bioprocessing of advanced cellular therapies congress

For more information please contact Tony at [email protected] more information please contact Tony at [email protected]

Bioprocessing of Advanced Cellular Therapies Congress2 - 3 June 2016, London, UK

The quest for cures as opposed to mitigation of diseases and the huge potential for improving quality of life that cellular and gene therapeutics offer, are the main reasons behind the growth of this industry.

But for the industry to actually take-off and sustain itself over the years, it needs to be cost-effective, robust and safe. Maintaining the critical quality attributes (CQAs) and paramount biological functions whilst processing at varying scales and locations in the world is far from trivial.

To overcome the economic, technical, and regulatory challenges for successful adoption of cell and gene therapies, The Bioprocessing of Advanced Cellular Therapies Congress, organized by MnM Conferences taking place on the 2-3 June 2016 in London (UK), strives to facilitate links between experts in the field to encourage collaborations and debate ways forward in finding novel strategies and technologies, identifying trends and generally working towards more cost – effective solutions.

Key sessions: Upstream process development for gene therapies Upstream process development for cell-based therapies

Downstream processing considerations

Analytics - tackling measurement assurance for advanced cellular therapeutics

Insights into business development and reimbursement

Regulatory perspectives

Bioprocessing of Advanced Cellular Therapies Congress2 - 3 June 2016, London Heathrow Marriot Hotel, London, UK

Event overview:

MnM COnFEREnCES



Gold Sponsors:

Sponsors & Exhibitors

Media Partners:

Supporting Association:



Bioprocessing of Advanced Cellular Therapies Congress

Speaker Panel:

Ivan WallSenior Lecturer, Cell Therapy

BioprocessingUniversity College of

London, UK

Mark McCallEnterprise Fellow, Centre for

Biological EngineeringLoughborough University,

UK

Marc BaradezLead Scientist Analytical

DevelopmentCell Therapy Catapult, UK

Olive SturtevantAdministrative Director of Connell

& O’Reilly FamiliesDana Farber Cancer Institute,

USA

Damian MarshallHead of Analytical Development

Cell Therapy Catapult, UK

Katharina Winnemöller Marketing Manager,Miltenyi Biotec, UK

Qasim RafiqAssistant Professor, Bioprocess engineering, Aston Medical Research Institute, Aston

University, UK

Jennifer MoodyVice President, CommercializationCentre for Commercialization

of Regenerative Medicine, Canada

Peter JonesHead of Manufacturing

DevelopmentOxford BioMedica,

UK

Michael S. KallosAssociate Director of the

Pharmaceutical Production Research Facility

University of Calgary, Calgary (AL), Canada

Margarida Menezes-FerreiraNational Scientific Advice

Coordinator, Senior Assessor/Medicines Evaluation Department

INFARMED, Portugal

Kevin MurrayVP Global Sales

BioSpherix

Robert J ThomasReader in Manufacturing for

Cell Based Therapies, Centre for Biological Engineering (CBE),

Loughborough University, UK

Nina KotsopoulouDirector,

Process DevelopmentAutolus Ltd, London, UK

Steve OhDirector Stem Cell Bioprocessing,

BIP Programme Director & Senior Principal Scientist, Stem

Cell Group, Bioprocessing Technology Institute, Singapore

Bo KaraHead Process Development, Advanced Therapy Delivery

GlaxoSmithKline, UK

Otto-Wilhelm MertenHead of the Applied

Vectorology and InnovationGénéthon, France

Jurjen VelthuisVice President CMCKiadis Pharma,

Netherlands

John GrayVP, R&D

Audentes Therapeutics, USA

Sven Kili, MDVP, Head of Gene Therapy

Development, R&D Rare Disease UnitGlaxoSmithKline, UK

Paula AlvesChief Executive OfficerInstituto De Biologia

Experimental e Tecnológica, Portugal

Anne PlantProgram Director for Biosciences

National Institute of Standards and Technology,

USA

Stephen MingerMember of the Board of DirectorsCentre for Commercialization

of Regenerative Medicine, Canada

Advisory Panel:

