The Role of ddPCR in Clinical Trials: Precision Quantitation for Gene Therapy
- No Comments
In the rapidly evolving landscape of cell and gene therapy, the margin for error is non-existent. As therapies move from preclinical models to human clinical trials, the need for absolute quantification of genetic material becomes critical. Traditional qPCR, while useful, often lacks the sensitivity and precision required for the complex matrices and low-abundance targets found in clinical samples.
This is where Digital PCR (dPCR) has emerged as the gold standard. At Accelevir, we leverage advanced ddPCR platforms to provide the regulatory-ready data needed to de-risk IND and clinical programs. Here is how we apply this technology to solve key bioanalytical challenges.
1. Precise Vector Copy Number (VCN) Analysis
Determining the average Vector Copy Number (VCN) per cell is a critical safety and dosing metric for gene-modified cell therapies. Regulatory guidelines require precise monitoring to mitigate the risk of insertional mutagenesis.
Unlike qPCR, which relies on standard curves, ddPCR offers absolute quantification by partitioning the sample into thousands of nano-reactions. This allows us to accurately measure VCN even in samples with variable efficiency or PCR inhibitors, providing the robust data required for drug product characterization.
2. Ultra-Sensitive Biodistribution & Shedding
Tracking where a viral vector goes (biodistribution) and how it leaves the body (shedding) is a fundamental component of safety testing.
We utilize GLP-compliant ddPCR workflows to detect vector genomes in a wide variety of biological matrices—from blood and bone marrow to tissue biopsies. The superior sensitivity of ddPCR allows us to detect low-level persistence in non-target tissues that other methods might miss, ensuring a complete safety profile for your biodistribution studies.
3. Accurate Viral Vector Titration
For AAV, Lentivirus, and other viral vectors, knowing the exact titer is essential for manufacturing consistency and accurate dosing.
We offer viral vector titration services using ddPCR to quantify physical titers with unmatched precision. By eliminating the variability of standard curves, we provide a reproducible metric that supports lot-release testing and comparability studies across manufacturing runs.
Partner with a dPCR Expert
Whether you are developing an in vivo gene therapy or an ex vivo engineered cell product, the quality of your molecular data drives your program’s success. Accelevir’s deep expertise in digital PCR assay development and validation ensures you have the precise, compliant answers you need.
Start your project with our molecular team today to discuss your ddPCR requirements.