Answering Frequently Asked Questions Around Challenges When Using qPCR, ddPCR and NGS in Regulated Studies for Cell and Gene Therapy Products
In April 2022, we discussed the role of molecular testing platforms, such as qPCR, ddPCR and NGS in enabling discovery and development of cell and gene therapy products. These technologies were recently introduced to bioanalysis of cell and gene therapy products, resulting in many unanswered questions on the appropriate use of these technologies in the field of bioanalysis, especially due to the lack of prescriptive guidance. We will use a redacted and anonymized qPCR case study to answer some of the questions that frequently come up.
Answering Frequently Asked Questions Around Challenges When Using qPCR, ddPCR and NGS in Regulated Studies for Cell and Gene Therapy Products
July 27, 2022 | 11:15 am ET
During our webinar in April 2022, Dr. Stephanie Pasas-Farmer discussed the role of molecular testing platforms, such as qPCR, ddPCR and NGS in enabling discovery and development of cell and gene therapy products. While these technologies are well-developed and well-supported in other areas of drug discovery and development, their presence in bioanalysis of cell and gene therapy products is a more recent development. This has resulted in many unanswered questions on the appropriate use of these technologies in the field of bioanalysis, especially due to the lack of prescriptive guidance. We will use a redacted and anonymized qPCR case study to answer some of the questions that frequently come up. We will then open the floor to discuss approaches to some common questions and requests.
Some topics to be discussed include:
- An approach to calculating group means for data sets with a small ‘n’ that are consistent, except for one or two points
- Are there any best practices or a statistical approach for the removal of outliers for a PCR method?
- Is it acceptable to re-test or remove data points if the justification is related to expectations of the biological response to treatment
- Approaches taken to visualize biodistribution data sets with many (e.g. >10) tissue types
- Identifying a threshold in terms of viral genomes (vg) or mRNA in particular tissue types where you are confident there will not be causative toxicity.