Fernanda MasriProcess Development Engineer

Centre for Commercialization of Regenerative Medicine,

Canada

Otto-Wilhelm MertenHead Of The Applied

Vectorology And InnovationGénéthon, France

Bo KaraHead Process Development, Advanced Therapy Delivery

GSK, UK

Anita JoshiBiotechnology

ConsultantMarketsandMarkets, India

Ana Sofia CoroadinhaHead of Cell Line Development

& Molecular BiotechnologyIBET/ITQB-UNL,

Animal Cell Technology UnitPortugal

MnM COnFEREnCES



Day 1, 2nd June, Thursday08:00 Registration and Coffee

08:50 MnM Conferences’ Welcome Address

08:55 Opening remarks by the Chair

09:00 From translation to commercialisation of advanced cellular therapies• An overview of where the field is at and the ongoing efforts to commercialise cutting edge products. • This talk will help us identify strategies to accelerate the development and success of novel cell and gene therapies as well as address key topics that

are the foundation for this 2 day congress.• What are the major hurdles identified by the industry that are slowing the success of these therapies and how are addressing them?

Invited: Enrico Bastianelli, CEO, Bone Therapeutics

Upstream Process Development of Gene TherapiesChair: Otto-Wilhelm Merten, Head of the Applied Vectorology and Innovation, Généthon, France

09:30 An overview of state of the art upstream processes for gene therapies• Updates on ongoing upstream processes to render manufacturing more efficient and apt to provide gene therapy medicaments at the required quantity and quality. • Current hurdles that need to be addressed will be defined and ways to overcome them will be proposed:

- Increase in product quantity- Increase in product quality- Reduction in production costs

Otto-Wilhelm Merten, Head of the Applied Vectorology and Innovation, Généthon, France

09:55 Strategies for efficient cell line selection and optimal stable cell line development• Challenges of stable virus vector production as distinct from recombinant proteins• Lessons learned from lentivirus producer cell line engineering• Leveraging new technologies for optimization of cell line derivation

John Gray, VP, Research and Development, Audentes Therapeutics, USA

10:20 Challenges and solutions for establishing commercial manufacturing processes for ex-vivo cell- gene therapies• Expression and manufacturing platforms for lentivirus production and cell processing• Commercilaisation – supply chain and manufacturing models

Bo Kara, Head of Process Development, GSK, UK

10:45 Company Introduction: ChemometecBen Mantle, Area Manager, Chemometec A/S, UK

10:50 Morning Refreshments – Networking – Poster presentation and One to One meetings

11:35 Cell therapy manufacturing - making the future now• Introduction of the T cell transduction Porcess on the CliniMACS Prodigy• Data of robustness of the process and preclinical outlook• Glimpse into the future of process development on the CliniMACS Prodigy Platform

Katharina Winnemöller, Marketing Manager, Miltenyi Biotec, UK

Upstream Process Development of Cell-Based TherapiesChair: Steve Oh, Director, Stem Cell Bioprocessing, BTI, Bioprocessing Technology Institute, Singapore

11:55 Establishing the four pillars of stem cell bioprocessing• Addressing the four main issues pertinent to stem cell bioprocessing:

- Fluorescent probes for identification of potent and senescent stem cell populations.- Developing integrated stem cell expansion and differentiation processes in bioreactors.- Biodegradable microcarriers for implantation of stem cells post expansion.- Reprogramming technologies that can be automated at higher throughput for selecting fast growing and differentiating stem cell populations.

Steve Oh, Director, Stem Cell Bioprocessing, BTI, Singapore

12:20 Advanced optimisation and process control strategies for haematopoietic cell therapies• Specific challenges and approaches to production of red cells, megakaryocytes and haematopoietic progenitors in conventional bioreactors.• Process models and process/bioreactor modifications that enable targeting limitations of production efficiency and cell quality. • Adherent cell bioreactor development work for MSCs.

