Cambridge Healthtech Institute ’s 2nd Annual

Cell Therapy CMC and Manufacturing

Characterizing and Commercializing Cell-Based Therapies

24-25 March 2020

 

Cambridge Healthtech Institute’s Cell Therapy CMC and Manufacturing conference takes an in-depth look at the practical challenges facing the manufacture and characterization of autologous and allogenic cell-based therapies, with dedicated sessions on emerging analytical methods, flexible CMC strategies, product release, cell processing, scalability, bioreactors, next-generation production technologies, automation, closed systems, supply chain and facility design.

Final Agenda

Monday, 23 March

Recommended Short Course*

13:00 - 16:00 SC2: Potency Assays for Cell and Gene Therapies

Instructor: Christopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.;

SC3: Technical Development of T Cell-Based Immunotherapies – Considerations and Lessons Learned

Instructor: Ali Mohamed, PhD, Vice President, CMC, Immatics

*Separate registration required.

Tuesday, 24 March

7:00 Registration and Morning Coffee

PROCESS CHANGES, COMPARABILITY AND SCALE-UP STRATEGIES

8:25 Chairperson’s Opening Remarks

Christopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

8:30 Genetically Modified Cells: Regulatory Considerations for the Modifying Agents

Niederlaender_ChristianeChristiane Niederlaender, PhD, Director, AMBR Consulting; Former Senior Quality Assessor for MHRA; Former Member, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA)

Different options are available for the in vitro genetic modification of cells, including viral vectors, plasmids and bacteria. Each of these modifying agents has distinct physical characteristics and therefore requires a tailored approach to demonstrate appropriate quality and application during the manufacture of GM cells. Different modifying agents are discussed in terms of their risk profile, and regulatory considerations are explained.

9:00 Advanced Therapies: Challenges and Opportunities for Standardization

Fouad_AtoufFouad Atouf, PhD, Vice President, Global Biologics, USP

This presentation will provide an overview of some of the analytical tools that are used to assess the quality control strategies of advanced therapies. Specifically, we will discuss global efforts for standardization of methodologies that will support product consistency.

9:30 KEYNOTE PRESENTATION: Of Apples and Oranges: Comparability in ATMP Development

Florence Salmon, PhD, Portfolio Lead Regulatory Affairs CMC, Cell and Gene Therapies, Novartis Pharma AG

Process changes are inevitable in product development, but for ATMP, this exercise is more frequently required, takes place later in development, and a high number of changes are implemented post-approval. Comparability is key to change management, but studies are more complex in design and execution than for biologics. They require full-scale runs and the outcome can be uncertain. Some examples from the development of Kymriah® will be presented.

10:00 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing

10:45 Developing Manufacturing Processes for the Production of Gene-Edited Allogeneic CAR T Cells

Jean-Charles_EpinatJean-Charles Epinat, PhD, Head, Process Development, Cellectis

This talk will discuss how gene editing is instrumental in moving cell therapies from grafts to off-the-shelf pharmaceuticals, evolving production and control concepts for gene-edited allogeneic cell product candidates, and how to approach manufacturing of genomically engineered designer cells.

11:15 Manufacturing Platforms for Endogenous, Autologous, and Allogeneic TCR T Cell Therapies for Solid Cancers

Ali_MohammedAli Mohamed, PhD, Vice President of CMC, Immatics US, Inc.

Immatics utilizes target discovery platform, XPRESIDENT®, which identifies tumor targets and screen cognate TCRs for off-target toxicities. TCRs against these tumor targets are used in various Immatics’ adoptive cellular therapy programs that include endogenous autologous, engineered autologous, and engineered allogeneic products for various cancer indications. Extensive process development led to the manufacturing approaches for the various product platforms and exemplary manufacturing and/or clinical data from various trials will be presented for the various platforms.

