Cell and Gene Therapy World Congress 2023
"Realising the promises of next generation therapies"
3rd - 4th July 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 cells— 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!
• Clinical Research Organizations
• Research and Development
• Personalised Medicine
• Regenerative Medicine
• External R&D Innovation
• Innovative Medicine
• Rare and Ultra-Rare Diseases
• Cell and Gene Therapy
• Translational Science
• Molecular Geneticist
• Program Management
• Patient and Patient Groups
• Public Affairs
• Medical Affairs
• Regulatory Affairs
• Market Access
• Pricing and Reimbursement
• Health Economics Outcomes Research
• Commercial Development
• Investments and Funding
• Product Specialist
• Global Strategic Services
• Business Planning and Operations
• Pharmacies
• 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 development
- 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 payers
CELL & GENE THERAPY WORLD CONGRESS 2023 EUROPE
"Realising the promises of next generation therapies"
Day 1 - Monday 3rd July 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
Panelist:
Zhimei Du, MD/PhD, VP of Process Sciences, Atara Biotherapeutics
Wenliang Dong, Chief Operating Officer, ORCA Therapeutics
OVERVIEW, CURRENT LANDSCAPE & FUTURE TRENDS
- 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
PANELIST: Wenliang Dong, Chief Operating Officer, ORCA Therapeutics B.V.
- Setting the scene: comparison of rare disease/CGT and traditional pharma
- 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
Marianthi Psaha, Global Head Cell & Gene Therapy, Santen Pharmaceutical
- Challenges and obstacles in bringing this highly complex therapies to market
- Stakeholder collaboration on the road towards a solution
- Lessons learned and applications for new entrants
- Strategies that can be taken to improve timely market access and access for patients
Doug Danison, Head of Commercial, Cell and Gene Therapy Unit, Bayer
- Standardized approaches being developed to address complexity of trial design and lack of
experience with CGT trials - Considerations to overcome constraints in manufacturing capacity
- Strategies and best practices in facilitating patient recruitment and retention in CGT clinical trials
Amir Hefni, Global Head, Cell & Gene Therapy, Novartis
Zhimei Du, VP, Process Sciences, Atara Biotherapeutics
- 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
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
- Leveraging success of gene therapy pioneers to enable accelerated development
- Front-loading manufacturing investment to reduce clinical trial timelines
- Bloomsbury Genetic Therapies case study: building a de-risked portfolio for maximal clinical
translation and approvability
Adrien Lemoine, Co-Founder & CEO, Bloomsbury Genetic Therapies
- 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 – Biologics / Cell & Gene Therapy, DPS Group
Anthony Newcomb, Chief Operating Officer, VectorY
- Current GMP and general process validation concepts and practices that applies to CGT manufacturing
processes and analytical methods - Key challenges in analytical method validation and proposed solutions.
- Extensive regulatory requirements for a controlled validation
- Robust CMC package that creates a path toward commercialization
- Vigorous process validation strategy through the life cycle of these novel therapies
CELL & GENE THERAPY WORLD CONGRESS 2023 EUROPE
"Realising the promises of next generation therapies"
Day 2 - Tuesday 4th July 2023
STEM CELL SCIENCE
- 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
behaviours
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
Research
- 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?