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Informa
07:40 - 08:40 60 mins
Main agenda
Registration and Morning Coffee
08:45 - 08:50 5 mins
Main agenda
Chairperson's Opening Remarks
  • Miguel Forte - CEO, Zelluna Immunotherapy, Oslo Cancer Cluster Incubator, Norway
more
08:45 - 09:05 20 mins
Main agenda
Challenges in the Manufacture, Release and Distribution of Cell Therapy Products
  • Prentice Curry - Vice President, Quality and Compliance, Kite Pharma, USA
more
09:05 - 09:25 20 mins
Main agenda
Delivering automated platforms for CAR-T cell manufacturing: A case study
  • Rodney Rietze - Senior Research Investigator, Cell and Gene Therapies, Novartis, USA
more
  • Automation is the key to delivering a safe, robust and cost-effective therapy
  • Delivering such platforms is a highly strategic process, guided by a deep understanding of your process and product.
  • The rationale and supporting data for the automation of the CTL019 manufacturing process/analytics is presented here.
09:25 - 09:45 20 mins
Main agenda
Patient Centricity when considering post approval needs for a Cell and Gene Therapy
  • Rohit Batta - Head of Global Medical Affairs Gene Therapy, GSK, UK
more
  • Considerations when setting up a post-approval observational registry for a C>
  • Patient centeredness and how can digital help?
09:45 - 10:05 20 mins
Main agenda
Designing technologies to meet the manufacturing needs of cell therapies
  • Dolores Baksh - Innovation Leader, Cell Therapy Technologies, GE Healthcare, USA
more

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.

10:05 - 10:20 15 mins
Main agenda
Process Automation Solutions for Autologous Cell Therapies
  • Nina Bauer - Associate Director, Commercial Development, Cell Therapy, Lonza, Switzerland
more
10:20 - 10:45 25 mins
Main agenda
Discussion panel: Strategies for the successful commercialisation of CART and gene therapies
  • Prentice Curry - Vice President, Quality and Compliance, Kite Pharma, USA
  • Rohit Batta - Head of Global Medical Affairs Gene Therapy, GSK, UK
  • Rodney Rietze - Senior Research Investigator, Cell and Gene Therapies, Novartis, USA
  • Bruce Levine - Barbara and Edward Netter Professor in Cancer Gene Therapy,, University of Pennsylvania Perelman School of Medicine
  • Ohad Karnieli - Chair, Process and Product Development committee, (ISCT), and VP, Technology and Manufacturing, Pluristem, Israel
more
  • Strategies for dealing with the regulators and regulatory submissions 
  • What does commercialisation mean from a facility point of view?
  • Process validation strategies
  • How do you plan and strategize to get a commercial process up and running?
  • Re-imbursement strategies (working with payers etc.)
10:45 - 11:15 30 mins
Main agenda
Morning Coffee and Networking
11:13 - 11:15 2 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Chairperson's Opening Remarks
  • Lior Raviv - Development Director, Pluristem Therapeutics Inc., Israel
more
11:13 - 11:15 2 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Chairperson's Opening Remarks
  • Prentice Curry - Vice President, Quality and Compliance, Kite Pharma, USA
more
11:13 - 11:15 2 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Chairperson's Opening Remarks
  • Rob Allen - UK Programme and Alliance Director, Asterias Biotherapeutics, UK
more
11:13 - 11:15 2 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Chairperson's Opening Remarks
  • Christopher Bravery - Director, Consulting on Advanced Biologicals Ltd., UK
more
11:15 - 11:50 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Enabling commercial scale-out of T-cell manufacturing
  • Jim Faulkner - SVP and Head Of Manufacturing, Autolus, UK
more
  • What are the key challenges in scaling an autologous process for commercial?
  • The role of closed systems in scalability
  • Understanding and controlling Key Quality Attributes
11:15 - 11:50 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Adherence cell lines for viral vector production – choosing the best cells
  • Bhargavi Kondragunta - Associate Director, Upstream Process Development, REGENXBIO Inc., USA
more
  • What are the best adherence cells lines for viral vector production?
  • How can you scale these up to a commercial scale?
  • Suspension culture and new technologies
11:15 - 11:50 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Predictive biomarkers and preclinical models of immune-gene therapy efficacy
  • Jan Joseph Melenhorst - Director, Product Development & Correlative Sciences laboratories (PDCS), Adjunct Associate Professor, Center for Cellular Immunotherapies, University of Pennsylvania, USA
more
  • Correlative studies show that expansion and persistence are key attributes of a successful CAR-redirected T cell therapy of cancer
  • We have recapitulated in a preclinical model of leukemia
  • Using RNA-seq and deep immunophenotyping of the infusion product we identified pathways differentially expressed between cells for responding and non-responding patients
  • We extended those findings to the pre-manufacturing T cells wherein we could identify responding patients before CART cells were manufactured
11:15 - 11:50 35 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Regulatory update on expectations surrounding potency assays
  • Ruti Goldberg - Quality Control Validation Project Manager, Pluristem, Israel
more
  • Case study: from development of a potency assay to validation of the potency assay
  • Experiences and limitations
  • Regulatory challenges surrounding validation
11:50 - 12:25 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Screening strategies for optimal process development
  • Yen Choo - CEO and Executive Chairman, Plasticell and Progenitor Labs, UK
more
  • Process optimisation for autologous therapies

