2023 ARCHIVES
Tuesday, 14 March
Registration and Morning Coffee (Garden Room)07:00
Chairperson's Remarks
Sanjin Zvonic, PhD, Senior Vice President, Business Development & Practice Expert, Dark Horse Consulting Group
The Challenges of Developing Bespoke Technologies to Close and Automate Complex Gene Therapy Manufacturing Processes
Precision cell therapies represent an opportunity for transformational change in solid tumour cancer treatment. Whilst novel modalities are supported by increasingly innovative manufacturing platforms, challenges surrounding process closure and reproducibility remain at the industry’s core. A dearth of fully closed systems to support tumour infiltrating lymphocyte (TIL) product manufacture has catalysed Achilles Therapeutics to develop a bespoke technology to address these shortcomings, with the challenges and learnings discussed herein.
Manufacturing Enhancements with a Focus on Robustness, Quality, and Speed of TCR T Product Release
IMA203 & IMA203CD8 are Immatics’ TCR T product candidate(s) using a PRAME-specific TCR with or without a CD8 co-receptor. Manufacturing improvements implemented enhance key features of the cell product. Continuous enhancements are in progress in preparation of later-stage clinical trials.
Jonas M. la Cour, MSc, PhD, Global Head of Product Management, Sales, ChemoMetec
No SI standard exists for cell count, so how do we streamline the quality and scalability of cell-based processes? The growth of allogeneic cell therapies and bioprocessing is shaping the development of cell counters. The NucleoCounter and its microfluidic single-use cassettes have become standard equipment in C>, but higher throughput and adaptation to Industry 4.0 are also required. ChemoMetec presents their new instruments for semi-automated and fully automated cell counting processes.
Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing (Verdi/Vivaldi)10:00
A Consortium Investigation for the Implementation of PATs for Immunotherapy Manufacturing
There are longstanding challenges in the commercialisation of ATMPs that can be addressed by the implementation of process analytical technologies (PATs). Cell and Gene Therapy Catapult (CGTC) has led a consortium project with 23 collaborators to investigate the application of PAT and advanced analytics for a T-Cell immunotherapy process. We will describe results from the online, in-line and at-line PATs employed in the study discussing the different applications and implementations.
Integrated Manufacturing for Autologous and Allogenic Cell Therapeutics
Gene and cell therapies are gaining more and more interest in personalized precision medicine. From a manufacturing perspective, cell therapies are fairly demanding, requiring chemical gene synthesis, plasmid DNA fermentation and purification, along with viral vector design and production via mammalian cell cultures, isolation of human T cells from the patient or allogenic T cells, propagation of the T cells and transfection with the viral vector carrying the gene of interest for a ligand on the T cell surface targeting the T cell to the tumor cell to express their cytotoxic effect. Or coupling monoclonal antibodies to the surface of the T cell to target the tumor cell. An end-to-end supplier for all required manufacturing technologies is advisable and will facilitate the process chain.
Advanced Manufacturing Processes
Toby Proctor, EngD, Head of Manufacturing Science and Technology, Achilles Therapeutics
Ali Mohamed, PhD, Vice President, CMC, Immatics US, Inc.
John Churchwell, PhD, Associate Lead Scientist, Cell & Gene Therapy Catapult
Rolf Werner, PhD, Professor, Industrial Biotechnology, University of Tuebingen
Gerd Steffens, PhD, Manager, CliniMACS, Clinical Marketing, Miltenyi Biotec B.V. & Co. KG
Whether manual operation or a series of chained instruments, the lack of integration compromises speed, reproducibility, and quality of CGT therapy manufacturing. The CliniMACS Prodigy Platform automates and integrates the entire process workflow in a single device. With the example of a CAR T cell process, we will talk about the CliniMACS Prodigy with its flexibility and standardization for a resilient and scalable CGT production.
