ATMPs – Hurdles and Achievements in Quality and Safety
8./9. April 2025, Wiesbaden/Germany
Objectives
This conference track is aimed at all those who develop and manufacture cells, tissues, cell- and tissue-based products and ATMPs. The conference will address manufacturing challenges, e.g. GMP regulations, but also quality control issues, appropriate ways to maintain, assure and control the expected quality. Experienced speakers from the field of ATMP will explain the current requirements and report on their experiences during inspections and the implementation in the company.
Registration
Background
Modern systems of regenerative medicines, such as cells and tissues or ATMPs (gene therapeutics, somatic cell-based products and tissue-based products) represent an innovative group of drugs that is becoming increasingly important. With the entry into force several regulatory guidelines e.g. of the European Directive EC 1394/2007 for ATMPs, such products were classified as medicinal products and must therefore comply as such with the EU requirements for medicinal products. Although the biopharmaceutical industry has considerably intensified its activities in this field, 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 fulfilling the compliance requirements for quality, safety and GMP aspects and approval. This is also forced by frequently given manufacturing conditions, e.g. the open manipulation of cells and tissues, which are necessary for obtaining such products on a medical/surgical level or by the short shelf life of the obtained final product.
Challenges for small batch manufacturing, rapid testing and analysis and storage are only some of the challenges for such short shelf life products in terms of:
- Comparability with Compendial Methods
- Sensitivity and Robustness
- Suitability Testing and Validation
- Variability
Target Audience
This conference is aimed at all persons who
- are involved in the extraction and manufacture of Cells, Tissues and ATMPs
- are responsible for quality assurance and control of Cells, Tissues and ATMPs
- are responsible for microbiological or analytical testing
- perform inspections or audits of ATMPs facilities
- deal with the authorisation
Moderators
Dr Ulrike Herbrand, Charles River Laboratories
Dr Sabine Hauck, Chair of ECA ATMP Interest Group
Speakers
Detailed Programme
Tuesday, 8 April 2025
09.00 - 17.30 h
Navigating the EMA Process: Key Insights from Filing for a TEMP
Dr Katja Aschermann, Astator
- Sharing Lessons and Insights
- CMC aspects
- Submission and Communication with the Authorities
Challenges and Special Requirements for GMP Inspections of ATMPs
Alexander Kammerlocher, Regierungspräsidium Tübingen (local competent authority)
- Overview of legal requirements for ATMPs
- Special requirements for GMP of ATMPs
- Challenges during GMP inspections based on specific examples
- Expectations of GMP inspectorates
Quality Assurance of mRNA Vaccines for Human Use: The Role of the European Pharmacopoeia
Prof Dr Gerrit Borchard, University in Geneva & member of the EDQM expert group
- Lipid based Carrier Systems, “the lesser-known sister”
- Approach of the EDQM
- Contents and Future Work
Challenges on the way to becoming a Contract Manufacturer
Dr Carolin Klemm, DKMS Stem Cell
- Challenges with ATMPs
- Transformation of DKMS Stem Cell Bank into a CDMO
- First CDMO pilot project: Hurdles and Challenges
HIGHLIGHT
Industrial Scale in Vitro Expression of Bacteriophages and other Proteins
Matthias Steiger, Invitris
Tomaz Kasunic, Jafral
- Introduction to cell-free expression - synthetic biology
- Advantages of cell-free expression - a game-changer for difficult to express proteins
- Case study: Tech Transfer and upscale of a cell-free expression process for bacteriophages
The Challenge of GMP Manufacturing of innovative Exosome-based Therapies
Sandrine Mores, ExoBiologics
- Advantages of exosomes-based therapies compared to CGTs
- Challenge of the regulatory pathway
- Groundbeaking Case Study: The view of the first company authorized by EMA for clinical trials with MSC-derived exosomes
Wednesday, 9 April 2025
09.00 - 18.00 h
From personalized Medicine to flexible Machine Solutions
Vilma Methner, Optima
- Same products but different categories for cell and gene therapies worldwide
- Individual production from manual handling up to automation
- A CAR-T cell production as an example in a fully automated production line
Visible and Subvisible Particle Control for Cell Therapy From Development to Commercialisation
Dr Roman Mathaes, Clear Solutions Laboratories
- Major Challenges for Cell Therapy Manufacturing Particle control
- Limitations of analytical methods
- Solution approaches
- Recent FDA warning letters
Process Validation Sterile Drug Products: Strategy, Execution and maintaining the validated State
Dr Anne Orillo, Novartis
- Strategy according to Site Validation Master Plan Stein Steriles
- Execution – set up validation runs, known challenges and audit learnings
- Maintaining the validated state, fit for purpose
- Filing and regulatory feedback
Vector Safety Assessment in Cell and Gene Therapy by NGS/TGS Sequencing
Dr Richard Gabriel, ProtaGene
- Biodistribution and Persistence of gene therapy vectors
- Vector sequencing - Identity, Integrity, Impurity
- Insertional Mutagenesis and Clonality assessment
- Evaluating vector-induced carcinogenesis
Viral Clearance ATMPs – What if the Product is a Virus?
Sandra Zucchet, CRL
- Challenges for viral clearance strategies during downstream manufacturing
- What are the possible problems and limitations?
- Potential virus safety strategies
Gloveless aseptic Fillers for small Batches and Cell & Gene Therapy Sectors: a novel Approach utilizing modular Design and magnetic Levitation Conveyors
Giacomo Guidi, IMA Life
- Increasing demand for small batch production and the need for flexible and safe systems
- Beyond robotic technology and introduction of a solution
- Modular and flexible system design with Focus on CGT
Programme last upated: 21 March 2025