- Scientific Topic 1: Molecular Cell Surgery
Isabelle Rivière (Memorial Sloan Kettering Cancer Center, USA)
Nicole Borth (BOKU, Austria)
The last decade has seen a surge in high resolution tools for engineering of the genome and gene expression patterns in mammalian cells that now enable detailed fine-chiselling of their molecular behaviour. These tools include precise genome editing techniques using nucleases such as CRISPR as well as efficient gene knock-in and knock-out methods. Other cell engineering platforms based on targeted modifications of chromatin state and thus epigenetic control of expression as well as new approaches to use non-coding RNAs such as miRNAs or lncRNAs are also emerging. We encourage submission of abstracts on applications that aim to achieve fine-tuned control of cell behaviour and phenotype in order to generate optimized cell lines for recombinant protein production and to engineer cells for cell therapies.
- Genome engineering (including methods i.e.: CRISPs, TALENs, KI/KO, miRNAs, lncRNAs,…);
- Impact of epigenetics on cell behavior;
- Epigenetic control of phenotypes;
- Non viral Delivery (Nanoparticles, Extracellular Vesicles, Electroporation, …)
- Scientific Topic 2: Beyond Evolution
Martin Fussenegger (ETH, Switzerland)
Nick Timmins (Artisan Bio, USA)
The convergence of modern gene editing technologies with data driven systems level understanding of cellular mechanics is enabling a previously unattainable degree of mastery over mammalian cell function. Increasingly sophisticated molecular programs can be introduced into mammalian cells to deliver predictable outcomes. This new era of mammalian synthetic biology is especially relevant to cell therapy, enabling new and improved therapeutic modalities, enhanced safety/reduced risk, and new opportunities to improve manufacturing and reduce cost.
Beyond simple single gene edits or even multiple edits for independent outcomes, the merger of systems and synthetic biology facilitates precision control of gene networks, enabling modularity, and extensibility to global control of cell function. We can now go beyond the limitations of evolution and the corresponding constraints of any given cell type, rationally engineering for predetermined outcomes of great societal and economic benefit. Doing so is, however, far from trivial. The complexity and scope of mammalian gene regulation and interactions, higher order epigenetic elements and protein level interactions, and correspondingly large number of “variables” poses significant challenges to wide-spread utilization of synthetic biology approaches in mammalian cells. Presentations addressing these issues are welcome in this Scientific Topic.
- Synthetic Biology
- Precision Control of Genes and Networks;
- New Cell Functionalities (Smart Cells; Logic Circuits)
- Scientific Topic 3: Challenges and Marvels of Bioprocess Intensification
Chetan Goudar (Amgen, USA)
Paulo Fernandes (Orchard, UK)
Stefanos Grammatikos (UCB, Belgium)
The biopharmaceutical industry is seeing increasing deployment of the principles of Bioprocess Intensification both in process development and clinical/commercial manufacturing of CHO-cell derived products.
Existing fed-batch processes are benefitting from the application of process intensification both in the N-1 and N steps and have realized very significant productivity gains. Additionally, significant advances have been reported in the development of novel integrated continuous biomanufacturing paradigms including clinical-stage applications.
Process intensification principles are broadly applicable and emerging therapeutic modalities such as cell and gene therapy are fertile ground for application to address clear challenges like yield and productivity, scale-up, process cycle time and economics.
In this topic we intend to bring together the state-of-the-art on Bioprocess Intensification including end-to-end elements of semi- continuous and fully continuous operations all the way to Integrated Continuous Bioprocessing across multiple hosts and product modalities. In addition to advances across cell culture unit operations, there will be an emphasis on overcoming challenges and complications downstream of the bioreactors including but not limited to harvest and filtration operations, protein/viral vector purification and product quality impact and implications. Presentation proposals focusing on titer or bioreactor productivity alone without discussion of ensuing process or scale-up considerations and associated yields are discouraged.
Further related aspects in scope of this session are PAT, automation, process control, process characterization strategies and regulatory implications, including considerations for building and obtaining regulatory approval for manufacturing facilities based on process intensification principles.