Polyplus-Transfection 11:45 Presentation to be Announced

 

12:15 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

13:00 Session Break

ADVANCING PRODUCT AND PROCESS CHARACTERIZATION

13:30 Chairperson’s Remarks

Rob Allen, PhD, Principal, Dark Horse Consulting

13:35 Product and Process Characterization of MSC-Based Cell Therapy Products

Foto_Maitane_OrtizMaitane Ortiz Virumbrales, PhD, Senior Scientist, Cell Therapies, Takeda

There is heterogeneity between mesenchymal stem cells (MSC) from different sources, manufacturing processes and donors. To guarantee the efficacy and safety of MSC in patients, there is a need to develop robust/standardized manufacturing processes, and a better understanding of the critical quality attributes (CQA). The established MSC identity markers do not predict bioactivity. We discuss the need to develop relevant predictors of efficacy, such as meaningful potency assays or predictive biomarkers.

14:05 Feasibility of a Large Scale Allogeneic Osteoblastic Cells Production

Lebrun_Anne-SophieAnne-Sophie Lebrun, PhD, Head, Manufacturing, Bone Therapeutics

The presentation will focus on designing a robust and cost-effective “off-the-shelf” supply chain for osteoblastic cell therapy, starting from a scientific concept to a commercially viable product. The case study will be based on a few examples of optimization implemented in the manufacturing process to reach the defined objectives.

14:35 Optimising Process Development

Labbrozzi_Juan_PabloJuan Pablo Labbrozzi, PhD, Principal Scientist, Process Development, Kiadis


15:05 Refreshment Break in the Exhibit Hall with Poster Viewing

15:45 Metabolomics in Stem Cell Therapy Manufacturing

Sakis_MantalarisAthanasios (Sakis) Mantalaris, PhD, Professor, W.H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology & Emory University

Analysis of the metabolome can be used for the evaluation of pluripotent and multipotent stem cell cultures, affording early detection of physiological changes indiscernible with traditional culture monitoring techniques. Finally, metabolomics can be used for the evaluation of the quality of stem cell differentiation in 2D and 3D biomaterial cultures, providing a sensitive and robust state-of-the-art technology guaranteeing high-quality cell therapy manufacturing.

16:15 Applying Machine Learning in Bioprocess Development Strategies

Tomishima_MarkMark Tomishima, PhD, Executive Director, Platform & Process, BlueRock Therapeutics

BlueRock Therapeutics is developing a new generation of living medicines from pluripotent stem cells. We harness developmental biology to create the foundation of our therapies, authentic cells, that replace cells lost to disease. Some therapies also use genome modification to engineer additional functionality into our medicines, creating our CELL+GENE platform. Here, we use a combination of single cell RNA-sequencing and machine learning to define and refine our cell therapies.

16:45 Breakout Discussion Groups

This session provides the opportunity to discuss a focused topic with peers from around the world in an open, collegial setting. Select from the list of topics available and join the moderated discussion to share ideas, gain insights, establish collaborations or commiserate about persistent challenges. Then, continue the discussion as you head into the lively exhibit hall for information about the latest technologies.

Comparability of Cell-based Therapies

Moderators: Christiane Niederlaender, PhD, Director, AMBR Consulting; Former Senior Quality Assessor for MHRA; Former Member, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA),

Florence Salmon, PhD, Portfolio Lead Regulatory Affairs CMC, Cell and Gene Therapies, Novartis Pharma AG

  • Process changes and demonstrating comparability
  • Coping with short development time frames
  • Using surrogate material
  • Determining the right assay panel

Developing and Controlling Cell-based Manufacturing

Moderator: Ali Mohamed, PhD, Vice President of CMC, Immatics US, Inc.

