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08:30 - 09:00


Showing of Streams
16:00 - 16:05
End of Workshop

End of Workshop

08:30 - 09:00 30 mins
09:00 - 09:40 40 mins
Workshop 1: Raw Materials Focus Day
Starting Materials and Standards for Cell Therapies
  • Jack Price - Head of the Division of Advanced Therapies, NIBSC, UK

The Division of Advanced Therapies at NIBSC is houses the UK Stem Cell Bank.  The Bank collects, archives, and distributes human ES lines produced in the UK, plus a number generated outside the UK—and makes them available for researchers and commercial developers.  We are currently generating a series of hES lines in GLP facilities to EUTCD-grade, to act as starting material for the production of cellular therapies.  In conjunction, we are developing a regulator-ready data portfolio to accompany each line, with a view to facilitating the development of clinical grade materials.

In conjunction with our role as a repository for human pluripotent stem cells, we are the leading producer of WHO standards, responsible for roughly 85% of such standards world-wide.  We are well placed, therefore, to coordinate the production of clinical grade materials with our experience in the production of standards and cell assays towards the clinical development of cell therapies.

In this presentation, I shall outline our work in the production of pluripotent cells and their derivatives, the generation of data portfolios to support regulatory submissions of those cells, and our first steps towards the generation of physical standards for cell therapies.

09:00 - 16:00 420 mins
Workshop 2: Comparability for ATMPs
Comparability for ATMPs
  • Christopher Bravery - Director, Training Courses Department, Consulting On Advanced Biologicals Ltd
  • Karin Hoogendoorn - Scientist ATMP Product Development, Leiden University Medical Centre (LUMC), The Netherlands

Change is inevitable and necessary both in development and over the post-approval product lifecycle. Whenever changes are made it is necessary to confirm they do not adversely impact the quality and therefore safety and efficacy of the product; this requires data beyond meeting current specifications. With any biological product this is challenging, for cell, gene and tissue products that cannot be fully characterised the challenges are greater still. Concerns about comparability undertaken during development are common issues raised during review and often delay market approval or contribute to failure. This course explains what comparability is and how to develop a successful comparability protocol.

Workshop Learning Overview:

  • What is comparability?
  • Why is meeting existing specifications not comparability?
  • How do I apply the principles of comparability to highly variable products?
  • Case studies: Common mistakes with comparability and their consequences.
  •  Interactive exercise: Spot the weaknesses and propose improvements to a worked comparability study.
09:40 - 10:20 40 mins
Workshop 1: Raw Materials Focus Day
Quality Management for Raw Materials
  • Jakub Cierny - Senior Quality Compliance Manager, Sotio AS, Czech Republic
  • Quality Agreements – what is expected and needed
  • Quality audits – luxury or a must
  • Quality of raw material – should we go for GMP grade?
  • Setting internal specifications
  • Testing of raw material
10:20 - 10:50 30 mins
Workshop 1: Raw Materials Focus Day
Morning Coffee and Networking
10:50 - 11:30 40 mins
Workshop 1: Raw Materials Focus Day
Oversight of 3rd Party Raw Material and Biological Starting Material Suppliers for Cell Therapy Manufacturing
  • Joseph Carosi - Associate Director, External CMO QA for Cell and Gene Therapies, Novartis, USA

For medicinal products derived from Cellular and Gene Therapy manufacturing, establishing quality controls on biological starting materials and other raw materials varies from traditional raw material quality controls in common biopharmaceutical manufacturing especially when the starting materials originate from human tissue or directly from human origins.  In a cell and gene therapy manufacturing process, there may be customized raw materials such as media and ancillary materials.  The expectation of meeting EMA guidances on testing for purity, potency, consistency and stability has unique challenges which are weighed against the impact of the material to the manufacturing process and the safety concerns for the patient who receives the finished product.  Evaluations of safety critical features may be emphasized more so than other analytical testing methodologies.  For biological starting material, the traditional EMA guidance in EudraLex, Volume 4 for Medcinal Products, Annex 2, Table 1 defers to the guidances in Directive 2004/23/EC and recent EudraLex Guidelines on Good Manufacturing Practice specific to Advanced Therapy Medicinal Products (effective 22 May 2018).  The overight of suppliers of these two materials will be contrasted to show similarities and differences in the management and quality control concerns for both.

11:30 - 12:10 40 mins
Workshop 1: Raw Materials Focus Day
Implementation of Serum-Free Medium in a Chimeric Antigen Receptor T Cell Manufacturing Process

Many early autologous chimeric antigen receptor (CAR) T cell manufacturing processes relied on the addition of complex animal-derived materials (ADM), such as human and bovine sera, to support T cell expansion.  In addition to country of origin sourcing controls and viral testing, viral inactivation treatments, such gamma irradiation, are recommended to further mitigate the risk of adventitious agents.  However, irradiation of serum may lower its performance, thereby further increasing the variability associated with complex ADMs.  The implementation of serum-free medium (SFM) may significantly reduce the variability and challenges associated with complexes ADMs, while simplifying sourcing and manufacturing.  Selection and implementation of SFM in a CAR T cell manufacturing platform presents several challenges.  First, the limited availability of patient T cells requires the use of healthy donor cells as a surrogate to screen large numbers of SFM formulations.  Second, CAR T cell manufacturing processes may not allow for the gradual adaptation of T cells to SFM conditions.  Finally, rather than tailoring a SFM formulation to a single cell line, it must provide sufficient robustness to support CAR T cell manufacturing across a diverse patient population.  Overall, the strategy and design considerations towards the selection and implementation of SFM in a CAR T cell manufacturing platform to support future programs will be discussed.          

12:10 - 12:50 40 mins
Workshop 1: Raw Materials Focus Day
Serum Traceability – how can you tell what is in your bottle?
  • Jenny Murray - Managing Director, Life Science Group Ltd, President, International Serum Industry Association (ISIA)
12:50 - 13:45 55 mins
Workshop 1: Raw Materials Focus Day
Lunch and Networking
13:45 - 14:00 15 mins
Workshop 1: Raw Materials Focus Day
Buses Leave for Site Visit
14:00 - 16:00 120 mins
Workshop 1: Raw Materials Focus Day
  • Patrick Burger - Project Leader Laboratory for Cell Therapy, Sanquin Research

Sanquin is a knowledge-driven not-for-profit organization that supplies life-saving products and focuses on health care needs. Research helps us find new solutions for medical problems in the fields of transfusion medicine, hematology and immunology. We are constantly aware of our responsibility to donors – to handle their gift carefully, efficiently and responsibly – and to patients – whose safety and wellbeing is a priority. 

With almost 3,000 committed colleagues and more than 330,000 blood donors, Sanquin can provide a better life for 300,000 Dutch patients each year.

In addition to collecting, processing and distributing blood products, Sanquin also:

  • produces plasma pharmaceuticals,
  • develops blood group and immune reagents,
  • performs a multitude of diagnostic services,
  • conducts high-quality scientific research,
  • collect, process, cryopreserve and distribute (stem)cell products
  • and provides education and training.

Sanquin is the only blood organization in the world to offer this combination of in-house medical, pharmaceutical and scientific knowledge and expertise.

Over the course of this 2-hour tour, attendees will get to see and hear about, on a tour on Sanquin’s new state of the art facility:

  • Blood bank
  • Reagents
  • Diagnostics
  • Cell Therapy Laboratory

Places are capped at 25 so please register early 

16:00 - 16:05 5 mins
End of Workshop