Philipp Nold, PhD, Dr. biol. hom, Infield Application and Stem Cell Specialist, Eppendorf Bioprocess
Human pluripotent stem cells (hPSCs) are a unique source for the production of functional human cell types, fueling advanced regenerative therapies. Bioreactors are a suitable platform to optimize cell culturing and to promote process development. In this talk, we show how to achieve cell densities of 35 X 106 cells/mL by precisely monitoring and controlling critical process parameters with Eppendorf bioprocess equipment. Pluripotency, differentiation potential, and karyotype stability were maintained.
Networking Lunch12:45
Decentralized Manufacturing and POCare Processing Platform
A roadmap to enabling cell and gene therapies (CGTs) in an affordable and accessible format at the point of care. The Orgenesis Point-of-Care (POCare) Platform enables a globally harmonized pathway for CGTs to reach large numbers of patients at the point of care at reduced costs through a data centric approach supporting scalable, and decentralized production. OMPULs (Orgenesis Mobile Production Units and Labs) are autonomous production units allowing rapid global deployment.
Scaling to Commercial Scale: Cord Blood Stem Cell Derived NK Cells (oNKord®)
Ongoing NK cell clinical trials suggest a therapeutic role for NK cells in various types of cancer. Impaired/dysfunctional immune cells in cancer patients highlight the need for an external supply of highly potent NK cells to repopulate the immune system. A scalable NK cell product, which is off the shelf and can be cryopreserved is the key to meet the unmet needs and for a successful commercialisation”. We will provide an overview of Glycostem’s allogeneic NK cell product oNKord® progress on the road to commercialisation.
Jens Matuszczyk, Manager of Process Technology Management, Cell Culture Technology Marketing, Sartorius
A single dose of MSC therapy requires billions of cells, but traditional workflows that use 2D culture methods are inefficient at commercial scale. Scaling up microcarrier-based 3D suspension processes are significantly more sustainable, efficient and cost-effective. This presentation will explore the successful transition and scale-up from a 2D culture process to a 3D suspension-based platform to enable more sustainable production.
Refreshment Break in the Exhibit Hall with Poster Viewing (Verdi/Vivaldi)15:20
End-to-End Automation Platform for the Manufacture of Complex Organoid-Based Implants
Organoid-based implant manufacturing may enable the production of complex 3D ATMP implants able to revolutionise defect regeneration and restore organ function. Engineering such complex ATMPs requires manufacturing technologies that can provide a robust backbone for phenotypic changes but also for increase in size. In this presentation a novel integrated and fully automated biomanufacturing platform will be showcased allowing the automated transition from single cells to organoids and finally to organoid-based 3D implants.
Biological Raw Material Sourcing for an Early Stage ATMP and Considerations for Late Stage Clinical Development
Amniotics has developed a MSC cell therapy for treatment of inflammatory lung diseases, currently tested in a Phase I clinical trial. The process uses multiple raw materials of biological origin, e.g., as source material (MSCs from amniotic fluid), sorting antibody, culture medium, detachment enzyme. Quality requirements, suitability for manufacturing of Phase I clinical material and supply issues are discussed here, as well as regulatory aspects and considerations for further development.
A Translational Services Business Model on the Grounds of Public Cell and Tissue Banking
Cell and tissue banks are establishments adhering to strict quality management standards and regulations, and we advocate its prominent role to enhance the value of donated Substances of Human Origin in innovative Advanced Therapy Medicinal Products. To achieve this, we have created a platform for collaboration with academic and industrial developers, supporting a range of activities from bioprocess design and Good Manufacturing Practice validation, to early phase I/II clinical trials.
Development of an End-to-End Closed iPSC Expansion Process Using a Cell Specific Perfusion Regime for Process Intensification
This work details a repeated batch process for reproducible expansion of pluripotent iPSCs in 3D with a closed seed train achieved using a hollow fibre bioreactor. This process was optimised using an acoustic filtration system to adapt the platform to perfusion. Perfusion processes demonstrate high yields of pluripotent iPSCs, with greater control of aggregate size. Ability to perform an integrated passage with equivalent growth profile allows for further intensification potential.
