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The realities of manufacturing and commercialising CAR-T cells and gene therapies
As patient specific, autologous cell therapies are in advanced clinical trial phases and are embarking on commercialization, there continues to be a need to have a robust, closed, automated and scalable manufacturing solution that can accommodate the processing of many patient samples in this growing patient population. What will be required is a means to process patient material in a fashion which maximizes the efficiency of the processing workflow for time and cost while meeting quality and regulatory requirements. This approach should retain the fundamental principles of preventing contamination, patient sample mixing, loss of identity or other events which interfere with the physical properties and integrity of the patient sample and final product. This talk will present details of what a manufacturing infrastructure needs to look like to take the above aspects into consideration and methodologies of how to achieve this infrastructure.
Late Morning Session
Process optimisation for autologous therapies
Reducing cost of goods
Reducing process timelines
Increasing cell potency
Informa is looking 5 speakers to join this panel looking at the below topics :
In this study multipotent Bone Marrow-derived Mesenchymal Stromal Cells (BM-MSC) were manufactured in compliance with regulatory and quality requirements for clinical use to be used as medicinal product. The use of living cells as an active ingredient poses great logistical challenges, as rapid loss of cell viability in the final formulation within hours from product release is typical. Cryopreservation allows the banking of off-the-shelf products, but yet post-thawing stability remains a challenge. Short shelf-life rises concerns in terms of logistics, as formulation, quality controls, product release and shipping need to be performed whithin hours, meanwhile, patients, surgeons and the surgery room must also be ready for clinical administration.
We will present data on a comparability study, in which the performance of GMP-grade recombinant human albumin and human serum albumin as stabiliser of critical quality attributes (CQA) was evaluated using clinical grade BM-MSC, aiming at solving logistical challenges by developing therapy formulations that can support longer shelf life.
The presentation will cover the current challenges for cell and gene therapy molecules.
The therapeutic potential of human induced pluripotent stem cells (hiPSCs) is explored in a large array of indications, ranging from acute myocardial infarction to diabetes. The inefficiencies in current differentiation protocols combined with the large numbers of cells recommended for clinical scale tissue engineering warrant the use of systems that can generate large batches of hiPSCs in a controlled manner. Prior studies have shown the ability to culture hiPSCs in stirred suspension culture, however the culture vessels have mostly been limited to small scale spinner flasks with no monitoring and control of pH and dissolved oxygen (DO). In this study, we will present data on a two-stage suspension culture of hiPSCs that achieved a 125-fold expansion after 14 days in culture. After expansion in the bioreactor, the hiPSCs were characterized by immunocytochemistry, flow cytometry, and differentiation into the embryonic germ layers. The results demonstrate the potential of hiPSC production in controlled stirred suspension systems that can support the production of large batches of cells for research and clinical applications.
Early Afternoon Session
Informa is looking for 5 speakers to join our expert led discussion panel to talk on the below topics:
Contaminated ATMPs constitute life-endangered risks for treated patients that are most often immunocompromised. A reliable microbial release test is indispensable prior to treatment to ensure patient safety. Current international pharmacopeia stipulate a duration of 14 days for sterility testing. Typically, ATMPs have a shelf-life of less than 48 hours. Hence, ATMPs cannot be released by current traditional methods prior to treatment. The risk of administering potentially contaminated ATMPs urges industry and authorities to find solutions for rapid releases. For the first time, a rapid testing system based on qPCR is under validation to detect total bacterial contamination within only 3 hours prior to treatment compliant to international guidelines.
This study shows the validation approach according to EP 5.1.6., EP 2.6.27 and USP<1223>.
Lentiviral and retroviral vectors are used in cell and gene therapy studies. In the context of advanced clinical phase a big challenge is the scale up of the vector production to an adequate large-scale platform. In order to address this issue development studies on Pall iCELLis®nano fixed-bed disposable bioreactor was performed to obtain a scalable and robust process to produce high titer viral vectors.
Late Afternoon Session
Talk 1: Title TBC
Reinout Hesselink, Cell Therapy Consultant, Process Development Manufacturing, Exmoor Pharma Concepts Ltd., The Netherlands
Talk 2: Early COGS evaluation as a tool for process design and planning: the case of allogeneic cell therapy for bone defects
Benoit Champluvier, Chief Technology and Manufacturing Officer, Bone Therapeutics, Belgium
Advanced Therapies are now becoming commercial reality. However this means that they need to be delivered to patients at commercial scale. With increased patient numbers the challenges of coordinating the inter-related supply chain matrix grow.
This session will look at the generic challenges facing the industry and use two case studies to illustrate the issues and lessons learned to highlight the supply chains that advanced therapy companies should be looking to establish
Talk 1: Scalable supply of lentiviral vector – clinical and commercial
Talk 2: Production scale up of ART-I02, an AAV gene therapy for rheumatoid arthritis
How can artificial intelligence be used to create a future without advertising and in which everyday products continually adapt? Join IntelligentX co-founder Dr. Rob McInerney as he shares how his company created a self-evolving beer brand that uses AI to optimise beer recipes using customer feedback, which Popular Science voted “the third greatest software innovation of 2016”.