Comparing Droplet Digital PCR to Quantitative PCR: Evolving the Gene Therapy Viral Vector Analytical Paradigm

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1 Comparing Droplet Digital PCR to Quantitative PCR: Evolving the Gene Therapy Viral Vector Analytical Paradigm Jarrod Dean Principal Research Associate, Analytical Development Sanofi Presented by GTC, a conference production company

2 Comparing Droplet Digital PCR to Quantitative PCR: Evolving the Gene Therapy Viral Vector Analytical Paradigm Jarrod Dean, Brenda Kinnes, Eric Pastor, Yinyin Huang, Francis Poulin, Claire Davies Jarrod Dean Principal Research Associate, Analytical Development Sanofi Biopharmaceutics Development: Framingham, MA GTCbio Digital PCR & qpcr Conference Boston, MA July 6 th 7 th,

3 Agenda Introduction to recombinant adeno-associated virus (raav) and it s use in Gene Therapy Description of a gene therapy program analytical paradigm Focus on dose defining assay (program deliverable impacts) Suitability of droplet digital PCR for the absolute quantitation of AAV vectors Comparison to traditional real-time qpcr Usefulness of droplet digital PCR as an orthogonal troubleshooting analytical tool Value assigning standards used for qpcr analyses Overcoming issues with difficult transgene configurations Conclusions and Acknowledgements 3

4 raav Mediated Therapeutics Advantages Non-pathogenic to humans Non-replicative: no Rep and Cap genes present Replaced by therapeutic gene of interest Low immunogenicity Multiple serotypes Infect both dividing and non-dividing cells, wide tropism Provides long-lasting expression of gene Stable episomal concatamer formation Disadvantages Constrained by packaging size of virus Difficult to package genes > 5 KB in size Dividing cells may lose efficacy over time Systemic delivery may be impacted by neutralizing antibodies Difficult to manufacture! 4

5 Gene Therapy Analytical Paradigms Adherence to FDA/CBER and EMEA guidance on Human Gene Therapy Products Constantly evolving with new emerging attributes and specifications Identification of Critical Quality Attributes for novel therapeutics based on: Manufacturing platform technologies Transgene configurations Limited clinical trial data (hard to score) Alignment of method development strategies U.S. Pharmacopeial (USP) requirements for analytical tests Develop per International Conference on Harmonisation (ICH) guidelines Adhere to corporate quality standards Overall goal is to release and characterize a safe and high quality product 5

6 What does a typical Viral Vector Analytical Paradigm look like? Strength / Content Product Variants Identity Concentration General Quality ph Purity Safety Endotoxin Is your product what you say it is? Process-Related Impurities Production Residuals Discrepancies in vector genomes per ml (vg/ml) titer may impact a program at various stages: Dose-defining attribute Clinical candidate screening and selection Accurate capacity planning for pre-clinical studies Regulatory agency interest (competitive space considerations) 6

7 ATTENTION! The entire presentation will be provided to registered attendees only at the conference! To view the rest of this exciting presentation and many more please register for the conference at

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