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"Realising the promises of next generation therapies"

Day 2 - Tuesday 4th July 2023


  • EU legal requirement and regulatory framework for classification of advance therapies
  • Comparative analysis between EU and US guidelines
  • Classification Criteria in Europe and US
  • Nuances that may affect development
  • Challenges in quality and regulatory perspective
  • Main sources of quality issues, and solutions that have been developed?
  • Best practices for robust regulatory strategies
  • Harmonisation among the various regulatory agencies
  • Tendon structure is hierarchically organised with defined subunits and membranous envelopes
  • Biologically distinct tendon cell sub-populations exist within these distinct compartments
  • Tendon-derived cell population promote distinct localised and far reaching immunological

Nick Forsyth, Faculty Dean of Research, Keele University

  • Elucidating the molecular mechanisms that underlie the maintenance of the integrity of the hPSC
    genome, and how disruption of these mechanisms can lead to undesired genetic changes
  • We are also studying the functional effects of genetic changes on the behavior of hPSCs in vitro.
  • Demonstrating that the dominance of variant hPSCs in mosaic cultures is enhanced through
    competitive interactions resulting in elimination of wild-type cells, akin to the cell competition
    behavior described in other cell and developmental models.
  • Understanding of the causes and consequences of genetic changes in hPSCs will help inform
    approaches to minimise their occurrence in hPSC cultures destined for clinical or research use.

Dr Ivana Barbaric, Centre for Stem Cell Biology, School of Biological Sciences, The University of
Sheffield, UK

Cecilia Gotherstrom, Research Group leader, Assoc Prof. in Stem Cell Science, Karolinska Institute

  • The experimental evidences strongly suggest that embryonic stem (ES) cell lines can be created from
    human blastocyst-stage embryos and stimulated to develop into practically all types of cells found in
    the body.
  • Cellular treatments produced from Stem cells have attracted fresh interest. The potential utility of
    Stem cells for gene therapy, tissue engineering, and the treatment of a wide spectrum of currently
    untreatable diseases is simply too vital to ignore; however, further improvements in our
    understanding of the basic
    biology of ES cells are required to deliver these forms of therapy in a safe and efficient manner.
  • In this meeting, I’ll share my research using ES cells and how they can be used to treat
    hematopoietic and neurodegenerative disorders.

Dr. Anand Srivastava, Chairman and Co-founder, Global Institute of Stem Cell Therapy and

  • 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


Mohamed Oubihi, Founder and CEO, YAKUMED


  • Can pharma companies demonstrate the cost-effectiveness of this therapies?
  • What are the innovative payment plans between pharma and payers?
    How to leverage, engage, and coordinate patient care to enable individual patient outcomes
  • Addressing the need for patient assistance programmes
  • Exploring alternative distribution capabilities for reimbursements


  • Changes in value assessment framework to improve adaptation
  • Encouraging payer decision-making while supporting patient access
  • How to ensure sustainability for healthcare system and patients
  • Innovative payment models to ensure timely access and alleviate the financial burden

Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross

  • Breakthroughs and innovations in research and development
  • Implementing next-generation technologies
  • Novel pricing and market access strategies pharma will employ to increase patient access
  • Various CGTs in the pipeline
  • What will the next decade hold for these life-changing therapies?


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