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08:25 - 08:30
Chairperson’s Opening Remarks

Chairperson’s Opening Remarks

  • Anthony Davies - Executive Chairman, Dark Horse Consulting Inc, USA
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Showing of Streams
10:25 - 11:00

Morning Coffee

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Showing of Streams
12:50 - 14:00

Lunch and Networking

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Showing of Streams
15:45 - 16:10

Afternoon Coffee

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Showing of Streams
16:45 - 16:50
End of Day Two

End of Day Two

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08:25 - 08:30 5 mins
Chairperson’s Opening Remarks
  • Anthony Davies - Executive Chairman, Dark Horse Consulting Inc, USA
08:30 - 08:50 20 mins
Joint Plenary Session
GMP for ATMP, the new EU guideline
  • Jean-Luc Golnez - GMP Inspector, FAMHP, Belgium
08:50 - 09:10 20 mins
Joint Plenary Session
International standardization efforts for accelerating the development and commercialization of cell and gene therapy products
  • Sheng Lin-Gibson - Chief of Biosystems and Biomaterials Division, National Institutes of Standards & Technology
09:10 - 09:45 35 mins
Info
Joint Plenary Session
Breakfast Surgery – FDA, EMA and PMDA Pinch Points
  • Andreea Barbu - Assessor, Farmacy & Biotechnology Department, Pharmaceutics and Biotechnology, MPA, Sweden
  • Jean-Luc Golnez - GMP Inspector, FAMHP, Belgium
  • Catherine Cancian - Vice President of Pharmaceutical Operations, GenSight Biologics
  • Sheng Lin-Gibson - Chief of Biosystems and Biomaterials Division, National Institutes of Standards & Technology
  • Francesco Cicirello - Chairperson PIC/S Working Group on Annex 2 Revision, Inspector, Australia

Knect365 is looking for 5-6 panellists, comprised of suppliers, manufacturers and regulators to join this breakfast surgery. Attendees will have the opportunity to submit their ‘pinch’ points anonymously to the panel in advance and this session will provide the perfect opportunity to hear from the experts on expectations

Topics to be covered include:

  • Statistical tools for data assessments
  • GMP for ATMP’s – the new guideline and what it means for industry
  • Raw materials and viral safety considerations
  • Analytics and CMC – Potency assays, comparability etc.
  • Interactions with competent authorities for drugs, tissues & cells
  • Compendial testing – safety testing, and sterility


09:45 - 10:05 20 mins
Info
Joint Plenary Session
Evaluation of Poros AAVX Pan-Affinity Resin for rAAV Purification and cGMP Manufacturing
  • Daniel Hurwit - Senior Associate Engineer, Voyager Therapeutics, USA

Affinity interaction based resins are the foundation for the platform purification of many different biological molecules. While some AAV affinity resins have existed for more than a decade, the growth in the AAV field now demands a true pan-affinity workhorse, analogous to Protein A for mAbs.  In this work we investigate POROS CaptureSelect AAVX (AAVX) for the purification of various serotypes from both crude and pure feed streams.  Performance of AAVX is directly compared against other commercially available AAV affinity resins.  We evaluate binding capacities, binding kinetics, product recovery, impurity clearance, washing and elution strategies, cleanability, cyclability, regeneration as well as the scalability and suitability of the new resin for commercial manufacturing. The results demonstrate that the new AAVX resin is a robust option for the scalable, platform purification of various rAAV capsids from crude feed streams.

10:05 - 10:25 20 mins
Info
Joint Plenary Session
Addressing the challenges of cell and gene therapy commercialization
  • Øystein Åmellem - Director, Cell Biology, Cell Culture and Cell Therapy, Thermo Fisher Scientific

The manufacturing of T cell and gene therapies is faced with multiple challenges that must be solved to create sustainable solutions to meet global commercialization requirements and quality standards. In addition to solutions for closed and automated manufacturing that improve safety and consistency, novel technologies are required to improve biological precision to utilize the potential of optimal T cell subsets in areas of unmet medical needs, such as solid tumors (90% of cancers).

