Recommendations for Method Development and Validation of qPCR and dPCR Assays in Support of Cell and Gene Therapy Drug Development

0583020 - BTÚ 2025 RIV US eng J - Článek v odborném periodiku
Hays, A. - Wissel, M. - Colletti, K. - Soon, R. - Azadeh, M. - Smith, J. - Doddareddy, R. - Chalfant, M. - Adamowicz, W. - Ramaswamy, S. S. - Dholakiya, Sanjay L. - Guelman, S. - Gullick, B. - Durham, J. - Rennier, K. - Nagilla, P. - Muruganandham, A. - Diaz, M. - Tierney, C. - John, K. - Valentine, J. - Lockman, T. - Liu, H. - Moritz, B. - Ouedraogo, J. P. - Piche, M. - Smet, M. - Murphy, J. - Koenig, K. - Zybura, A. - Vyhlidal, C. - Mercier, J. - Jani, N. - Kubista, Mikael - Birch, D. - Morse, K. - Johansson, O.
Recommendations for Method Development and Validation of qPCR and dPCR Assays in Support of Cell and Gene Therapy Drug Development.
AAPS Journal. Roč. 26, č. 1 (2024), č. článku 24. ISSN 1550-7416. E-ISSN 1550-7416
Institucionální podpora: RVO:86652036
Klíčová slova: real-time pcr * storage * cellular kinetics * dPCR * gene therapy
Obor OECD: Pharmacology and pharmacy
Impakt faktor: 4.5, rok: 2022
Způsob publikování: Open access
https://link.springer.com/article/10.1208/s12248-023-00880-9

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.
Trvalý link: https://hdl.handle.net/11104/0353352