PDA Advanced Therapy Medicinal Products Conference 2025

Topics we are looking for:

• 1. Cell and Gene Products
  • Gene Modified Stem Cells
  • Gene Edited Products
  • Gene Modified Lymphocytes e.g. CAR T-Cells
  • Tumor-Infiltrating Lymphocytes (TILS)
  • Natural Killer Cells
  • Viral Vectors (Adeno-Associated Virus, Lentiviral)
  • mRNA Products
• 2. Regulatory and Policy Updates
  • EU Guideline on GMP for ATMPs
  • PIC/S Revision of GMP Guide Annex 2A (Manufacturing of Advanced Therapy Medicinal Products)
  • PIC/S Revision of GMP Guide Annex 2B (Biological Medicinal Substances and Products for Human Use)
  • Correlation Between Part IV EU Guideline on GMP for ATMPs and Annex 1: 2022
  • Harmonization Efforts in GMO Regulation
  • Regulatory Requirements for Process Validation
  • ICH Q5A
  • Harmonization Between EU and National Regulations
  • Comparability Guidance
  • European Health Technology Assessment Regulation
  • Mutual Recognition Agreement
• 3. Raw and Starting Materials and Equipment
  • Cells, Sera, Buffers, Plasmids, Complex Raw Materials, Proteins/Enzymes
  • Characterization of Starting Materials and Cell Lines
  • Securing Raw Material Supply Throughout Full Life Cycle
  • Equipment for Cell Harvest
  • Application Systems/Delivery Devices
  • Digitalization of Manufacturing/Testing Equipment
  • Single-Use Equipment
  • Viral Vectors (Adeno-Associated Virus, Lentiviral, Adherent and Suspension)
  • Transition from R&D Grade to High-Quality/GMP Grade Material and Equipment
  • Induced Pluripotent Stem Cells
  • Scaffolds
  • Regulatory Requirements for Manufacturing of Final Product Packaging
  • Media and Reagents
• 4. Manufacturing
  • Manufacturing Process and Unit Operations
  • Managing Deviations/CAPAs/Change Controls
  • Chemistry, Manufacturing and Controls (CMC) Aspects (Including Phase-Appropriate GMPs)
  • Facility Design for Shared Manufacturing
  • Challenges with Multi-Product Manufacturing Environment
  • Centralized vs Decentralized Manufacturing Models
  • Non-Viral Modification (Transfection, Transduction)
  • Handling Cell Clumps/Aggregates
  • Acceptable Quality Level (AQL)/Visual Inspection/Visible Particles
  • mRNA Platform
  • Platform Technologies
  • Closed Systems Versus Open Systems
  • Final Formulation Design
  • Viral Vector Production
  • Aseptic Processing
  • Process Control Strategy
  • Contamination Control Strategy/Sterility Assurance
  • Validation Strategies
  • Automation and Robotics
  • Virus Safety
  • GMP, Environment, and Occupational Safety
  • Cost of Goods and Actions for Cost Reduction
  • Suspension and Adherent Manufacturing for Viral Vectors
  • CRISPR and Gene Editing/Transposons
  • Managing Manufacturing Failures
  • Short Hairpin RNA Technology
  • Exosomes
  • Lipid Nanoparticles (LNPs)
  • Autologous, Allogeneic, or Xenogeneic Pathways
  • Inhibiting Cell Apoptosis
  • Differentiating Tissues into Expected Cells/Tissue
  • Operator Qualification and Training
• 5. Analytical Development and Testing
  • Development and Qualification of Bioassays
  • Challenges in Specification Setting (e.g. Potency, Comparability)
  • Specificity, Precision, and Robustness of Quality Control Tests
  • Characterization of Viral Vectors
  • Characterization of ATMP Material
  • Contamination-Related Quality Tests (e.g. Mycoplasma, Bacteria, Fungi)
  • Next-Generation Sequencing
  • Validation of Analytical Methods
  • Viral Safety Aspects
  • Challenges in Demonstrating Product Comparability Throughout Development
  • Cost and Speed of Quality Control Testing
  • Conditional Release
  • Automation/Robotics in Quality Control
  • Platform Methods
  • Paperless Systems
• 6. Supply Chain
  • Labelling
  • Chain of Custody/Chain of Identity
  • Primary Containers and Closure Systems
  • Cryo Storage
  • Freezing/Thawing Equipment
  • Bio-Preservation
  • (Ultra-) Cold Chain Logistics
  • Shipping Validation
  • Hold Studies
  • Logistical Challenges and Solutions
  • Turnaround Time
  • End-to-End Supply Chain Setup
  • CDMO Management
  • Manufacturing Localization Strategies (Colocalized/Decentralized)
  • Inventory Policies/Strategies
• 7. Clinical Aspects
  • Clinical Development
  • Design of Clinical Trials and Role of Pivotal Studies
  • Immuno-Profiling
  • Novel Therapies for Various Cancers
  • Measuring Treatment Response
  • Correlation of Data on Manufacturing and Outcome
  • Importance of Potency Assays for Clinical Outcome
  • Drug Delivery Devices and Solutions for ATMPs
  • Point of Care Standards for Drug Administration
  • Companion Diagnostics
  • Standardization of Apheresis Collection
  • Mastering the Inflammatory Effect
• 8. From Lab to Market
  • Role of Academia in ATMP Development
  • Similarities and Differences of Pre-Clinical, Clinical and Commercial Products
  • Yield and Scalability Strategies
  • Intellectual Property Considerations
  • CMC Strategies
  • Technology Transfer
  • Market Requirements and Framework for Commercialization of ATMPs
  • Innovative Payment Models
  • Funding as Financing Model
  • Challenges in Financing, Pricing, and Reimbursement
  • Combining One-Time Treatment with Value of Life-Long Benefit
  • Payment Upon Treatment Success
  • Models of Collaboration Between Hospitals, Small or Medium-Sized Companies, and Big Pharma Companies
  • Hospital Exemption Versus Centralized Approval
  • Decentralized Manufacturing
  • Risk-Based Strategies in Qualification
• 9. Sustainability
  • Ecological Footprint
  • Energy Consumption Reduction
  • Environmental Protection
  • Carbon Footprint
  • Biosafety During and After Manufacturing
  • Adeno-Associated Viral Vector Containment
  • Waste Management
  • Alternatives to Liquid Nitrogen