Examples on how industry addresses these challenges will be discussed, including a solution that reduces variability in the biological starting material by simultaneous isolation and activation of naïve and early memory T cells with a demonstrated capacity to persist in vivo. This step is particularly important in autologous therapies where the quality of the patient’s T cells vary heavily with the disease and prior therapies. Moreover, solutions to improve productivity and scalability will be presented, including a xeno-free serum replacement that eliminates the use of human serum, a novel viral vector production system that enhances productivity, and a rapid time-to-result Mycoplasma detection kit that supports in-process monitoring during T cell manufacturing, release of critical raw materials (e.g. CAR vector) and batch release.

10:25 - 11:00 35 mins
Morning Coffee
11:00 - 11:05 5 mins
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Chairperson's Opening Remarks
  • Ohad Karnieli - CEO & Co-Founder, Atvio, Israel
11:00 - 11:05 5 mins
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
Chairperson's Opening Remarks
  • Greg Stromberg - Associate Director, Global Manufacturing Sciences, Biogen, USA
11:00 - 11:05 5 mins
Stream 3: Clinical Development for Cell, Gene and Immunotherapies
Chairperson's Opening Remarks
  • Shashi Murthy, PhD - Professor of Chemical Engineering, Northeastern University, USA
11:05 - 11:40 35 mins
Info
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Enabling commercial scale-out of T-cell manufacturing
  • Sandro Gomes - Investigator, Cell & Gene Therapy Product Development, GlaxoSmithKline
  • 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
11:05 - 11:40 35 mins
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
Revision of PIC/S Annex 2 for ATMP, challenges with automation and facilitation of cross-border manufacturing
  • Francesco Cicirello - Chairperson PIC/S Working Group on Annex 2 Revision, Inspector, Australia
11:40 - 12:15 35 mins
Info
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Adopting Various Manufacturing Closed Systems to Gene Modified T Cell Therapies
  • Ali Mohamed - CMC Vice President, Immatics US Inc,
  • Immatics developed a proprietary tumor antigen targets discovery
  • platform, XPRESIDENT®
  • The platform identifies novel tumor-specific targets and TCR candidate.
  • It also screens TCR candidates based on these targets against off-target
  • toxicities in absence of reliable in vivo models
  • Natural and engineered TCRs against these tumor targets have been used
  • in various Immatics’ Adoptive Cellular Therapy programs in “First In Man”
  • clinical trials
  • Extensive process development was carried out using primary T cells
  • derived from multiple healthy donors and cancer patients to optimize
  • each step in the manufacturing of TCR T cells for 3 clinical trials (IMA101,
  • IMA201, IMA202, and IMA203)
  • Manufacturing for engineered TCR T cell therapies has been adapted into
  • 3 different closed systems with for future phases of clinical trials with
  • excellent results
11:40 - 12:15 35 mins
Info
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
Challenges of Manufacturing Gene Therapies Into Commercial Realities
  • Steven Goodman - Senior Director, Drug Product Manufacturing, bluebirdbio, USA
  • Overview of bluebird bio’s efforts to advance gene therapies for severe genetic disorders and cancer immunotherapy
  • Challenges faced in manufacturing cell therapies as they move toward commercial phase
  • Approaches bluebird bio is implementing to overcome some of these challenges
11:40 - 12:15 35 mins
Info
Stream 3: Clinical Development for Cell, Gene and Immunotherapies
Challenges of Manufacturing Gene Therapies Into Commercial Realities
  • Steven Goodman - Senior Director, Drug Product Manufacturing, bluebirdbio, USA
  • Overview of bluebird bio’s efforts to advance gene therapies for severe genetic disorders and cancer immunotherapy
  • Challenges faced in manufacturing cell therapies as they move toward commercial phase
  • Approaches bluebird bio is implementing to overcome some of these challenges
12:15 - 12:30 15 mins
Info
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
How to support and improve CAR-T process performance through advanced data analytics
  • Kai Touw - Biopharma Market Manager, Sartorius Stedim Data Analytics, Sweden

