Author:
Hays Amanda,Wissel Mark,Colletti Kelly,Soon Russell,Azadeh Mitra,Smith Justin,Doddareddy Rajitha,Chalfant Melanie,Adamowicz Wendy,Ramaswamy Swarna Suba,Dholakiya Sanjay L.,Guelman Sebastian,Gullick Bryan,Durham Jennifer,Rennier Keith,Nagilla Pruthvi,Muruganandham Anamica,Diaz Manisha,Tierney Cassandra,John Kaarthik,Valentine Jenny,Lockman Timothy,Liu Hsing-Yin,Moritz Benjamin,Ouedraogo Jean Paul,Piche Marie-Soleil,Smet Muriel,Murphy Jacqueline,Koenig Kaylyn,Zybura Agnes,Vyhlidal Carrie,Mercier Jonathan,Jani Niketa,Kubista Mikael,Birch Donald,Morse Karlin,Johansson Oskar
Abstract
Abstract The emerging use of qPCR and dPCR in regulated bioanalysis and absence of regulatory guidance on assay validations for these platforms has resulted in discussions on lack of harmonization on assay design and appropriate acceptance criteria for these assays. Both qPCR and dPCR are extensively used to answer bioanalytical questions for novel modalities such as cell and gene therapies. Following cross-industry conversations on the lack of information and guidelines for these assays, an American Association of Pharmaceutical Scientists working group was formed to address these gaps by bringing together 37 industry experts from 24 organizations to discuss best practices to gain a better understanding in the industry and facilitate filings to health authorities. Herein, this team provides considerations on assay design, development, and validation testing for PCR assays that are used in cell and gene therapies including (1) biodistribution; (2) transgene expression; (3) viral shedding; (4) and persistence or cellular kinetics of cell therapies.
Graphical Abstract
Publisher
Springer Science and Business Media LLC
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