Welcome Reception in the Exhibit Hall with Poster Viewing (Verdi/Vivaldi)17:40
Close of Day18:45
Wednesday, 15 March
Registration and Morning Coffee (Garden Room)08:00
Michael Delahaye, Director, Team Leader Cell Therapy Bioprocessing, AstraZeneca
Harnessing the Capabilities of Big Pharma to Drive Cell Therapy
Newly created Biopharmaceuticals R&D Cell Therapy Department holds the ambition of developing and delivering cell therapies to address cardiovascular, renal, metabolic, respiratory, immunological, and rare disease. With focus on ex vivo manipulation of cells, the department continues to build on its diverse skillset, working cross functionally both internally and externally with collaborative partners to deliver therapeutic candidates coupled with innovative manufacturing building blocks to achieve clinical realisation.
Development of an Allogenic Mesothelin CAR T Cell Therapy Production Process with a Representative Scale-down Model
CAR T cell therapy success in solid tumors has been limited due to the lack of tumor-specific antigens, to tumor heterogeneity, and to the immuno-inhibitory nature of the tumor microenvironment. To address these challenges, we engineered CAR T cells that use the tumor-specific mesothelin antigen as a discriminatory target and have enhanced therapeutic properties provided by multiple attributes. Its manufacturing method has been designed using a representative small-scale model that led to a new large-scale workflow.
Strategies for Cryopreservation and Banking of iPS Cells and Their Derivatives
Cryopreservation of iPS cells in large numbers or of functional, differentiated cells is also crucial for later success in therapy or other applications. Therefore, to meet the increasing demand in downstream applications, we have established robust, scalable cryopreservation protocols for iPS cells and several derivatives. This includes the use of large volume cryobags, ready-to-use formats and cryopreservation of tissue engineered products.
Xiao Pan, Director of Platform R&D Dept., Platform R&D Dept., GenScript ProBio
The plasmid is a small, extrachromosomal DNA molecule that is widely used in cell and gene therapy. It can serve as the raw material for viral vector production, the template of mRNA manufacturing, the delivery vehicle to transfer the gene of interest, or the final drug product in the case of DNA vaccine. Therefore, a robust plasmid platform is the foundation for the fast developing cell and gene therapy application field.
Coffee Break in the Exhibit Hall with Poster Viewing (Verdi/Vivaldi)10:30
Chairperson's Opening Remarks
Margit Holzer, PhD, Owner, Ulysse Consult
Overcoming CMC and Supply Chain Challenges for mRNA Technologies
Thanks to the rapid development of mRNA vaccines for COVID-19, the industry now has the momentum and resources to overcome many of the early CMC challenges and realize its enormous potential. This presentation will discuss the strategies in place to overcome CMC and supply chain challenges for mRNA technologies already and future innovations primed to take it to the next level.
Affinity Proteins for Biotechnological and Medical Purposes
Affinity proteins are crucial for life, for building structures, performing reactions, and for signaling purposes. In life sciences and medicine, affinity proteins are used to generate knowledge, but also for diagnostic and therapeutic purposes. This talk will cover how antibodies and small affinity molecules can be used to map the human proteome, develop diagnostic tools for in vivo visualization as well as efficiently purify therapeutics based on antibodies.
Transition to Sessions12:20
Judith Koliwer, PhD, Principal Consultant, Consulting & Training, Körber Pharma
Using our experience of over thirty CGT implementations, we will provide a best practice phased approach to establish a digital ecosystem, where different software solutions are used efficiently and feasibly in every stage of the product lifecycle including R&D, clinical trials and commercial production. We furthermore describe an approach to using all available sources of digital data for automatic data analysis using a centralized data hub to maximize data efficiency.
Networking Lunch13:00
Close of Gene Therapy Manufacturing Conference14:00
2025 Programs