CELL & GENE THERAPY WORLD CONGRESS 2023 EUROPE
"Realising the promises of next generation therapies"
Day 2 - Tuesday 24th October 2023
Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross
ADVANCE THERAPEUTICS
- 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
MARKET ACCESS, AND REIMBURSEMENT
- 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
Research
- 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?
MODERATOR:
Yue (Dina) Du, Senior Scientist, Gene Therapy R&D, AstraZeneca
PANELLIST:
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