The new frontier: the bioanalysis of cell and gene therapies
Our sister site Bioanalysis Zone has launched a new podcast series to explore the important role of bioanalysis in cell and gene therapies!
Cell and gene therapies are comprised of multiple elements including living cells or viral vectors and therefore pose additional bioanalytical challenges compared with chemical or biological drugs. The capability of bioanalytical methods to monitor cell and gene therapy safety and efficacy plays a significant role in the therapy’s success. In this six-part series, Ellen Williams, Digital Editor of Bioanalysis Zone, is joined by industry experts exploring topics from regulatory implications to immunogenicity testing, delving into every corner of the cell and gene therapy landscape in bioanalysis.
Regulatory implications for cell and gene therapies
Executive Director and Global Head of Regulatory Services at QPS Holdings, LLC (DE, USA) Kimberley Buytaert-Hoefen speaks about her experience with CGT approvals and the barriers to getting these important therapeutics to the patient. We discuss key barriers to CGT approvals including manufacturing and immunogenicity and Kim voices her excitement for the newly approved CRISPR-Cas9 technology to treat sickle cell disease.
Immunogenicity testing for cell and gene therapies
We spoke to Senior Director of Bristol-Myers Squibb (NJ, USA) Johanna Mora about her experience with immunogenicity testing for CGTs and how she prepared for the expansion of BMS’s portfolio to include CGTs. We discuss the importance of having diverse teams with a broad range of experiences to solve complex problems, as well as the role of artificial intelligence in improving our efficiency and spotting patterns in the data.
Using LC–MS/MS for quantitative assays to support gene therapies
Daniel Schulz-Jander, Senior Director of Mass spectrometry Bioanalysis at QPS Netherlands (Groningen), explains how he uses immune-precipitation and immunoaffinity LC–MS/MS techniques for his work relating to macromolecular pharmaceuticals like gene therapies, as well as their benefits and challenges. Daniel also covers bottom-up, middle-down and top-down approaches and their suitability for clinical support versus R&D.
ddPCR for cell therapies
We’re joined by Sebastian Guelman, Senior Principal Scientist for Bioanalytical Sciences from Genentech (CA, USA). Sebastian talks about his experience with optimizing ddPCR assays to cell therapy targets and gives us his top tips for ensuring ddPCR assays run smoothly. We discuss his contributions to a recent White Paper and how these publications are integral tools for the bioanalytical community.
qPCR vs ddPCR for cell and gene therapies
Paul Byrne, Senior Director of Genomics from ProtaGene (MA, USA), describes his experience working with qPCR vs digital and digital droplet PCR assays and advises on their pros and cons, as well as when it’s best to use each.
Assessing efficacy and safety of CGTs using biomarkers
Catherine Vrentas, Life Sciences Lead Associate at Booz Allen Hamilton (VA, USA), explains how biomarkers can be used to predict the therapeutic outcome and efficacy of CGTs and the biomarkers in particular that have shown strong predictive value. We delve into the common challenges of working with biomarkers in CGTs and how biomarker kits can be adapted for CGT work.