  • Reducing cost of goods

  • Reducing process timelines

  • Increasing cell potency

11:50 - 12:25 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
The solutions for production of biologicals
  • Hanna Lesch - Research & Development Director, FinVector Vision Therapies
more
  • Commercial scale manufacturing of adenoviral and lentiviral vectors in iCELLis500 fixed-bed bioreactor
  • Disposable stirred tank and ATF perfusion for suspension process scale up
  • Supporting process development with modern analytical tools such as MiniTEM
  • What are the funding possibilities for all this
11:50 - 12:25 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Novel targets and T-cell receptors for adoptive cell therapy
  • Steffen Walter - CSO, Immatics, USA
more
  • A major constraint for the broad and safe application of adoptive cell therapy (ACT) is the limited number of validated tumor targets and T-cell receptors (TCRs), especially for solid tumors.
  • Immatics’ proprietary antigen discovery platform XPRESIDENT® uses ultra-high sensitive and quantitative mass spectrometry, transcriptomics, immunology profiling and bioinformatics for high-throughput identification of novel antigens with high tumor selectivity. This approach can identify and rank shared antigens, neoantigens as well as novel classes of antigens such as cryptic epitopes or RNA-edited epitopes.
  • Immatics’ high-throughput TCR generation platform enables the identification of 100+ natural TCR candidates per target. Immatics is using the XPRESIDENT® normal tissue database to enable faster and better screening of TCR candidates with decreased risk for off-target cross-reactivity and on-target/off-tumor toxicities.
  • ACTolog® and ACTengine® are Immatics’ most advanced ACT programs, conducted in close collaboration with MD Anderson Cancer Center, which aim to deliver safer, best-in-class immunotherapies in multiple solid tumor indications.
11:50 - 12:25 35 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Potency Assay Discussion Panel
  • Jan Joseph Melenhorst - Director, Product Development & Correlative Sciences laboratories (PDCS), Adjunct Associate Professor, Center for Cellular Immunotherapies, University of Pennsylvania, USA
  • Anthony Lodge - Regulatory Affairs Manager, ATMP, GSK, UK
more

Informa is looking 5 speakers to join this panel looking at the below topics :

  • Similarities and differences between ex vivo and in vivo
  • Different types : Cytokines, cell based assays, profilin, cytometry
  • What type of profiling should be developed to ensure the cells have the right mode of action
  • How do you validate your assays  
12:25 - 12:55 30 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Single device-based manufacturing of CAR T cell products in a closed system
  • Ian Johnston - Senior Project Manager, Research and Development, Miltenyi Biotec GmbH, Germany
more
12:25 - 12:45 20 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Improvements in post-freeze stability of clinical grade hMSC to solve logistical challenges for cell therapies
  • Clémentine Mirabel - Research Scientist, Albumedix A/S
more

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.

12:25 - 12:55 30 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
ATMP’s – Science led development and analytical challenges
  • Paul Byrne - Lead Scientist, Cell and Gene Therapy – Biopharmaceutical CMC Solutions, Covance, UK
more

The presentation will cover the current challenges for cell and gene therapy molecules.

  • Comparability of manufacturing changes
  • Developing a Potency assay for a Stem Cell Therapy
  • Biodistributions. Design and analytical challenges
    • In life and QPCR/RT-QPCR
  • Developing and validating CMC assays


12:25 - 12:55 30 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Navigating the cell therapy supply chain – when exceptions are the norm
  • Amy Ronneberg - President, Be The Match BioTherapies, USA
more

We are entering a new age of medicine with the introduction of the first approved cell and gene therapies to market. Commercial strategies for supply chain remain critical to successfully delivering these therapies to patients. Through the collective experience of managing more than 80,000 stem cell transplants over 30 years, the National Marrow Donor Program®/Be The Match® has learned that exceptions are the norm. We will share techniques for mitigating some of the risks associated with autologous and allogeneic cell therapy supply chain management including the use of dedicated patient and therapy management staff supported by our technology platform, MatchSource.

