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"Realising the promises of next generation therapies"

Day 1 - Thursday 9th March 2023

  • Complex challenges and key considerations for cell & gene therapy development
  • Pre-clinical solutions to address scientific, regulatory, and commercial risks to accelerate development
  • How to maximize the potential and simplify the transition from basic cell therapy research to clinical manufacturing and beyond
  • Current trends and innovations to advance cell & gene therapies


Zhimei Du, MD/PhD,  VP of Process Sciences, Atara Biotherapeutics

Wenliang  Dong, Chief Operating Officer, ORCA Therapeutics


  • Focus on more challenging purification requirements of gene therapy and oncolytic viruses
  • Factors that can complicate the downstream processing for viral vectors
  • Tailored purification process design for greater process understanding
  • Methodologies, cell manufacturing platforms and technologies that contributes to defining CAR-T cell
  • Core components of autologous CAR-T cell manufacturing that remains constant
  • Quality requirements & quality control assays enabling the release of clinical CAR-T cell products for infusion
  • Challenges in cell manufacturing
  • Recent advances and benefits from different techniques
  • Potential for different applications wide usage of microfluidic devices.
  • Risk assessment analysis
  • Equipment requirements and facility characteristics
  • Beneficial effect of regulatory T cell transfer on the maintenance of self-tolerance and immune homeostasis in different conditions


  • Overcoming the manufacturing obstacles to meet specifications and regulatory approval
  • Ongoing innovation in CGT process development, manufacturing, and clinical testing based on quality by design (QbD)
  • Early implementation of broad and robust analytics to accelerate optimization
  • Planning for comparability studies for ongoing innovations from discovery to commercialisation


  • Upstream and downstream processes for production of rAAV
  • Analytical methods for quantification of rAAV quality attributes
  • Key challenges in transitioning from traditional to newer methods
  • Methods of detect and classify key contaminants in CGT
  • New advances in technology that play a significant role in ensuring the safety of therapeutics
  • Design model where contamination is minimized before manufacturing begins
  • Increase control and implementation of QbD principles and adoption of new assay methodologies


  • Monitoring and control challenges resulting from the extensive range of cell and gene therapy products
  • Understanding the intricacy and processes required to produce these products
    • Challenges in perspective and implementation of regulatory framework
  • Muscular dystrophies as paradigm for orphan drug development
  • Most promising gene therapy strategies for the treatment of muscular dystrophy
  • Future perspectives; orphan drugs in the pipeline

Alberto Malerba, Lecturer in Gene Therapy, Royal Holloway, University of London

  • Process challenges associated with manufacturing gene therapies and strategies in overcoming them
  • Major components that impact innovation and optimization
  • What can manufacturers do to guarantee a seamless process as the treatment progress form early to commercial stage and through regulatory approval?
  • Future for next generation technologies and process developments
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