HERCULES, Calif. —Five researchers developing new methods for cell and gene therapy quality control (QC) using Bio-Rad Droplet Digital PCR (ddPCR) technology presented their research at the Gene Therapy Analytical Development meeting in November and the Cell Therapy Analytical Development Summit, held in December. The studies addressed how ddPCR technology is helping to alleviate an array of pain points in cell and gene therapy QC in the areas of product safety testing, QC scale-up, and contamination detection.
Below are highlights from the talks during the conferences that demonstrate the unique value of ddPCR in performing QC for cell and gene therapies.
Gene Therapy Safety Testing
Speaker: Matthew Hankinson, Associate Director, Analytical Development, Allergan
Accurately measuring viral titer in gene therapies is critical for determining a safe and effective patient dose. Matthew Hankinson, Associate Director, Analytical Development, Allergan, discussed the advantages of ddPCR over qPCR in AAV titer analysis. He reported that ddPCR results had a coefficient of variation (CV) of 15%, while qPCR produced a CV of 50%. Hankinson noted that since ddPCR delivers more reliable results, his team has transitioned to using the technology instead of qPCR to help determine proper patient dosage.
Speaker: Johannes Lengler, PhD, Leader, Cell Culture & In Vitro Testing Group, Takeda
Using ddPCR technology, Johannes Lengler, PhD, Leader, Cell Culture & In Vitro Testing Group, Takeda, shared how he and his team developed an in vitro potency assay for AAV-mediated gene therapy for the treatment of hemophilia. Their team’s ddPCR method achieved 98% accuracy and a CV of 1.4%.
Speaker: Anthony Leyme, PhD, Senior Scientist, Analytical Development, ElevateBio
Anthony Leyme, PhD, Senior Scientist, Analytical Development, ElevateBio, described how they are developing reliable and highly sensitive gene therapy assays based on ddPCR. Assays they create to determine titer of infectious lentiviral vectors reduce hands-on time, enabling scientists to skip cell lysis and therefore increasing their output more than twofold. They were also able to transition from a 6-well format to 96-well format and achieve an assay CV of less than 5%.
Gene Therapy Scale-Up and Contamination Testing
Speaker: Alice Lin, PhD, Associate Director, Quality Control, Fate Therapeutics
Using induced pluripotent stem cells (iPSCs) as a substrate for allogeneic cell therapy products supports more extensive genetic engineering. However, due to the intrinsic instability of iPSCs, patients are exposed to additional risks related to genetic integrity. Alice Lin, PhD, Associate Director, Quality Control at Fate Therapeutics, reviewed strategies being used to scale up quality control testing of the company’s off-the-shelf hemopoietic stem cell products using ddPCR. Generating standard curves, which is required for the analysis of qPCR-based assays, can take years of experience by a researcher to do this process well, creating opportunities for human error. By implementing ddPCR to quantify residual iPSCs and DNA, Lin’s team was able scale up more quickly.
Speaker: Jennifer Dashnau, PhD, Head of Analytical Development and Quality Control, Century Therapeutics
Century Therapeutics is using multiple methods to test for iPSC contamination, according to Jennifer Dashnau, PhD, Head of Analytical Development and Quality Control. Based on next-generation sequencing methods, Dr. Dashnau and her team selected genomic loci to monitor contamination. They view ddPCR as a powerful option for quantifying these genetic markers since ddPCR technology can detect as few as three contaminating iPSCs in one million total cells.
Please visit bio-rad.com/ddPCR-CellAndGeneTherapy learn more about Bio-Rad’s portfolio of cell and gene therapy QC products.
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