With the growing interest in CAR-T processing, the industry is looking for more process understanding, which can be achieved using advanced data analytics. The nature of the process, from leukapheresis to vector manufacturing, can be a cause of variation, which might impact the patient response. In this talk opportunities for using Multivariate Data Analysis and Design of Experiments during the different stages of the CAR-T manufacturing process are discussed.

12:15 - 12:30 15 mins
Info
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
Characterization of viral vector samples at different process steps; morphology, integrity, purity and percentage full
  • Vanessa Carvalho - Senior Scientist, Vironova, AB
12:15 - 12:45 30 mins
Info
Stream 3: Clinical Development for Cell, Gene and Immunotherapies
Routine automated manufacture of CAR-T cell products in a closed system
  • Ian Johnston - Industrial and Academic Cooperations Manager, Senior Project Manager, Miltenyi Biotec GmbH

Using closed system manufacturing reduces the risks of contamination and the requirements for in process controls and clean room infrastructure. Using the example of CAR T cell-based immunotherapies, I will demonstrate how production of cell products can be fully automated in a single-use closed system, the CliniMACS Prodigy®. Routine manufacture of CAR T products has been established at a new state-of-the-art Cell Factory and the automated procedure has been transferred to a number of other sites in a robust manner. The use of closed systems and automation has allowed an easy establishment of robust processes at academic institutes and paves the way for an effective scaling out of these procedures for commercialization.

12:30 - 12:45 15 mins
Info
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Enhancing economic reality for cell based medicine through manufacturing optimization
  • Jean - Yves Lombard - Business Manager, Bioprocessing Solutions, Saint-Gobain, France
12:30 - 12:50 20 mins
Info
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
Spinning Membrane Filtration: Applications in Washing, Concentrating, and Fill/Finish Manufacturing for Cell and Gene Therapies
  • Steven Binninger - EU Cell Therapy Leader, Fresenius Kabi, Germany
12:50 - 14:00 70 mins
Lunch and Networking
14:00 - 14:35 35 mins
Info
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Principles, implications and benefits of applying QbD to analytics
  • Margit Jeschke - Global Head, Analytical Stewardship and CMC Correlation, Novartis, Switzerland
  • Characterising the product
  • Case study tisagenlecleucel, a CAR T cell product
14:00 - 14:35 35 mins
Info
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
Designing a manufacturing facility to meet scaling up requirements
  • Amanda Skulte - Cell & Gene Therapy Product Steward, Novartis, Switzerland

Case study

14:00 - 14:35 35 mins
Info
Stream 3: Clinical Development for Cell, Gene and Immunotherapies
Low Cost Automation to Drive Efficiency and Reproducibility: Case Studies
  • Shashi Murthy, PhD - Professor of Chemical Engineering, Northeastern University, USA
  • When should investments into automation be made?
  • Early in POC for long term process?
14:35 - 15:10 35 mins
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Creating sustainable process development strategies for Gene Therapy products
  • Barbara Kraus - Head of Gene Therapy Process Development Process Development & Technical Services, Shire
14:35 - 15:10 35 mins
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
Solving challenges in development, manufacture and delivery of cell and gene therapies
  • Lisa Fox - SVP, Cell Therapy Operations, Orchard Therapeutics North America, USA
14:35 - 15:10 35 mins
Stream 3: Clinical Development for Cell, Gene and Immunotherapies
Solving challenges in development, manufacture and delivery of cell and gene therapies
  • Lisa Fox - SVP, Cell Therapy Operations, Orchard Therapeutics North America, USA
15:10 - 15:30 20 mins
Info
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Streamlining the Cell Therapy Workflow with Silicon Chip Technology
  • Liesbet Lagae, PhD - Program Director Life Sciences Technologies, IMEC

A major hurdle to scale up the market of cell therapies is the cost and complexity of the workflow. We propose a novel approach based on a standardised workflow which will be easy to use, reliable and reproducible. Using silicon chip technology, fully integrated solutions can be built, combining microfluidics, photonics-based biosensors, PCR, and single cell manipulation. We will present a portfolio of technologies which will be the building blocks for bioprocessing and delivery of future personalised therapies.

