Cell & Gene Therapy

Stem Cell Science & Regenerative Medicines

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Key Industry Expert Speakers

Zhimei Du, MD/PhD

VP of Process Sciences

Atara Biotherapeutics

Marianthi Psaha

Global Head Cell & Gene Therapy, Corporate Officer

Santen Pharmaceuticals

Wenliang Dong

Chief Operating Officer

ORCA Therapeutics

Mohamed Oubihi Ph.D.

Founder and CEO

YAKUMED LIMITED

Alberto Malerba

Lecturer in Gene Therapy

Royal Holloway, University of London

Cecilia Götherström

Research Group leader, Associate Professor in Stem Cell Science

Karolinska Institutet

Cell & Gene Therapy World Congress 2023 Europe

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!

GAIN LATEST INSIGHTS ON:

  • 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 
WHO SHOULD ATTEND?                                 
This congress is specially created for valued stakeholders in the Cell & Gene therapy community: Presidents, Heads/Chiefs, VPs, Directors, and Managers in the area of:
 

• 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

STEM CELL SCIENCE & REGENERATIVE MEDICINES 2023

INTRODUCTION

Stem cell research, tissue engineering, and regenerative medicine hold the potential to revolutionize the way human diseases are treated, and many nations have therefore invested heavily in these genetically engineered components and its applications. Considerable research has enabled the fabrication of sophisticated grafts and cell manipulation technologies to analyze the molecular level of controlling cell behavior and repairing tissue. However, these revolutionary medicines are also controversial with many ethical and regulatory questions that impact a nation’s policies.

The upcoming 2023 Stem Cell Science and Regenerative Medicines Congress focuses on a diverse range of topics and addresses the challenges, obstacles, and opportunities for innovation in the design and execution of clinical trials.

This will also offer and provide a unique platform for knowledge sharing and convergence from Pharmaceuticals, Biotechnology, Regulatory, and many more.

We look forward to welcoming you to the CGT World Congress 2023!

Gain insights on:

  • Trends in stem cell-based disease modelling and drug discovery
  • Challenges, solutions, and progress on cell manufacturing and standardization
  • The clinical and regulatory landscape of regenerative products
  • Commercializing stem cell therapy and regenerative medicine and the importance of collaboration
  • Ethical, legal, and social issues in regenerative medicine
  • Driving productivity and economic growth in advanced therapies
  • Current and Future approaches to GMP manufacturing and Standardization
  • Scalable production of Human Induced Pluripotent Stem Cells
  • Cell manipulation platform
  • Regenerative Medicines Models and Technologies
  • Stem Cell and Tissue Engineering Applications

Who Should Attend

Network with scientists, analysts, senior VPs, Heads, Directors, and Managers of leading biopharmaceutical, biotech companies, Governments agencies, and Academia, whose responsibilities includes:

  • Pre-clinical and clinical trials of stem cell therapy
  • Stem Cell Manufacturing
  • Stem Cell Therapy
  • Stem cell reprogramming
  • Regulatory policies
  • Molecular Stem Cell Biology
  • Developmental Biology
  • Stem Cell-Based Assay Development
  • Translational Medicine
  • Stem Cell Biology
  • Regenerative Medicine
  • Tissue and Cell Engineering

The programs

CELL & GENE THERAPY WORLD CONGRESS 2023 EUROPE

"Realising the promises of next generation therapies"

Day 1 - Thursday 9th March 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

MANUFACTURING AND PROCESS DEVELOPMENT

  • Focus on more challenging purification requirements of gene therapy and oncolytic viruses
  • Factors that can complicate the downstream processing for viral vectors
  • Tailored purification process design for greater process understanding
  • Methodologies, cell manufacturing platforms and technologies that contributes to defining CAR-T cell
  • Core components of autologous CAR-T cell manufacturing that remains constant
  • Quality requirements & quality control assays enabling the release of clinical CAR-T cell products for infusion
  • Challenges in cell manufacturing
  • Recent advances and benefits from different techniques
  • Potential for different applications wide usage of microfluidic devices.
  • Risk assessment analysis
  • Equipment requirements and facility characteristics
  • Beneficial effect of regulatory T cell transfer on the maintenance of self-tolerance and immune homeostasis in different conditions

CASE STUDY

  • 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

CHARACTERISATION AND VALIDATION

  • Upstream and downstream processes for production of rAAV
  • Analytical methods for quantification of rAAV quality attributes
  • Key challenges in transitioning from traditional to newer methods
  • Methods of detect and classify key contaminants in CGT
  • New advances in technology that play a significant role in ensuring the safety of therapeutics
  • Design model where contamination is minimized before manufacturing begins
  • Increase control and implementation of QbD principles and adoption of new assay methodologies

