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"Realising the promises of next generation therapies"

Day 2 - Tuesday 24th October 2023

Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross


  • An introduction to the way ATMPs are commissioned in England, including what has been achieved to date

Ben Doak, Head of Innovative Treatments, NHS England

  • targetable VLPs for selective tissue tropism.
  • Enhanced safety for gene therapy approaches through selective tissue targeting.
  • Large cargo capacity (36.5 Kb) for building more regulated gene circuits.

Dr Matthias Bozza, Director, Vector Biopharma AG

  • Compacting and modularising mRNA product manufacture to accelerate discovery, and scaling to align with demand.
  • Implementing novel intensification technologies at GMP level for pandemic readiness.
  • Evaluating the opportunity of smart manufacturing to transform manufacturing methods towards costeffective commercialisation.
  • Democratising access to advanced mRNA therapies and vaccines through novel continuous manufacturing.

Francois Hallac, Research Associate, King’s College London

  • 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 behaviours

Vera Citro, P4FIT Early Stage Researcher, Keele University

  • Introduction of GCT Market Potential and Trends in Japan (commercial, regulatory and reimbursement)
  • Opportunities in Japanese GCT market (political considerations and incentives)
  • Challenges and Recommendations

Mohamed Oubihi, Founder and CEO, YAKUMED


  • 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

  • Past (learnings…)
  • Benefit Structures / Cost / Contracting (e.g., Outcomes Based)
  • Evidence & its Evaluation
  • Special Considerations (e.g.,

– Dealing with surrogate endpoints
– Modifications & Flexibilities for Evaluation of Rare Diseases)

  • Examples:

– Gene Therpaies
– Cellular Therapies
– Aduhelm & Leqembi (not gene therapies… but relevant for general understanding)

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?

Yue (Dina) Du, Senior Scientist, Gene Therapy R&D, AstraZeneca

Chris Williams, SVP Corporate Development, Autolus Ltd.
Stefanos Theoharis, Independent Expert
Reagan Jarvis, Chief Executive Officer and Co-founder, Anocca

Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross

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