Dutch ATMP Summit
ATMP Innovations
Dutch ATMP Summit Manufacturing
2023
2022
Recap of the day
The second edition of the Dutch ATMP Summit focused on innovations in ATMPs. The program was extended to a full day, and industry representatives presented their learnings on improving the production of cell therapy products in particular.
Presentations and discussions on emerging topics in the ATMP field were prepared, including: Standardization for Chain of Custody, Effective Microbial Control Strategies, and Alternative Approaches for Established Cell Therapy Products.
On average the participants of the 2023 edition rated the Dutch ATMP Summit with a 8,0
- “A deep dive into a very interesting topic “
- “It covered comprehensive topics”
- “Interactive even full of opportunities to network”
- “Good combination of networking and scientific content”
- “Inspiring talks and broad community sharing opinions/ideas/backgrounds “
- “Diverse audience and thought-provoking talks in a variety of ATMP-relevant subjects”
Some of the participants of the 2023 edition
Speakers
Speakers
Topics of the summit
Translational Research on Oncolytic Viruses: a Consortium Approach
The use of oncolytic viruses forms an appealing approach for cancer treatment. On the one hand the viruses replicate in tumor cells, leading to their intra-tumoral amplification. On the other hand the viral infection will activate virus-directed immune responses, and may trigger immune responses directed against tumor cells and tumor antigens. To date, a wide variety of oncolytic viruses is being developed for use in cancer treatment. Relatively few pre-clinical and clinical studies evaluated different oncolytic viruses in comparative studies. I will summarize the steps and considerations essential in the development and characterization of oncolytic viruses. In addition, I will outline our multidisciplinary academic consortium that collaborates on the development of oncolytic viruses.
New developments in the use of iPSC-based cell platforms in drug discovery and cell therapy
Induced pluripotent stem cells (iPSCs) can be expanded indefinitely and can be differentiated into every cell type. As such, iPSC technology opened a new era of treating human diseases. Recent developments in iPSC technology will be presented. Ranging from human in vitro models in drug development (safety testing and discovery screening) to allogeneic cell therapy.
Next generation ATMPs
Cell and Gene Therapies offer amazing potential for curative therapies. Melissa will discuss the current and next generation of therapies in this rapidly evolving field.
Genetic characterization of ATMPs: What and why the regulators want you to check
Advanced therapy medicinal products (ATMPs) hold great promise in offering effective and targeted treatments for otherwise poorly treated diseases and injuries. While the field of cell and gene therapies only emerged over the past decade, at this time more than 1,200 active clinical trials are registered globally and several treatments are already commercially available. Genetic engineering of primary immune cells, including (viral) transgene integration or gene editing using tools including CRISPR/Cas9 and TALENs, is a critical step in generation of autologous or allogeneic cell therapies. Sadly, the past years have shown us that undesired editing outcomes including oncogenic translocations can severely affect product safety and efficacy. As such, regulatory agencies including the FDA and EMA are developing and continuously updating regulatory guidelines for manufacturers to comply with. This seminar will cover the current requirements for genetic characterization of ATMPs as stipulated by the FDA/EMA, and subsequently discuss which screening strategies are available to assess these characteristics. The final part of the presentation will cover which proprietary services Cergentis, a Dutch CRO providing genetic QC services, offers for genetic characterizations of pharmaceutical cell lines and ATMPs in support of regulatory (IND/BLA) filings.
Performance of Aseptic Process Simulation studies in ATMP manufacturing
ATMPs are generally produced using aseptic process steps to ensure a sterile medicinal product.
At regular intervals and at minimum 2x per year aseptic processes must be evaluated for the effectiveness of process controls safeguarding aseptic conditions and for its capability to yield sterile products (EU-GMP, annex-1; FDA Guide for Industry; Sterile drug products- produced by aseptic processing; USP <1116>). Such aseptic process evaluation is performed by means of simulating the process using a suitable culture medium instead of product.
Next to the verification of the process capability such Media Fill is often also used to (re-)qualify the personnel involved in performance of aseptic processes. These personnel members must be kept in a trained and qualified state in order to be allowed to perform aseptic processes.
Administration of out of specification ATMP products
In the exceptional circumstances the administration of a cell/tissue-based ATMP that does not comply with the specifications may be considered to avoid an immediate significant hazard to the patient. The supply of an OOS batch can only occur after evaluation of the risk of the product and considering the specific condition of the patient. This presentation will discuss the various aspects before an OOS ATMP product can be administered.
Microbial control strategy for ATMPs
Assurance of absence of microbial contamination is essential for the safety of ATMPs including cells based ATMPs. A microbial control strategy should be established that goes beyond testing of samples for microbial quality attributes sterility, bioburden, endotoxin and mycoplasma. The strategy should use risk assessment tools to get microbial contamination under control. Risk assessments covers microbial contamination risks of raw and starting materials, facilities and equipment, personnel, process and methods. This presentation will provide you with an outline for establishing a microbial control strategy for ATMPs.
