Day 1

2nd CELL & GENE THERAPY WORLD CONGRESS 2024 EUROPE

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

Dilip Patel, VP, Head of Global Market Access, Autolus

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.

To Follow

Adam Glen, Co-founder, Unicorn Bio

13:00 - NETWORKING LUNCH

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

Dr Lilian Hook, Director Cell, Apheresis & Gene Therapies, NHS Blood and Transplant

In contradistinction to other forms of therapy RNA activation activates gene expression via transcription to physiological levels
Transferrin RNA aptamer can deliver various RNA oligonucleotides to the central part of the brain.

Dr Nagy Habib, Professor of Surgery, Imperial College London

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