Cell and Gene Therapy World Congress 2023
"Realising the promises of next generation therapies"
23rd - 24th October 2023, Copthorne Tara Hotel London Kensington, LONDON, UK
INTRODUCTION
Cell and Gene therapies are life-changing new categories of medicines whose full potential is only just beginning
to emerge. They are cutting-edge and rapidly progressing fields of Biological Therapies. Both therapies aim to
treat, prevent or potentially cure diseases and both approaches offer the potential to diminish the fundamental
cause of genetic and acquired diseases by substituting the missing protein or cells causing the disease’s
symptoms. Cell and gene therapies may offer longer lasting effects than traditional medicines. They are
overlapping fields of biomedical research and treatments. However, cell and gene therapies work
differently. Cell therapy aims to treat diseases by restoring or altering certain sets of cells or by using cells to
carry a therapy through the body while gene therapy treats diseases by replacing, inactivating or introducing
genes into cellsā either inside the body (in vivo) or outside of the body (ex vivo). Some therapies are considered
both cell and gene therapies. These therapies work by modifying genes in specific types of cells and inserting
them into the body.
With the promising significance of Cell and Gene therapy, it is poised to transform the healthcare and
biopharma industry soon. The market is estimated to increase expeditiously, encompassing sales forecasts of
EUR 27.9 billion by 2026. The development of biological therapies is rapidly expanding, however, it poses
different challenges to research and development, authorisation, manufacturing, accessibility, and ensuring
safety throughout the lifecycle of the product.
The two-day Congress will provide an interactive, cutting edge, and comprehensive discussions in collaborative
format led by key experts with intimate knowledge in the industry. Gain practical strategies and best practices
on challenges, innovations, technologies, and concepts to help you gain access to the booming CGT market.
We are putting together an agenda that focuses on “Realising the promises of next generation therapies”. We
are gathering high calibre expert speakers to participate and share their knowledge and expertise through
Keynote Presentations and Panel discussions, addressing all aspects of the industry.
We look forward to welcoming you to the CGT World Congress 2023!
ā¢ Clinical Research Organizations
ā¢ Research and Development
ā¢ Personalised Medicine
ā¢ Regenerative Medicine
ā¢ External R&D Innovation
ā¢ Innovative Medicine
ā¢ Rare and Ultra-Rare Diseases
ā¢ Cell and Gene Therapy
ā¢ Translational Science
ā¢ Molecular Geneticist
ā¢ Program Management
ā¢ Patient and Patient Groups
ā¢ Public Affairs
ā¢ Medical Affairs
ā¢ Regulatory Affairs
ā¢ Market Access
ā¢ Pricing and Reimbursement
ā¢ Health Economics Outcomes Research
ā¢ Commercial Development
ā¢ Investments and Funding
ā¢ Product Specialist
ā¢ Global Strategic Services
ā¢ Business Planning and Operations
ā¢ Pharmacies
ā¢ Academia
- Gain the latest insights to successfully navigate the complex access landscape of CGT
- Explore ways on how to strategically navigate the state of the art technologies in cell and gene therapies
- Understand approaches in advancing research and drug developmentĀ
- Learn the production process which is crucial for new players entering the field
- Know how to engage early with regulators and payers to enable swift roll-out of new therapies for the benefit of the patients
- Have a meaningful conversation on how to improve access efficiently to innovative treatment in order to reach patients in a shorter period of time
- Network and collaborate with key stakeholders, regulators, academia, HTAs, and payersĀ
CELL & GENE THERAPY WORLD CONGRESS 2023 EUROPE
"Realising the promises of next generation therapies"
Day 1 - Monday 23rd October 2023
Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross
OVERVIEW, CURRENT LANDSCAPE & FUTURE TRENDS
- The importance of stakeholder engagement: perspectives across different functions
- Creating a CGT platform: stakeholder engagement as a tool to support not only the development/commercialization of specific assets, but to build a sustainable platform
- Complex challenges and key considerations for cell & gene therapy development
- Current trends and innovations to advance cell & gene therapies
Stuart M Curbishley, Chief Manufacturing and Chief Development Officer, adthera bio Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross
Yue (Dina) Du, Senior Scientist, Gene Therapy R&D, AstraZeneca
Daniel Gibson, Director of Cell and Gene, Anthony Nolan
- Swissmedic Innovation Office:
-Mission, Role and Responsibilities
– Presence and visibility near the innovation, promote Innovation
– Early dialogue and reduce barrier between Regulator and Stakeholders
Swissmedicās Competence Center Advanced Therapy Medicinal Products (ATMP) :
Advanced Therapy Medicinal Products as Pilot Project to develop the Innovation Office
– ATMP Risk Based —Ā — Approach ā accelerate and facilitate the access of innovative therapies to the patients
Dr. Thomas Schochat, Clinical Assessor, Division Advanced Therapy Products, Swissmedic
- Improving clinical trials, strengthening regulatory authorities
- Foster use of health data for innovation
- Promoting innovative research projects
- Reimbursement for gene therapies and ATMPs
Thomas Muller, Head Of Directorate General, Federal Ministry of Health Germany
- T-Cell Receptors modified T-cell therapies (TCR-T) unlock immense tumour-selective target space
- Identifying the best targets and TCRs requires a systematic Precision Biology approach.
- Precision Biology platform that uses programmable human cells to systematically map the target space and engineer TCRs.
Reagan Jarvis, CEO and Co-founder, Anocca
- Utilising NHSBTās supply chain and infrastructure to deliver ATMPs into NHS
- Manufacturing of red blood cells in vitro for RESTORE clinical trial
- Manufacture of gene therapy products at NHSBTās Clinical Biotechnology Centre
Dr Lilian Hook, Director Cell, Apheresis & Gene Therapies, NHS Blood and Transplant
- PDC*line is a new potent and scalable therapeutic cancer vaccines based on a proprietary allogeneic
cell line of Plasmacytoid Dendritic Cells - PDC*line is much more potent to prime and boost antitumor antigen, including neoantigens, specific
cytotoxic T-cells than conventional vaccines and improves the response to checkpoint inhibitors - The technology can be applied for any cancer
Ā Eric Halioua, President & Chief Executive Officer, PDC*line Pharma
- Introduce the development of novel gene therapies
- Introduce pivotal non-clinical studies
- Discuss the relevant non-clinical considerations within the context of a case study of a novel gene
therapy
Mark Singh, Non-Clinical Manager, Cell and Gene Therapy Catapult
- 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
- Present innovative approaches for flexible Cell Therapy facility design solutions.
- Provide an overview of the latest Cell Therapy manufacturing technologies.
- Outline the benefits of implementing robust methods of process closure in the manufacturing
operations. - Discuss sustainability factors associated with the design of modern manufacturing facilities.
- Outline how all these factors can increase speed to market while reducing the environmental footprint
of a facility.
Stephen Judd, Process SME ā Biologics / Cell & Gene Therapy, DPS Group
- Why we need to consider the long-term when developing processes, even at phase I
- When is it appropriate to consider automation?
- Why automation early in the product development lifecycle matters.
Stuart M Curbishley, Chief Manufacturing and Development Officer, adthera
Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross
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 (learningsā¦)
- 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 therapiesā¦ but relevant for general understanding)
Eugean Jiwanmall, Senior Research Analyst, Independence Blue Cross
Ā
- 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