CASE STUDY 

  • Monitoring and control challenges resulting from the extensive range of cell and gene therapy products
  • Understanding the intricacy and processes required to produce these products
    • Challenges in perspective and implementation of regulatory framework
  • 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

  • Process challenges associated with manufacturing gene therapies and strategies in overcoming them
  • Major components that impact innovation and optimization
  • What can manufacturers do to guarantee a seamless process as the treatment progress form early to commercial stage and through regulatory approval?
  • Future for next generation technologies and process developments

CELL & GENE THERAPY WORLD CONGRESS 2023 EUROPE

"Realising the promises of next generation therapies"

Day 2 - Friday 10th March 2023

KEYNOTE PANEL DISCUSSION

  • 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

REGULATORY, ETHICAL AND QUALITY CONSIDERATIONS

  • 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
  • Opportunities for businesses in EU to influence changes in EU law and policy that could make development and commercialization of transformative new treatment less costly.
  • Policy, regulations, and legislative changes to help overcome barriers to more widespread adaptation of CGTs towards its full potential and industries steady progress
  • Ethical issues involved in current gene therapy research and problems inherent to future gene therapies
  • Moral responsibilities of those involve in the development and use of CGTs
  • How to recognize individual’s right to choose to accept or reject testing or treatment?
  • Possible dilemmas involved in the use of genetic science in ways that go beyond prevention and cure of disease
  • Economic impact associated with advance therapies

PANEL DISCUSSION 

  • Conducting early research to better understand patient and indications
  • Smarter clinical trial study designs that support implementation
  • Relieving barriers in manufacturing and supply chain
  • Accelerated pathways for the investigation and approvals of CGTs
  • Strategies and tactical approaches that companies can undertake to aid in achieving consistent and appropriate reimbursement levels
  • How can CGT developers expand capabilities for broader adaptation?

 

  • 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 
  • 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

PANEL DISCUSSION

  • 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?

Panelist: 

Wenliang  Dong, Chief Operating Officer, ORCA Therapeutics

 

STEM CELL SCIENCE AND REGENERATIVE MEDICINES CONGRESS WORLD CONGRESS 2023 EUROPE

“Revolutionizing the way human diseases are treated”

Day 1 - Thursday 9th March 2023

  • Recent policies that support clinical application of induced pluripotent stem cell-based regenerative therapies
  • How different markets approach the regulation and commercialization of advanced therapies
  • Current data on how product effects clinical outcomes

PRODUCT, PROCESS AND MANUFACTURING DEVELOPMENT

  • Implications and advances in stem cell therapy (T-Cells, NK Cells, DCs, Vaccines, iPSCs & MSCs)
  • Setting global standards for stem cell research and clinical translation
  • Use of chip-based microsystems for genomic and proteomic analysis
  • Developing nanostructured thin films as biomimetic tissue-engineered platform for cancer research
  • Applications of high-throughput cellular microarray platforms in drug discovery, toxicology and stem cell research
  • Generation of human pluripotent stem cell-derived hepatocyte-like cells for drug toxicity screening
  • Using mouse and human pancreatic organoids to infer resistance to targeted therapy
  • Patient-derived organoids as an in vitro model of neuroendocrine tumors
  • Brain-region-specific organoids using mini-bioreactors for modeling ZIKV exposure
  • Regulation of ribosome biogenesis and protein synthesis controls germline stem cell differentiation
  • Global regulatory developments for clinical stem cell research
  • Issues surrounding the authentication and quality of induced pluripotent stem cells

STEM CELL PRODUCT, PROCESS AND MANUFACTURING DEVELOPMENT

  • Flow-induced epigenetic mechanisms of endothelial gene expression
  • Emerging role of nutri-epigenetics in inflammation and cancer
  • Epigenomic annotation of regulatory changes in adult tissue and stem cell based models
  • Advancing biomaterials of human origin for tissue engineering
  • Bio-inspired 3D microenvironments for stem cell reprogramming
  • Implementing oxygen control in chip-based cell and tissue culture systems

Process parameters for the high-scale production alginate-encapsulated stem cells for storage and distribution throughout the cell therapy supply chain

CASE STUDY

  • Computational tools for stem cell biology
  • Developing an automated robotic factory for novel stem cell therapy production
  • Issues of supplying new technologies into highly regulated markets

