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Flemming Ornskov, M.D., M.P.H.
A Vision for Rare Disease: How We Can Champion Underserved Patients
James Collins, Ph.D.
Synthetic Biology: Biomedical Applications Come of Age
Massachusetts Institute of Technology
Commercializing CAR-T Cell Treatments
Following 30 second introductions on their platform, indication, stage of developments etc., a panel of speakers from a range of T-Cell based immunotherapies manufactures will be quizzed about their products, commercialization strategies, fears, concerns, experiences and visions for the future.
Potency Assays – Ensuring the Right Mode of Action and Characteristics
Potency assays should be quantitative, and in most cases the methods employed do quantify an analyte, e.g. (increasing) cell number, dead cells (cytotoxicity), cytokine release etc. However, when setting a specification for potency the questions become;
Commercialization Strategy Case Studies
Cell therapy is now entering an era where it has proven that it can succeed. However, there are several opportunities for improvement to enable this success. This talk would highlight some of the challenges currently encountered across functions during scale-up and commercialization of cell therapy programs and present them as opportunities for improvement to enable better success of future programs.
With the advancement of the CAR-T and the emerging gene editing platforms, autologous cell therapies are on the frontier of commercialization. These complex therapies require careful planning early on in the lifecycle in order to avoid pitfalls towards commercial scale. This presentation will discuss these potential pitfalls and strategies to avoid them.
Converting an academic or research proof of concept (POC) study into a clinical grade cell therapy is a complex multi-step process. Successful transition from demonstrating POC to first in human clinical trials involves identifying and obtaining financial and technical support, mapping your process, and obtaining guidance for manufacturing, pre-clinical, and clinical studies.
Automation, Closed Systems and Big Data Processing
Early stage cell therapy production processes utilize open and manual manipulations from the cell isolation stage and up to final product filling. Open manipulations are operator depended and can increase the risk of contamination causing large gaps in the ability to develop a robust and cost effective product. This presentation will describe how adoption of closed system can change the “art” of growing cells into an industrial and reproducible process
Dendritic cells play an important role in emerging cell-based cancer immunotherapies. However, their production from autologous monocytes is mostly manual and is low yielding and operator dependent. This presentation will describe a new alternative for automated dendritic cell production. The MicroDEN system employs a perfusion based approach that closely mimics the manual process but provides higher yield and consistency.
In the world of bioprocessing of cell and gene therapies, numerous challenges exist. Choosing the optimal data processing and analysis technologies, and ensuring scientific leadership in this process, are both key to success. This presentation will share data strategies that not only enable leaders to take immediate action that can impact their current pipeline, but provide flexibility for the future.
Cell-based immunotherapy is widely captivated due to its clinical potential. Generation of sufficient, desired cells is an essential for the success of any cell-based immunotherapy, which requires an effective ex vivo process with consistency. Case studies of how suitable chemically-defined, animal-component-free culture media for major immune cells were developed to foster a consistent bioproduction process will be shared.
Semma Therapeutics was founded to develop transformative therapies for patients with Type 1 diabetes. Recent work led to the discovery of a method to generate functional, insulin-producing beta cells in the laboratory. These cells develop in islet-like clusters grown from stem cells. Data for a scalable process using PBS SUBs for expansion &directed differention of pluripotent stem cells will be presented.
Automating the manufacturing process of a cell-based therapy is not a one-time event. Rather, it is a step-by-step, highly strategic process that is guided by a deep understanding of critical process parameters and the critical quality attributes of the resulting drug product. As understanding (and confidence in efficacy) evolves through positive clinical testing, so does confidence in receiving a positive return on investment. This presentation will describe the rationale and supporting data which guided the strategic automation of the CTL019 manufacturing process and associated analytics.
Luncheon Presentation (space is limited) or Lunch on your own
For more information, contact: Jennifer Wickett (Companies A-L): +1.857.504.6694 • Jennifer.Wickett@knect365.comKristen Schott (Companies M-Z): +1.857.504.6685 • Kristen.Schott@knect365.com
Potency Assays – Ensuring the Right Mode of Action and Characteristics
Logistics and Supply Chain Management
Allogenic cell therapies do not require the just-in-time supply chain design typically needed for autologous products. This allows for substantial flexibility in supply chain design, primarily because inventories can be built in anticipation of future demand. It is therefore critical to identify what options are available and what key trade-offs exist between allogeneic cell therapy supply chain designs.
The presentation will highlight key considerations and strategies for successful design, management and execution of the supply chain related to autologous or patient specific products. Various elements of the supply chain and associated requirements will be discussed for the full spectrum of the supply chain phases. Key strategies for risk management, reduction and for addressing regulatory requisites will be reviewed.
Developments and Progress in Upstream and Downstream Processing
In the first case study, the impact that different gassing methods have on DO and how hypoxic growth conditions affect MSC function are examined. The second case study demonstrates the application of Zwietering’s equation for suspension of solids in stirred tanks. Identifying optimal control strategies for microcarrier-based bioreactor expansion of adherent cells is paramount for the development of a robust cell therapy manufacturing platform.
Leukapheresis products often have a high platelet content which poses challenges for upstream processing steps. New data will be presented for efficiently removing platelets from non-mobilized leukapheresis products,while achieving high lymphocyte recovery and formulating the cells in the appropriate volume for immunomagnetic selection.
Supply Chain Challenges when Anticipating Transfer from the Clinic to Commercial Scale
This presentation will discuss the application of innovative technologies designed to expand global access to biotherapeutics. These integrated technologies will assist biotherapeutic discovery through the application of molecular design tools to improve candidate molecule developability, improve process design through intensification efforts, and reduce fixed costs associated with manufacturing facility construction and operation through the design of facilities with flexible, low cost features.
Although each rare disease affects a relatively small number of patients, together they are one of the largest under-served patient populations in the world. To make sustained progress for rare disease patients, the biopharma industry must continue to innovate and embrace new technologies to drive advances that result in faster diagnosis, better access and more effective treatments.
Synthetic biology is bringing together engineers, physicists and biologists to model, design and construct biological circuits out of proteins, genes and other bits of DNA, and to use these circuits to rewire and reprogram organisms. These re-engineered organisms are going to change our lives in the coming years, leading to cheaper drugs, rapid diagnostic tests, and synthetic probiotics to treat infections and a range of complex diseases. In this talk, we highlight recent efforts to create synthetic gene networks and programmable cells, and discuss a variety of synthetic biology applications in biotechnology and biomedicine.