Athanasios (Sakis) Mantalaris, PhD, Professor, W.H. Coulter Department of Biomedical Engineering, Georgia Institute of Technology & Emory University

  • Regulatory approaches for IND approvals
  • Raw material, quality control, monitoring
  • Vein to vein times, closed manufacturing

Testing Approaches for AAV vectors: Challenges and Specifications

Moderators: Christine Le Bec, PhD, Head of CMC Analytical, Technology Development, Genethon

Eduard Ayuso, DVM, PhD, Team Leader, Innovative Vectorology; Scientific Director Translational Vector Core (CPV), Translational Gene Therapy for Genetic Disorders, Inserm, University of Nantes

  • Need for novel analytical tools
  • Biological limits for upscaling viral vector manufacturing
  • Vector genome titration
  • Ratio of empty/full particles, Host Cell DNA, Aggregates

17:30 Welcome Reception in the Exhibit Hall with Poster Viewing

18:30 End of Day

Wednesday, 25 March

8:00 Registration and Morning Coffee

PROCESS DEVELOPMENT AND VALIDATION

8:25 Chairperson’s Remarks

Rob Allen, PhD, Principal, Dark Horse Consulting

8:30 Closed, Automated and Controlled Processing for Cell-Based Therapies

Mustafa_Mohamed_MunyeMustafa Mohamed Munye, Lead Scientist, Industrialisation, Cell and Gene Therapy Catapult

Most manufacturing processes for cell-based therapies remain reliant on manual, open and poorly scalable technologies with limited in-process monitoring, increasing the risks of batch failure. We will present a number of solutions to close and automate bioprocessing for autologous cellular immunotherapies and iPSC and demonstrate the utility of novel process analytical technologies in driving manufacturing decisions, enabling better control and reduced risk of failure.

9:00 Efficient Processes for the Commercial Manufacture of Lentiviral Vectors

Carol_KnevelmanCarol Knevelman, PhD, Vice President, Head, Process R&D, Oxford Biomedica

Advanced therapeutics now attract significant interest due to the increasing number of exciting and high-profile products on the market and in clinical development. With over 20 years’ experience, the presentation will outline Oxford Biomedica’s (OXB) strategies to develop the next-generation manufacturing processes yielding suitable product quality attributes, in order to maximise capacity and advance development of a diverse product portfolio in therapeutic areas which currently present significant challenges.

9:30 Process Validation for Cell-Based Therapies

Christopher_BraveryChristopher Bravery, PhD, Consulting Regulatory Scientist, Consulting on Advanced Biologicals Ltd.

Before market approval, it is necessary to complete process validation of the commercial process. This can only be undertaken if sufficient process knowledge has been accrued to build on earlier process qualification activities to develop a suitable validation approach. This talk will explore some of the challenges with validation of manufacturing processes for cell-based medicines, and consider the various approaches that can be taken.

10:00 Presentation to be AnnouncedPolyplus-Transfection

 

10:15 Sponsored Presentation (Opportunity Available)

10:30 Coffee Break in the Exhibit Hall with Poster Viewing

PLENARY SESSION:

NEXT-GENERATION PROCESSES AND PRODUCTS

11:15 Chairperson’s Remarks

Jarka Glassey, PhD, Professor, Chemical Engineering, Engineering, Newcastle University

11:20 Current Opportunities and Challenges in Biotherapeutic CMC

Schumacher_RalfRalf Schumacher, PhD, Global Head, Development Biologicals, Boehringer Ingelheim Pharma GmbH & Co. KG

As biologic pipelines continue to grow and diversify, there is an increasing need to standardize, automate and find efficiencies along the entire value chain. This presentation will discuss the current challenges and opportunities in biotherapeutic CMC and the impact new modalities are having on upstream and downstream processing, analytics and formulation. The advantages of predictive process parameters in early stage development and digital development concepts to speed up the CMC development will also be discussed.

11:50 Gene Therapy Manufacturing and Technical Development

Diane_BlumenthaDiane Blumenthal, PhD, Head, Technical Development, Spark Therapeutics

In the past few years, several cell and gene therapy products have gained regulatory approval in the US and EU with many more in the pipeline. Manufacturers of cell and gene therapy products must tackle technological challenges under the pressure of short timelines resulting from streamlined clinical development. This presentation will focus on the key technical development challenges facing the industry as product development programs move the into the later stages of process development and scale-up, process performance qualification and ultimately commercialization.

12:20 Session Break

12:30 Bridging Luncheon Presentation: To be Announced

13:00 End of Cell Therapy CMC and Manufacturing

Japan-Flag Korea-Flag China-Simplified-Flag China-Traditional-Flag