CELL AND GENE THERAPY INSIGHTS

Spotlights 2024

Gene therapy  analytics and CMC
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July

Gene therapy analytics and CMC

Stuart Beattie
Guest Editor:
Stuart Beattie, Associate Director, Gene Therapy Global Regulatory CMC Clinical Lead at Biogen
  • How best to leverage the expanding analytical toolkit for viral vector characterization and QC?
    • Maximizing the benefits of high-performance liquid chromatography (HPLC) and liquid chromatography/ mass spectrometry (LC/MS)
    • Harnessing the cutting edge in capsid characterization tools
    • Characterizing aggregation
  • Addressing ongoing CMC challenges and associated regulatory evolution:
    • Dissecting key recent guidances—implementation and points to consider for the field
    • Where is further guidance and standardization most needed?
      • How many assays do you require for your potency assay matrix?
      • What does a phase-appropriate potency assay actually look like?
      • What if none of the your potency assays fail to correlate with a positive clinical outcome?
    • What are the keys to successfully integrating QA into viral vector manufacture?
    • Comparability—how to find the optimal balance in early development (costs vs quality)?
    • How will the empty-full-partially full capsid analysis picture continue to develop?
  • Exploring key areas of need for (affordable) analytical innovation and its application
    • Priorities for future lentiviral vector-specific analytical technology innovation
    • How is the LNP QC/analytics field evolving?
    • Rapid/real-time process analytical technologies
    • Key directions for derisking vector manufacture through analytical technology
      • How can we make assays more agnostic/standard across different vectors/capsids?
      • What impact are instruments capable of multiple in-process assays having on cost and efficiency?
      • How will we continue to move towards automating data analysis and enhancing predictive abilities?
Scale-up/scale-out of cell & gene therapy manufacturing
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September

Scale-up/scale-out of cell & gene therapy manufacturing

  • Cell therapy downstream processing
    • Exploring recent technological innovations and initiatives driving shorter manufacturing timeframes, reduced Cost of Goods, improvements in flexibility and interoperability, and distributed manufacture
    • Optimizing cell therapy fill-finish: what are the key innovation gaps?
  • How are developers interpreting and responding to recent regulatory guidance regarding potency assays?
    • Can we identify any best practices, particularly in terms of potency assay matrix development?
    • What are the preclinical ‘must-do’s’ to prepare for potency assay success?
    • How can we advance to improved, standardized analytical toolkit to allow developers to implement potency assay matrixes that correlate with clinical outcomes?
  • Comparability: what exactly do we need to show, and how to prepare from an early stage?
    • Finding the optimal balance between product development and speed to patient post-IND
    • What knowledge can we leverage from cell therapy products that have already been through the clinic in terms of how best to approach process changes?
  • What does phase-appropriate analytical control of cell therapy manufacture look like?
  • Are novel cell therapy analytical tools and technologies delivering the required degree of repeatability and precision?
  • How can we move further towards the automation of data analysis in cell therapy manufacture?
  • How are regulatory CMC compliance strategies and analytical toolkit innovation evolving to address the ever-increasing complexity of engineered cell therapy products?
    • What do/don’t we understand about the associated risk?
    • How and where specifically can off-the-shelf therapy developers leverage the fund of autologous cell therapy-derived CMC knowledge to help advance the allogeneic cell therapy field?
  • Dissecting critical areas of international regulatory divergence impacting cell therapy CMC and QC
Cell therapy downstream processing & analytics
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November

Cell therapy downstream processing & analytics

  • Cell therapy downstream processing
    • Exploring recent technological innovations and initiatives driving shorter manufacturing timeframes, reduced Cost of Goods, improvements in flexibility and interoperability, and distributed manufacture
    • Optimizing cell therapy fill-finish: what are the key innovation gaps?
  • How are developers interpreting and responding to recent regulatory guidance regarding potency assays?
    • Can we identify any best practices, particularly in terms of potency assay matrix development?
    • What are the preclinical ‘must-do’s’ to prepare for potency assay success?
    • How can we advance to improved, standardized analytical toolkit to allow developers to implement potency assay matrixes that correlate with clinical outcomes?
  • Comparability: what exactly do we need to show, and how to prepare from an early stage?
    • Finding the optimal balance between product development and speed to patient post-IND
    • What knowledge can we leverage from cell therapy products that have already been through the clinic in terms of how best to approach process changes?
  • What does phase-appropriate analytical control of cell therapy manufacture look like?
  • Are novel cell therapy analytical tools and technologies delivering the required degree of repeatability and precision?
  • How can we move further towards the automation of data analysis in cell therapy manufacture?
  • How are regulatory CMC compliance strategies and analytical toolkit innovation evolving to address the ever-increasing complexity of engineered cell therapy products?
    • What do/don’t we understand about the associated risk?
    • How and where specifically can off-the-shelf therapy developers leverage the fund of autologous cell therapy-derived CMC knowledge to help advance the allogeneic cell therapy field?
  • Dissecting critical areas of international regulatory divergence impacting cell therapy CMC and QC