Robert J Thomas, Reader in Manufacturing for Cell Based Therapies, Loughborough University, UK



12:45 Bioprocess Development Using Stirred Suspension Bioreactors• Bioreactor modelling: Shear can influence pluripotent stem cell phenotype• Expanding skin derived Schwann cells on microcarriers

Michael S. Kallos, Associate Director of the Pharmaceutical Production Research Facility, University of Calgary, Canada

13:10 Process development strategies towards scalable and consistent manufacture of stem cells• Control of raw materials significantly reduces variation and improves process consistency• Development of suitable small-scale models allows for process optimisation • Advances in technology and process understanding will facilitate scale-up and enable successful translation

Qasim Rafiq, Assistant Professor, Bioprocess engineering, Aston Medical Research Institute, Aston University, UK

13:30 Lunch – Networking – Poster presentation and One to One Meetings

Moving away from clean rooms to modular closed manufacturing

14:35 Features for next generation bioprocesses: modular, closed, scalable and compliant manufacturing systems • Developing fully closed automated end-to-end cellular bioprocessing technology• Get rid of the humans in the processing of cells• Developing next generation tissue/organoid products

Keynote: Stephen Minger, Member of the Board of Directors, CCRM

15:00 Panel discussion - Modular closed manufacturing: issues and approaches• Operating principles• Converting existing assets• Current facilities

1. Jürgen Velthius, Vice President CMC, Kiadis Biopharma 2. Robert Thomas, Academic Fellow, Loughborough University 3. Stephen Minger, Member of the Board of Directors, CCRM

15:30 Modular Closed Platform for Total Quality Cell and Gene Therapy Production• A total quality approach produces a safer, more consistent product• Modular closed systems offer dramatic advantages over clean rooms• Fast, efficient scale up/scale out capabilities

Kevin Murray, VP Global Sales, BioSpherix

16:00 Afternoon Refreshments – Networking and One to One Meetings

Downstream Processing ConsiderationsChair: Paula Alves, Chief Executive Officer of IBET, Portugal

16:50 Overview to downstream processing strategies and needs for cell and gene therapies• As the upstream technologies mature, critical downstream processing steps need to have the capacity to handle large volumes of product.• After harvest, the cells or viral vector typically need a combination of the following operations: concentration, washing, formulation for

cryopreservation, mixing and vialling into final containers.• With such labile products, these are far from trivial operations and small variations can have a significant impact on product quality and/or

recovery. This talk will present an overview of the considerations during downstream processing for both cell and gene therapies.Paula Alves, Chief Executive Officer, IBET, Portugal

17:15 Overcoming challenges in large-scale downstream processing of lentiviral vector-based gene therapies• Industrialisation of process development and manufacturing technologies to deliver production systems for lentiviral vectors.• Delivering commercially viable and regulatory compliant platforms for lentiviral vectors.

Peter Jones, Head of Manufacturing Development, Oxford BioMedica, UK

17:40 Process Development for CAR-T therapies• Considerations for early phase processes for vector and T cell production• Implementing process changes for later stage development/commercial• Drug Product release and characterisation testing and managing comparability studies

Nina Kotsopoulou, Director, Process Development, Autolus Ltd.

18:05 Closing remarks by the chair

18:10 Short Talk by CCRM (Drinks Reception Sponsor)Jennifer Moody, Vice President, Commercialization, CCRM, Canada

18:15 Networking and drinks reception



Day 2, 3rd June, Friday08:15 Registration and Coffee

08:50 Opening remarks by the ChairAnne Plant, Chief, Biosystems and Biomaterials Division, NIST, USA

09:00 Opening Keynote: Measurement Challenges for Cell Therapies• Robust and meaningful assays that provide metrics of safety and effectiveness are needed for characterization of product, and for release assays. • As manufacturing processes mature, it becomes increasingly important to establish comparability between product produced at different times and

places and after changes in the manufacturing process. • A key is to apply the principles of measurement assurance, supporting data, and documentation to provide confidence in the comparability of

measurements. Examples in cell counting, flow cytometry, and quantitative imaging will be presented. Anne Plant, Chief, Biosystems and Biomaterials Division, NIST, USA

