinformatics tools you need to drive meaningful insights and fuel gene therapy discoveries.