Gene Therapy Bioanalysis 

Over a decade of experience in gene and cell therapy bioanalysis, working with more than a hundred sponsors building customized assay solutions that meet the relevant regulatory standards, from GxP to CLIA and IDE/IVDR.

Extensive experience providing bioanalytical solutions for gene therapy programs across both viral and nonviral vectors, including the development and implementation of appropriate clinical screening assays for the inclusion of patients in trials. Commonly requested assays include:

• Non-clinical biodistribution (PCR)
• Anti-vector antibodies (ADA)
• Neutralizing anti-vector antibodies (NAb)
• Anti-vector ADA and NAb assays for CLIA/IDE/IVDR use
• Anti-transgene antibodies (ADA)
• Neutralizing anti-transgene antibodies (NAb)
• Transgene detection/quantitation
• Biomarkers
• Cellular immunogenicity (ELISpot or Flow Cytometry)

Committed to supporting our customers’ gene therapy innovations, we have invested in state-of-the-art instrumentation that delivers the sensitivity and reliability these analyses demand. Our technology suite for transgene detection, immunophenotyping, persistence, cellular or humoral immunogenicity assays, and more includes:

• Flow cytometry
• qPCR/ddPCR
• ELISA
• ELISpot

Gene therapy involves the treatment of a disease or condition at the genetic level, which covers a broad category of therapeutics with a variety of structures. Viral vectors, such as adeno-associated viruses (AAVs) and lentiviruses (LVs), or nonviral delivery mechanisms, such as lipid nanoparticles (LNPs) or naked DNA/RNA, can be leveraged to deliver functional gene copies in vivo to correct the effects of a disease-causing mutation. Wild-type and modified AAVs are some of the most frequently used viral vectors for gene therapy work today due to the lack of pathogenicity and efficient transduction of host cells.

With its targeted nature, gene therapy has shown great promise for the treatment of numerous diseases such as various cancers, Parkinson’s, Alzheimer’s, heart disease, diabetes, and more. However, the complexities that make gene therapies so successful also make the bioanalytical support for drug development challenging. 

Recent regulatory guidance provides some of the expectations for monitoring the safety and efficacy of these products, but it is crucial to partner with a CRO that has a deep scientific understanding of the field of gene therapy and is up to date on the most recent developments for bioanalysis.