Analytics – tackling measurement assurance for advanced cellular therapeuticsChairperson: Damian Marshall, Head of Analytical Development, CT Catapult, UK

09:30 Evolving strategies for cell therapy product characterisation• Emerging approaches for cell therapy characterisation with an emphasis on:

- The role of QbD in defining a characterisation strategy- The challenge of multiplexed data analysis during product characterisation - Emergent approaches for characterising cell therapy products

Damian Marshall, Head of Analytical Development, CT Catapult, UK

09:55 Developing potency assays and making appropriate measurements of function for clinical application of stem cells• Challenges of measuring potency in MSCs• Developing suites of assays that corroborate function• Ensuring assays are linked to physiologic scenarios

Ivan Wall, Senior Lecturer, Cell Therapy Bioprocessing, UCL, UK

10:20 Analytical Challenges for the development of Cell Therapies• Requirements for product characterisation• Analytical strategies to de-risk cell therapy development• The convoluted road to standardisation

Marc Baradez, Lead Scientist Analytical Development, CT Catapult, UK

10:45 Morning Refreshments – Networking – Poster presentation and One to One meetings

11:30 Solution Provider Presentation; Please contact Steve Hambrook at [email protected]

Insights into Business Development and Reimbursement

11:45 The relevance of cost modelling tools for bioprocessing• Tools to confidently predict and reduce cell therapy cost of goods and associated manufacturing of regenerative medicine products.

Mark McCall, Enterprise Fellow, Centre for Biological Engineering, Loughborough University, UK

12:10 Evaluation of early stage technologies and strategies for advancement and partnership• How can translation centres de-risk and advance technologies along the commercial path?• This talk will address some key points the CCRM’s team considers during the assessment of incoming technologies.

Jennifer Moody, Vice President, Commercialization, CCRM, Canada

12:35 Developing market access strategies for cell-based and gene therapies: reimbursement pathways, price strategy and decision making for profitable therapies

• Reimbursement is not everything, but it does play a crucial role in the success of a therapy.• This talk will present different price strategies and decision making for profitable enterprises.

Speaker: TBA

13:00 Lunch – Networking – Poster presentation

14:00 CAR-T mock example for reimbursement – what is the optimum business model for CAR-T therapy space today?• This case study will look into reimbursement strategies of major players.• Participants will be grouped to compare and contrast approaches, rationale, challenges faced and end game of these key player’s strategies.

Invited: Christian Homsy, CEO, Ceylad



Regulatory PerspectiveChairperson: Margarida Menezes-Ferreira, National Scientific Advice Coordinator, Senior Assessor, Lisbon, Portugal

14:30 Regulatory environment for cell and gene therapy medicinal products• Efforts and progress towards a consolidated regulatory framework for these innovative medicines has recently led to many discussions and

developments in this area.• This talk will give an overview of the European regulatory environment for cell and gene therapy.

Margarida Menezes-Ferreira, National Scientific Advice Coordinator, Senior Assessor, INFARMED, Portugal

14:55 Understanding the regulatory space and strategies, specific for gene therapy products • This talk will address strategies for regulatory compliance, value creation, risk management and product safety for gene therapies.

Sven Kili, VP and Development Head for the Gene Therapy Division, GSK, UK

15:20 Practical considerations when designing a clinical trial to enhance trial compliance and patient safety• Review current applications for the use of Novel Cellular Products in various clinical trials while also addressing some of the trial design strategies,

considerations and unique features that create operational and logistical issues.• Discuss some of the unique manufacturing challenges in using autologous and directed allogeneic cellular products verses off-the-shelf standardise

materials• Review the need for and obstacles in capturing data both during and after the clinical trial

Olive Strurtevant, Administrative Director, Cell Manipulation Core Facility, Dana Farber Cancer Institute, USA

15:45 Chair’s closing remarks

15:50 Afternoon refreshments and end of conference

MnM COnFEREnCES

bioprocessing of advanced cellular therapies congress

Science