PANEL DISCUSSION 

  • Innovative, comprehensive mapping and multiscale analysis of registered trials for stem cell-based regenerative medicine
  • Failure mode and effects analysis to reduce risks of errors in the good manufacturing practice production of engineered tissue and cells
  • Regulatory requirements in the good manufacturing practice production of an epithelial cell graft for ocular surface reconstruction

STEM CELL SCIENCE AND REGENERATIVE MEDICINES CONGRESS WORLD CONGRESS 2023 EUROPE

“Revolutionizing the way human diseases are treated”

Day 2 - Friday 10th March 2023

KEYNOTE 

  • Results and lessons from recent successes in clinical development
  • Science, ethics and communication contribute to success of cell-based therapies

CELL MANIPULATION PLATFORM

  • Innovative tools and technology for analysis of single cells and cell-cell interaction
  • Microfluidic particle manipulation using high frequency surface acoustic waves
  • Gene editing and manipulation using CRISPR/Cas9 technology

REGENERATIVE MEDICINE MODELS AND TECHNOLOGIES

  • Evaluation of chick embryo cryoinjury model for the study of embryonic organ development and repair
  • Artificial thymus in recreating microenvironmental cues to direct T Cell differentiation and thymic regeneration
  • Computational modeling of muscle regeneration and adaptation to advance muscle tissue regeneration strategies
  • Contribution of dermal-derived mesenchymal cells during liver repair in two different experimental models
  • Gingival fibroblasts as autologous feeders for induced pluripotent stem cells
  • Direct cardiac cellular reprogramming for cardiac regeneration
  • Bone regeneration using freshly isolated autologous stromal vascular fraction of adipose tissue in combination with calcium phosphate ceramics
  • Tumor-free transplantation of patient-derived induced pluripotent stem cell progeny for customized islet regeneration

Cecilia  Götherström, Research group leader, Associate Professor in Stem Cell Science, Karolinska Institutet

CASE STUDY

  • Contribution of complex systems to entrepreneurship
  • Literature review and proposed guidelines for social entrepreneurship and cross-sector collaborations
  • Clinical development and commercialization of one-time potentially curative therapies

STEM CELL AND TISSUE ENGINEERING APPLICATIONS

  • Mesenchymal stem cells can be recruited to wounded tissue via hepatocyte growth factor‐loaded biomaterials
  • Co-culture-expanded human basal epithelial stem cells for application in tracheal tissue engineering
  • Adipose-derived stem cell differentiation as a basic tool for vascularized adipose tissue engineering
  • Selective differentiation into hematopoietic and cardiac cells from pluripotent stem cells based on the expression of cell surface markers
  • PRC2 component JARID2 for critical maintenance of leukemic stem cells
  • Targeting PTPRK-RSPO3 colon tumours for differentiation and loss of stem-cell function

REGULATORY, ETHICAL AND QUALITY CONSIDERATIONS

  • Regulatory aspects of pre-clinical studies for regenerative medicine
  • Recent policies that support clinical application of induced pluripotent stem cell-based regenerative therapies
  • The future for regulation and reimbursement in bringing regenerative medicines to the clinic
  • Clinical landscape and manufacturing challenges in regenerative medicine
  • Specific GMP legislation for quality requirements in tissue engineering
  • Quality assurance in bio-banking for pre-clinical research
  • Use of organ chips for quality assurance in regenerative medicine

PANEL DISCUSSION 

  • Convergence of regenerative medicine and synthetic biology to develop standardized and validated models of human diseases with clinical relevance
  • Possible trends in product safety and standardization of manufacturing regenerative products
  • How to address ethical pluripotent stem cells in the United Kingdom and Japan

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Testimonials

Thank you for a good experience with the PARADIGM Global Events… very high level, very good Organization, we overpassed our expectatives, we will be pending for another congress related to pharmaceutical and biotech… we learned a lot, and i think that the content of agenda was state of the art, thank you again.
SALVADOR BERRIOS
ERP/ CRM / Product Configurator Manager
The consistently high quality of international faculty was most impressive. It was also obvious that they were carefully chosen not only for their particular expertise but also for their ability to communicate the subject matter to clinical and non-clinical participants alike. As market access consultant for pharma clients of various sizes, I now feel much better prepared to explain the nuances & dynamics involved in bringing their products to market. In addition, I am in a much better position to intelligently advice my clients & help them set realistic expectations
THOMAS MARTIN
Associate Vice President, VCGA - Quintiles
I was impressed with the organization of this event. I attend many conferences per year and find the organizers on site to be detached and not focus on good service to the delegates. This team was even providing weather forecast warning to ensure everyone was aware and made appropriate plans. Well Done.
ADRIANA E. MANZI
PhD, Managing Director, Atheln, Inc

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