12:45 - 13:05 20 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Manufacturing Solutions for Production of Human Induced Pluripotent Stem Cells
  • Antoine Heron - Head of Market & Portfolio Development, Stem Cell Bioprocessing Group, MilliporeSigma, Germany
more

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.

12:55 - 13:15 20 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Process characterization and validation for CAR-T: autologous cell therapy challenges and approaches
  • Ryan Shorr - Bioengineer 2, Celgene, USA
more
  • Develop analytical capabilities and product understanding, leveraging them towards meaningful product attributes
  • Track and trend clinical data set to evaluate process performance and identify potential correlations
  • Characterize process and seek control strategy to account for variability in patient material
12:55 - 14:10 75 mins
Main agenda
Lunch and Networking
14:10 - 14:45 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Closed systems for CAR T-Cell production
  • Lothar Germeroth - SVP Juno Therapeutics, Inc.,, Managing Director Juno Therapeutics GmbH, Germany
more
14:10 - 14:45 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Assessing the gaps in DSP for cell therapy products
  • Francesca Bellintani - Development Supervisor, MolMed, Italy
more
  • Where do the gaps currently lie with  regards to DSP?
  • Bioreactors – after completing this how do you deal with DSP?
  • Case study of successful DSP strategies in place
14:10 - 14:45 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Please move to another stream
14:45 - 15:20 35 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
AST-VAC2 – An allogeneic dendritic cell immunotherapy
  • Rob Allen - UK Programme and Alliance Director, Asterias Biotherapeutics, UK
more
14:45 - 15:20 35 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Automation Expert Panel
  • Lior Raviv - Development Director, Pluristem Therapeutics Inc., Israel
  • Rodney Rietze - Senior Research Investigator, Cell and Gene Therapies, Novartis, USA
  • Valerie Steenwinckel - Industrialization Director, Celyad, Belgium
  • Ohad Karnieli - Chair, Process and Product Development committee, (ISCT), and VP, Technology and Manufacturing, Pluristem, Israel
more

Informa is looking for 5 speakers to join our expert led discussion panel to talk on the below topics:

  • Case studies/feedback on closed systems in general
  • Is there a need to have closed systems in clean rooms?
  • Process automated sampling
  • What can we learn from automation for MAbs?
  • How do you make thinks connectable between different devices?
  •  Automation linked to cost of goods
14:45 - 15:20 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Avoiding the Ultracentrifuge: The Challenges of Developing Robust, Scalable AAV Platform
  • Joseph Earley - Senior Process Development Scientist, Allergan, UK
more
14:45 - 15:20 35 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Anticipating clinical success and innovating processes
  • Miguel Forte - CEO, Zelluna Immunotherapy, Oslo Cancer Cluster Incubator, Norway
more
  • How long and expensive is it to build your processes in the clinic?
  • At What phase do you start thinking in advance to a product that will treat patients?
  • Innovations to your process as a result of clinical success
15:20 - 15:50 30 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
A presentation from TrakCel
  • Matthew Lakelin - CSO , Trakcel
more
15:20 - 15:35 15 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
High-resolution monolithic columns as efficient tool of viral particles purification used for gene therapy
  • Mojca Tajnik Sbaizero - Project Manager, BIA Separations, Slovenia
more

HPLC is one of the key methods to analyse the concentration of molecules and substantially help to study their structures. Although being vastly used for small molecules, for most of biomolecules the HPLC has not been much applied. Viral based structures can be, from the point of their chromatographic properties, regarded as very big protein structure, and as such analysed. From this point of view the HPLC could easily be applied for the said tasks, but the chromatographic resin structure with diffusive end pores was too small to allow most of viral structures to enter, which was limiting factor regarding the use of the HPLC in this field.

Introduction of monolithic resins with open channels and no diffusive pores enabled the HPLC to be extensively applied for viral structures separations. Entrapment of the viral structures and carry-over has been dumped and time of the analysis accelerated. Surprisingly also the resolution power has been greatly improved when compared to traditional chromatographic resins. As a result, the HPLC columns based on CIM (Convective Interaction Media) monolithic chromatographic supports are able to further separate and analyse subpopulations of the viral capsids with extreme purity and yield, determine the ratio of full and empty capsids including their fast separation and enrichment/concentration of the desired viral populations.

