Day 1
2nd CELL & GENE THERAPY WORLD CONGRESS 2024 EUROPE
"Driving Innovation, Addressing Significant Unmet Medical Needs, and Ultimately Improving Patient Outcomes"
Day 1 - Thursday 3rd October 2024
COMMERCIALISATION & REGULATORY LANDSCAPE
- Latest trends and valuable insights to drive innovation and remain competitive
- Cutting-edge technologies revolutionizing treatment paradigms
- Aligning with international regulatory standards in order to support the broadest possible patient access.
- Ensuring affordability and accessibility of these therapies
- Key drivers for attracting investment
Panellist:
Jeff Liter, CEO, OX2 Therapeutics
- Introduction
- Demonstrating the UK’s continuous strength in the cell and gene therapy sector.
Challenges facing companies in this space
Collaborative work and initiatives to guarantee continued growth of the sector
Lessons learned and strategies to unlock the full potential of this developing field
- There are a number of changes and policy initiatives currently being developed which will start going into effect in 2025 and have a significant impact on the future of ATMPs in the EU.
- At the EU level, we have the EU HTA Regulation, discussions on the General Pharmaceutical Legislation, the Biotechnology Strategy, and a renewed focus on the competitiveness agenda.
- At national level, different Countries are reviewing their P&R and early access programmes.
- For developers, it is important to take a step back and look at the landscape holistically when considering pipelines and forecast the direction of travel in the EU for the next 5 years, and beyond.
Dr. Alexander Natz, Secretary General, EUCOPE
- Understanding the key logistics requirements in operationalizing Cell and gene therapy trials
- Comprehensive strategies to logistics management
- Addressing a whole range of intricacies across all types of CAGT assets
- Significance of stakeholder partnerships to optimize supply chain and enhance patient experience
- Ways to navigate and succeed in this promising yet competitive sector
Strategic portfolio assessments and management critical to success
Importance of robust clinical pipeline to attract interest from investors
Considering the complexity of the manufacturing process, supply chain requirements of each, along
with the required regulatory pathway to market.
- Improving clinical trials, strengthening regulatory authorities
- Foster use of health data for innovation
- Promoting innovative research projects
- Reimbursement for gene therapies and ATMPs
DEVELOPMENT & MANUFACTURING
- Last clinical results in NSCLC cancer patients in association with Pembrolizumab
Eric HALIOUA, President & Chief Executive Officer, PDC*line Pharma
- Muscular dystrophy as paradigm for orphan drug development
- Most promising gene therapies strategies for the treatment of Duchenne muscular dystrophy
- Future perspectives; orphan drugs in the pipeline
Dr Alberto Malerba, Lecturer in Gene Therapy, Gene Medicine Laboratory for Rare Diseases, Royal Holloway, University of London
- Current landscape of CAR-T cells manufacturing
- Challenges limiting production capacity
- Latest technical developments in GMP production platform design
- Challenges associated with translation of new scientific developments into clinical products for patients
Dr Theres Oakes, Head of Product Characterisation, Achilles Therapeutics
- Utilising NHSBT’s 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 NHSBT’s Clinical Biotechnology Centre
Dr Lilian Hook, Director Cell, Apheresis & Gene Therapies, NHS Blood and Transplant
- To Follow
Adam Glen, Co-founder, Unicorn Bio
- State of continuous process implementation and some ongoing challenges.
- Technological advancements that helps drive batch productivity and reduce the costs for patients
- Improving the design and efficiency of facilities to focus on quality, getting into manufacturing quicker
- Lowering the overall cost of goods and the amount of raw materials used
- Lessons learned and best practices
Moderator: Jeff Liter, CEO, OX2 Therapeutics
Panellist:
Alex Blyth, Founder & CEO, Lift Biosciences