Conference: Cell and Gene Therapies/ ATMPs - Quality and Safety
25/26 November 2025
Objectives
This meeting is aimed at manufacturers and developers of cells, tissues, cell- and tissue-based products or ATMPs and deals with microbiological and analytical quality requirements, suitable methods and test systems and their implementation and validation. Representatives from authorities and colleagues from small-scale and industrial manufacturing and consulting will explain the current regulatory requirements and share their knowledge.
Background
Modern regenerative medicine systems such as cells and tissues or ATMPs (gene therapeutics, somatic cell-based products and tissue-based products) represent an innovative group of medicinal products that is becoming increasingly important. With the entry into force of several regulatory directives, e.g. the European Directive EC 1394/2007 for ATMPs, such products have been classified as medicinal products and as such must comply with EU requirements for medicinal products. Although the biopharmaceutical industry has significantly intensified its activities in this area, many of these products are developed and manufactured at universities, hospitals and in small and medium-sized enterprises. These university or medical roots lead to special challenges for the respective institutions as well as for the regulatory authorities in meeting compliance requirements for quality, safety and GMP aspects and approval. The frequently given manufacturing conditions also contribute to this, e.g. the open manipulation of cells and tissues necessary for obtaining such products on a medical-surgical level, or the short shelf life of the obtained end product. And potentially there are always conflicts when it comes to the relevance of different guidelines, e.g. when an Annex 1, or an Annex 2 or a WHO Guideline does not harmonize with the ATMP Guideline.
Target Audience
- Representatives of the regulatory and authorisation authorities
- Specialists for biopharmaceutical manufacturing processes
- QA/QC personnel in the biopharmaceutical environment
- Project managers and outsourcing personnel
- Analytical Experts
Moderation
Dr Sabine Hauck, Chair of the ECA ATMP Board
Dr Ulrike Herbrand, Charles River Laboratories
Tuesday, 25 November 2025
KEYNOTE on 25 November 2025: Artificial
Intelligence in Pharmaceutical Industries
Dr Marcel Franke, Senior Scientist Predictive Formulation,
Process Solutions/Upstream & Process Materials R&D
Merck Life Science
Update on ATMPs in the Ph. Eur.
Dr Solène Le Maux, EDQM
- Adoption of new general chapter 5.32 Cell-based preparations for human use
- Revision of general chapter 5.34. Additional information on gene therapy medicinal products for human use to include mRNA-based GTMPs
- Elaboration of a new general chapter 5.47 Gene-edited cells for human use
Understanding of Analytical Critical Quality Attributes (CQAs) for Viral Vector Characterization in Gene Therapy
Dr Maribel Navarro, Kymos Group
- Quality attributes definition
- Capsid characterization
- Transgene characterization
- Entire vector characterization
- Product and process-related impurities determination
Strategy for Potency Determination of Gene Therapy Products
Dr Ulrike Herbrand, Charles River Laboratories
- Benchmarks current approaches to potency strategy
- Provides guidance on potency strategy considering phase, MoA, QC-friendliness and other variables based on industry consensus
- Aims to reduce time to market and reduce the risk of delays due to not meeting quality/regulatory expectations
High-Throughput Single-Cell Potency Assays for Cell Therapy Development Using Droplet Microfluidics
Dr Stephanie van Loo, Livedrop
- Principle of bioassays performed in microfluidic droplets
- Testing and cell sorting in a droplet microfluidic benchtop device
- Case studies on secretome-based cell characterization
Cell and Gene Therapy CQA Analysis using Mass Spectrometry
Dr Daniel Waldera-Lupa, Protagene
- Virus protein ratio determination
- Identification of primary sequence and modifications
- Host cell proteins profiling
- Assessment of the critical HCP
Optimization of Analytical Methods for Engineered Cell Therapy Products: Lessons Learne
Dr Daniela Rozkova, SCTbio
- Digital PCR (dPCR) method development and application
- Infectious titer quantification strategies
Validation of VCN Determination by dPCR on a Retroviral CAR-T Cell Product in Compliance with ICH Q14 and Q2(R2)
Dr Katy Haussmann, Charite & Dr Daniel Lindemeier, Labor LS
- Aspects of release testing in CAR-T cell manufacturing
- Introduction into the absolute DNA quantification with a Digital PCR System
- GMP parameters for a quantitative method in accordance with ICH Q14 and Q2(R2) guidelines
- Stepwise development and validation of an assay for VCN estimation of retroviral transduced CAR-T-cells according to the implementation life cycle
Wednesday, 26 November 2025
KEYNOTE on 26 November 2025: From Bog to
Bedside: Lessons from the First Dedicated Phage
Therapy Center in North America
Prof Dr Steffanie Strathdee, University of California San
Diego School of Medicine/Co-Director at the Center for
Innovative Phage Applications and Therapeutics
>> Find out more
Developing Control Strategies for Off-the-Shelf Allogenic Cell Therapy Products
Dr Steven Braem, 3D-PharmXchange
- Control strategy of starting material
- Control strategy of in process controls and final process controls in a continuous manufacturing
- Challenges in identity testing
- Challenges in fill-finish processes
Considerations of Viral Safety Strategies for Cell Therapies – A Suggested Approach for What to Include in Viral Safety Packages for Cell Therapies
Dr Kerstin Brack, Charles River Laboratories
- Cell therapy (CT) products - high viral contamination risk
- Viral safety strategy – limitations in viral clearance and focus on prevention and detection
- Discussion of the critical viral safety aspects in the manufacturing process of CT products
- Presentation of a risk assessment template and structure to assess and mitigate viral contamination risks for CT products
- CAR-Ts as an example
Avoiding Common Sterility Testing Compliance Gaps and Regulatory Observations During ATMP Sterility Testing
Marsha Steed, Steed MicroBio
- USP <72>, <73>, <1071> Overview for RMM for Short Shelf Life Products
- The importance of using good aseptic behaviors in sterility testing in an isolator
- Glove use in sterility test isolators
- Environmental Monitoring in isolator Sterility Testing
- Validation of Loading Patterns
- Regulatory Observations in Sterility Testing
Enhancing Microbiological Quality Control with Ready-to-Use Microorganisms
Dr Megha Bajaj, bioMérieux
- Role of microbial strains in pharma QC
- Common microorganisms found in pharma labs
- Regulatory landscape around use of microbial strains in QC testing
- Challenges and considerations when utilizing pharmacopeia strains and local isolates
- Use of standardized inoculum to ease pharmaceutical practices
How to Build a Digitalized End-to end Process for Environmental Monitoring for ATMPs Release
Johannes Oberdörfer, Rapid Micro Biosystems & Willem Dullaers, Lonza
- How to create a unique digitalized and automated E2E EM process
- How streamlined testing enables the delivery of critical medicines to cell & gene therapy patients faster
- The increased cost savings with enhanced compliance robustness
The New Guideline on ATMPs and Further Regulatory Update on ATMPs
Dr Sabine Hauck, dequra pharma consult
- Pharm. Eur. general monograph 3186 for mRNA/LNPs as Gene Therapy Medicinal Products
- Comparison of the Pharm. Eur. texts for mRNA/LNPs as vaccines and as GTMP
- Further sources helping to define your tests methods and acceptance criteria