This feature is opening new era in the study of viral structures and understanding of their properties, efficiency and safety. Moreover, their use is being highly accepted in the production and analytics of viral biopharmaceuticals.

15:20 - 15:50 30 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Rapid detection of bacteria in ATMP prior treatment – Validation of a qPCR-based test
  • Kai Nesemann - Junior Product Manager Microbiology, Sartorius Lab Instruments GmbH & Co KG, Germany
more

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>.

15:20 - 15:40 20 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Vector manufacturing production using disposable fixed-bed bioreactors iCELLis® system
  • Margherita Neri - Upstream Process Development Manager, MolMed, Italy
more

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.

15:35 - 15:55 20 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Challenges of cell therapy automation: A perspective on disposables
  • Benjamin Le Quéré - Business Manager, Bioprocess Solutions, Saint-Gobain, USA
more
15:35 - 15:55 20 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
New evidence for pathogen-reduced human platelet lysate
  • Steve Harris - HPL Business Lead, Cook Regentec, USA
more
15:50 - 16:20 30 mins
Main agenda
Afternoon Coffee Break
16:20 - 17:30 70 mins
Stream 1: Cell, Gene and Immunotherapy Manufacturing, Process Automation and Cold Chain
Dual Dialogue: Cost of goods calculations for cell therapy products
  • Reinout Hesselink - Cell Therapy Consultant, Exmoor Pharma Concepts Ltd., The Netherlands
  • Benoit Champluvier - Chief Technology and Manufacturing Officer, Bone Therapeutics, Belgium
more

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

  • Cell therapy solutions for bone defects and spinal fusion procedures do address very large number of patients.
  • Future manufacturing process shall deliver products with a sustainable cost/benefit
  • COGS analysis early in the projects is key to define the process development strategy
  • Advantages and limitations of early COGS assessment will be discussed

Benoit Champluvier, Chief Technology and Manufacturing Officer, Bone Therapeutics, Belgium


16:20 - 16:55 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Appropriate in process controls for process characterisation
  • Christine Le Bec - Head of Analytical Development, Genethon, France
more
  • Quantification of Vector genome by qPCR and ddPCR
  • Quantification of AAV vector (full and empty particles) by using orthogonal methods
  • Quality control: assays for process impurities
16:20 - 17:30 70 mins
Stream 4: Clinical Development for Cell, Gene and Immunotherapies
Commercialising the advanced therapy supply chain
  • Chair Robert Jones - Global Director, Advanced Therapies, Fisher BioServices, UK
  • Luděk Sojka - Chief Operating Officer, Sotio a.s., Czech Republic
  • Simon Ellison - Cell & Gene Therapy Service Director, World Courier, an AmerisourceBergen company, UK
more

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

16:20 - 17:30 70 mins
Stream 3: Preclinical Strategies for Cell, Gene and Immunotherapies
Dual Dialogue: Ensuring enough supply as magnitude in terms of patient and doses increases
  • James Miskin - Chief Technical Officer, Oxford Biomedica, UK
  • Janneke Meulenberg - COO, CEO, Arthrogen, Vicinivax, The Netherlands
more

Talk 1: Scalable supply of lentiviral vector – clinical and commercial

  • Overview of lentiviral vector technology
  • Vector development and production strategies
  • Meeting clinical and commercial demand


Talk 2: Production scale up of ART-I02, an AAV gene therapy for rheumatoid arthritis

  • Production of clinical material for Phase I
  • Pro’s and con’s of next generation AAV production platforms for scale up
  •  Strategy for production of ART-I02 for Phase
16:55 - 17:30 35 mins
Stream 2: Cell, Gene and Immunotherapy USP, DSP, PAT and Analytics
Manufacturing of personalized tissue-engineered transplants – How can we monitor the process?
  • Raimund Strehl - CTO, NovaHep, Sweden
more
  • The challenge of producing tissues instead of cells
  • Monitoring the tissue engineering process
  • Generic processes – The future of tissue engineering?
17:40 - 18:25 45 mins
Main agenda
Closing Plenary Session - Using artificial intelligence to make everyday products better, one beer at a time
  • Rob McInerney - Founder and CEO, Intelligent Layer, Co-Founder, IntelligentX Brewing Company, UK
more

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”.

18:25 - 20:25 120 mins
Main agenda
Networking Drinks and Evening Social Event