- Scaling and Intensifying Cell and Gene therapies manufacture
- Process Engineering; Bioprocess tricks to produce; continuous and semi-continuous operations;
- Downstream; Quality issues in the product; Packaging cells; Disposables; Integrating UPS & DSP from harvest to filtration; Cell line stability; Scale up/scale out viral; Platform processes for viral (& vaccines) production; Manufacturing challenges for non-viral products
- Scientific Topic 4: Adaptive manufacturing: engineering quality into your process
Anne Tolstrup (AbtBioConsult, Denmark)
Laura Palomares, (IBT-UNAM, Mexico)
Quality output understanding of cell culture process technologies is critical for process development, scale-up, and for taking a product to the market. Can product quality be predicted from in-process information? Can the process be changed without impacting your product? Or can you fine-tune a desired product quality attribute via process controls? How to do it with minimal effort? These and other questions regarding process and product quality are the subjects of this scientific topic. Presentations regarding advanced analytics, the effect of cellular and process variables on quality, novel approaches to facilitate and accelerate product and process development based on quality are welcome. Quality by design, process monitoring and control, in-line and at-line tools, predictive modelling, statistical process control, and PAT approaches are especially relevant for this topic. Strategies for the characterization and control of complex processes and products of cell technologies in an integral way will be especially valuable.
- Cell process control & analytics
- Quality by design; PAT; Automation; Predictive modeling & control
- Statistical process control (a good QbD strategy)
- Scientific Topic 5: Data, Data, Data: how to get it and how to use it
Damian Marshal (Catapult, UK)
Mike Betenbaugh (Johns Hopkins Univ, USA)
The incorporation of high content analytics as well as the analysis and interpretation of “big data” are now integral components of cell-based research. These tools and their application enable users to extract previously unknown relationships and insights into biological and biotechnological systems and are fueling the development of increasingly sophisticated therapies, the advent of advanced personalized medicines, and more efficient biomanufacturing processes. The ability to generate large amounts of high-quality data combined with machine learning (ML), artificial intelligence (AI), and hybrid modelling/AI approaches is emerging and being applied across all stages of drug discovery and process development. This data, alongside increasing levels of machine connectivity, is enabling the development and implementation of new technologies, which could revolutionize the manner therapies are manufactured and pave the way for industry 4.0.
This topic will explore:
- The latest advances in the application of “omics” technologies for high content cell characterisation and process optimization
- The application of technologies to support data-driven bioprocessing
- The application of machine learning, AI, and multivariate analysis among others in therapeutic discovery and process development
- New approaches and applications for data modelling including those based on mechanistic, machine learning, and hybrid approaches in order to increase product and process understanding
- Future approaches for product manufacture using industry 4.0 principles
- Data integrity and quality
- Making it, connecting it and using it; Digitally enabled manufacturing
- Role for omics in process development; Industry 4.0; advanced process control
- Machine learning; Artificial Intelligence, MVDA (multivariate data analysis)
- Data modeling; end users perspective, contextualize, …
- Scientific Topic 6: Bio breakthroughs
Luis Almeida (CNC Universidade de Coimbra, Portugal
Laura Cervera (Univ Autònoma Barcelona, Spain)
Transformative emerging modalities for treating diseases are rapidly becoming an important area in the biopharmaceutical industry. The use of T-Cells, CAR-Ts, TILs, and oncolytic viruses to treat cancer, viral vectors, stem cells and vesicles for gene and cell therapy is becoming more and more a reality.
This topic is designed for bold projects. We encourage submission of abstracts describing innovative work on disruptive engineering tools and technologies for:
- Novel virus modalities for gene therapy
- New therapies based on nanoparticles, exosomes and other non-viral therapeutic delivery systems of genes or medicines
- Accelerate and de-risk the development of new vaccines
- New bioprocesses to generate rapid response vaccines: counteract possible pandemics.
- Advanced Cell Models in Pre-clinical Research and Molecular Mechanisms of Disease Understanding: 2D, 3D, bioprinting and beyond
- Next-generation human in vitro systems and assays (e.g. microfluidics)
- Disruptive therapies; Novel viruses modalities, Vaccines, technologies;
- Don t put all eggs in one basket: Rapid response vaccines
- Bioprinting; Alternative test methods; Advanced models in Pre-clinical research