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Ideal Assay Profile™ (IAP): Potency Assay Development and Validation to Support Gene Therapy (GT) Product Approval

All Cell & Gene Therapy (CGT) products must have tests in place to demonstrate identity, purity, and strength (including potency).  Different from other tests required for lot characterization and release, the cell-based potency assay needs to be customized for each product to ensure it adequately reflects the complex mechanism(s) of action (MOA) by gene expression (transfer of the genetic sequence into the cells) and functional activity (the transferred gene produces the desired biological effect).

To demonstrate this complex MOA, CGT therapy products typically have critical quality attributes (CQAs) with higher variability, compounded with fewer manufacturing runs with which to establish critical process parame­ters (CPP). As a result, significant challenges in potency assay development include establishing appropri­ate assay suitability criteria and generating sufficient data for statistical analysis. To overcome these challenges, unique approaches may be needed, such as developing multiple assays to characterize both expression and activity and employing alternative statistical techniques to account for smaller sample sizes.

The Webinar will not only focus on the challenges to develop an Ideal Assay Profile for cell-based potency assay but will also walk you through its transition from the R&D stage to the GMP (QC) lab to support late-stage clinical trials and commercialization.  

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Key Messages: 

  • Start potency assay discussion/development at early stages
  • Discuss the layout and POC results during early interaction with regulatory bodies
  • Strategize your potency assay layout in a phase appropriate manner
  • Have a backup plan ready
  • Involve the QC lab early
  • Prepare QA to qualify R&D vendors and validate R&D equipment & software
  • Create a Process Map to streamline the transfer into the GMP lab

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Moderated by: Vibhor Gupta - Director of Business Operations, Cell and Gene Therapy at Pharmaron

Key Opinion Leader:  Xiaodong Fang, Ph.D - Sr. Director of Bioanalysis and Bioassay at Asklepios BioPharmaceutical (AskBio)

Dr. Xiaodong Fang, Ph.D. is the Sr. Director of Bioanalysis and Bioassay at Asklepios BioPharmaceutical (AskBio), a wholly owned, independently operated subsidiary of Bayer AG. One major responsibility for his group is the bioassay support for the gene therapy programs. Xiaodong received his Ph.D. in Biochemistry and Molecular Biology from Sun Yet-sen University in China and post-doc training in Cell Biology and Molecular Genetics from University of Pennsylvania School of Medicine.

Silvia Pires Lourenco, M.S. - Associate Director, Scientific Operations for the Cell and Gene Therapy Division at Absorption Systems, a Pharmaron company

Silvia Pires Lourenco, M.S. is an Associate Director, Scientific Operations for the Cell and Gene Therapy Division at Absorption Systems. Since joining in October 2019, Silvia’s focus is on the development of Potency Assays for drug product release testing and bioanalytical methods for preclinical and clinical support, including but not limited to biodistribution, shedding and NAb assays. During her research career, Silvia’s projects focused on developing two pronged approaches using lentiviral vectors as a cure for beta-globinopathies. Silvia has presented her work in multiple conferences and has contributed to the preparation of several manuscripts/review articles, in multiple fields of life sciences including Biochemistry, Metabolic disorders, and Cell & Gene therapy.

Deepika Jain, Ph.D., MBA - Project Manager – CGT, GMP at Absorption Systems, a Phamaron company

Deepika Jain, Ph.D., MBA is the Scientific Operation Manager – CGT, GMP at Absorption Systems, supporting Cell & Gene Therapy services since Dec 2020. She has a wide scientific experience spanning academia, high throughput medical diagnostic devices, pharmaceuticals and CROs.  Her research focus in academia was translational medicine, with an emphasis on pulmonary and cardiovascular physiology and immunology, characterizing innate immune mechanisms in animal models of acute lung injury.  Since 2015, she has led multiple projects through validation & verification activities in GMP environment taking them from development through BLA filing and 410K submission. She has over 8 years of experience leading scientific teams, managing collaborations and day-to-day activities of Analytical/QC labs, in depth scientific and quality data review as well as managing project timelines for multiple oncology, cell and gene therapy NMEs. She has authored 20+ scientific papers in peer-reviewed journals and has presented in multiple scientific conferences in her scientific career

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