15:10 - 15:25 15 mins
Info
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
The use of improved traditional GMP manufacturing techniques contributes to the success of Large Scale State-of-the-Art production of EMA-approved Cartilage Substitute
  • Marco Fadda - R&D Specialist, Comecer, Italy

Recent advancements in several different fields of Advanced Therapies manufacturing showed an increasing demand for new, enhanced, effective processing and expansion methods, aimed at reducing the impact of the COGS in the development, adoption and finally commercialization of ATMPs.

Moreover, discovery and clinical proof of different, improved treating methods for certain diseases, opens demand for having available high quantities of a given product, impacting on and challenging the production capacity for suitable and successful scale-up.

The approach described here, while not changing the process as initially developed and approved by the regulatory authorities for low-scale production, addresses the issues above, boosting the production capabilities in a brand-new full GMP-compliant designed plant. Using isolation technology, a new modular and flexible approach for large quantities incubation, an accurate matching between the low- and large-scale process steps, a significant level of process automation (both in worklist and patient material management) and finally, a complete top-class track&trace software control and management system, the plant will be able to deliver the required doses of the ATMP drug (several thousand patients per year), under full respect of all the quality and regulatory issues.


15:10 - 15:40 30 mins
Info
Stream 3: Clinical Development for Cell, Gene and Immunotherapies
Progress of first-in-class dominant negative PD-1 armored CAR-T cell clinical study
  • Lei Xiao - CEO, Innovative Cellular Therapeutic Co., Ltd, China
15:25 - 15:40 15 mins
Info
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
The need for decentralisation in closed loop supply chain management
  • Andreas Göbel - Managing Director, Hypertrust Patient Data Care, Germany
  • A closer look at closed loop supply chain processes of personalized medicine
  • How distributed ledger technology can secure and optimize the chain of identity and custody of personalized medicine
15:30 - 15:45 15 mins
Info
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Made for purpose manufacturing solutions - The key to commercialization
  • Nina Bauer - VP Business Development, FloDesign Sonics Inc, USA
15:45 - 16:10 25 mins
Afternoon Coffee
16:10 - 16:45 35 mins
Info
Stream 1: Manufacturing and Process Development – Analytical Strategies, Design Space, QbD and Cost of Goods Considerations
Meeting the challenge of delivering allogeneic therapies in the commercial sector
  • Robert Leach - Quality Control Manager, Cellectis, France
  • What is the future for autologous therapies?
  • Are they commercially viable
  • What are the strategies for ensuring that they are?
16:10 - 16:45 35 mins
Info
Stream 2: Facility Design, Manufacturing Networks and End to End Solutions for Commercial Success
Optimizing the cell collection network to meet manufacturing expectations
  • Petr Machalik - International Collection Network Manager, Be the Match BioTherapies, USA
  • What expectations for consistency and quality do manufacturers have for collection sites?
  •  How has the increased demand for cell and gene therapies impacted the efficiency of collection sites?
  •  What are key tools and strategies for effectively managing collection networks?

  • How can standardization create efficiencies within centres to ensure consistent, high-quality starting material is provided to manufacturers?

 

16:10 - 16:45 35 mins
Stream 3: Clinical Development for Cell, Gene and Immunotherapies
Current state of play for the cell therapy industry in the clinic – stats and facts
  • Tijana Ignjatovic - Senior Director, Trends and Market Access, Datamonitor, UK
16:45 - 16:50 5 mins
End of Day Two