ATMPs - Hurdles & Achievements in Quality and Safety
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.
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
This conference is aimed at all persons who
- are involved in the extraction and manufacture of Cells, Tissues and ATMPs
- responsible persons from 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
Tuesday, 19 March 2024
Quality and Regulatory Strategies for the successfull Registration of an ATMP
Yogesh K. Davé, Cypress Quality Consultancy
- Understanding of Cell and Gene Therapy Products
- Autologous vs. Allogenic
- Is CAR-T the same as stem cell transplant?
- Pros and Cons of each therapy
Challenges in the Bench-to-Bedside Translation of Gene and Cell Therapeutics (GCT)
Prof Dr Sven Stegemann, DWI – Leibniz-Institut für Interaktive Materialien e.V.
- GCTs continue to emerge into personalized first line treatments especially in oncology and immunology
- Major challenges in clinical and commercial manufacturing remain to be solved
- Multidisciplinary collaboration will be crucial to assure the bench-to-bedside translation of innovative GCTs
Distributed Manufacturing for T-Cell Therapies
Dr Gülbengü Yüksel, Tigen
- Difficulties with centralized production
- Development of distributed manufacturing for clinical and commercial supply
- Regulatory hurdles with distributed manufacturing
Design Considerations for allogeneic Cell Therapies
Erik Steffensen, Spot-on Pharma Consulting
- How does a typical allogeneic manufacturing process look?
- What is critical to control during manufacturing of allogenic cell therapies?
- Considerations regarding upscaling and process transfer
Challenges in allogeneic CAR-T Manufacturing using Viral Vectors and LNPs
Dr Juliane Heilig, CMT Cellex Manufacturing Transports and Logistics
- Comparison of autologous & allogeneic concept
- Viral vector transduction & LNP knock out rates
- Challenges in manufacturing and product characterization
- Storage of off the shelf products
Contamination Control Strategy (CCS) for ATMPs
Marsha Steed, Resilience
- Developing a CCS for ATMP manufacturing products & processes
- Microbial contamination risks and challenges for cell therapy products
- Human intervention risk relation to environmental monitoring program design
- Design of Aseptic Processing Simulation (APS) for cell therapy products
Wednesday, 20 March 2024
Viral Clearance ATMPs – What if the Product is a Virus?
Sandra Meier, Charles River Laboratories
- Challenges for viral clearance strategies during downstream manufacturing
- What are the possible problems and limitations?
- Potential virus safety strategies
Effective Cell Culture Operations by accurate, non-invasive Determination of the critical Process Parameter pH in Roche`s Drug Substance Network
Christian Klinger, Roche
- Opportunity statement: Limitations of current industry standard
- Proposed technical solution
- Manufacturability and Implementation in commercial manufacturing
- Results and value proposition
Digital PCR for In-Process Control and Lot Release Testing of Gene Therapy Applications
Dr. Nicole Paland, Minerva Biolabs
- Determination of vector copy number (VCN) in CAR T-cells for cancer therapy by duplex dPCR
- Standard for accurate calculation of the VCN
- Determination of the titer of adeno-associated viral (AAV) vectors for In-process control and lot release testing
- Parallel detection of residual DNA by duplex dPCR
Definition of CQA – What and When – and is PAT an Option?
Dr Sabine Hauck, Leukocare
Dr Ulrike Herbrand, Charles River Laboratories
Chairs of ECA’s ATMP Interest Group
- How to define CQAs for ATMPs?
- Best time to validate the assays
- The challenge of Bioassay development
Biopharma Use Cases applying Process Analytical Technology (PAT)
Dr Veronika Nindl, VTU Engineering
- Implementation of biomass sensors for an accurate in-line analyses and an on time process control
- Establishment of DLS as an in-line impurity check to increase the clearance of hcDNA and HCPs prior protein A chromatography and thereby enhance purification
- Total protein determination of an inhomogeneous precipitation flow through for and automated protein concentration adjustment