Cambridge Healthtech Institute ’s 2nd Annual

Cell Therapy CMC and Manufacturing

Characterizing and Commercializing Cell-Based Therapies

New Dates - 21-22 JULY 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

Tuesday, 21 july

8:55 Welcome Introduction

PROCESS CHANGES, COMPARABILITY AND SCALE-UP STRATEGIES

9:00 Chairperson’s Opening Remarks

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

9:05 Genetically Modified Cells: Regulatory Considerations for the Modifying Agents

Niederlaender_ChristianeDirector, AMBR-Consulting; at Former Senior Quality Assessor for Biologics, Medicines and Healthcare ProductsRegulatory Agency (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.

9:25 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.

9:45 Sponsored Presentation (Opportunity Available)

10:05 Q&A, Session Wrap-up

10:20 Break Time to View our Virtual Exhibit Hall

10:50 Chairperson’s Opening Remarks

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

10:55 Developing Manufacturing Processes for the Production of Gene-Edited Allogeneic CAR T Cells

Enda Shevlin, 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 Optimising Process Development

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

 

 

 

 

11:35 Sponsored Presentation (Opportunity Available)

11:55 Q&A, Session Wrap-up

12:10 Lunch Break - Come and View our Virtual Exhibit Hall

ADVANCING PRODUCT AND PROCESS CHARACTERIZATION

12:40 Chairperson’s Remarks

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

12:45 COG for ATMPs

Robert Allen, PhD, Consultant, Dark Horse Consultancy

13:05 Closed, Automated and Controlled Processing for Cell-Based Therapies

Macown_RhysRhys Macown, PhD, 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.

13:25 Q&A, Session Wrap-up

13:40 Break Time to View our Virtual Exhibit Hall

13:55 Chairperson’s Remarks

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

14.00 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.

14:20 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.

14:40 Q&A, Session Wrap-up

14:55 Virtual Happy Hour in our Virtual Exhibit HallPurolite_Life_Sciences

15:15 Breakout Discussion Groups

This Virtual 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.

Comparability of Cell-based Therapies

Moderators:

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.

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

    Wednesday, 22 july

    PROCESS DEVELOPMENT AND VALIDATION

    9:00 Chairperson’s Remarks

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

    9:05 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.

    9:25 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.

    9:45 Sponsor Presentation (Opportunity Available)

    10:05 Q&A, Session Wrap-up

    10:20 Break Time to View our Virtual Exhibit Hall

    ADVANCING CMC AND ANALYTICAL STRATEGIES

    10:40 Chairperson’s Opening Remarks

    Christine Le Bec, PhD, Head, CMC Gene Therapy, Sensorion

    10:45 Analytical Challenges for Gene Therapy

    Clare_BlueClare Blue, PhD, Director, Analytical Development, Biogen

    One of the biggest challenges for AAV gene therapy products is establishing an appropriate analytical strategy to support product manufacture, release, stability, comparability and characterization at different stages of development. This presentation will highlight some of the existing analytical challenges and provide guidance on development of an appropriate strategy to help overcome these.

    11:05 Relative vs. Absolute Quantification of Purified and In-Process rAAV Productions

    Pytel_KamilaKamila Pytel, PhD, Lead, CMC Analytical Development, Gyroscope Therapeutics Ltd.

    Gene therapy delivery of a drug product requires precise determination of vector titre by a suitably qualified analytical method. Despite the importance of titre assays for product release, optimisation of R&D methods is also crucial to allow for better understanding of the changes in rAAV titre and yield from upstream to downstream unit operations. qPCR is widely considered the gold standard for this purpose. In addition, we adopted orthogonal analytical tools including ddPCR and HPLC as robust and rapid titration alternatives to qPCR.

    11:25 Optimizing CAR-T Process Development by Utilizing a Novel Small Scale Stirred Tank BioreactorSartorius_NEW

    Fuerstenau-Sharp_MayaMaya Fuerstenau-Sharp, PhD, Regenerative Medicines, Sartorius Stedim Biotech 

    Currently, static and shake flasks for suspension processes are widely used in the process development of advanced therapies. However, these cell expansion approaches are labor-intensive and require a high level of highly skilled operator manipulation and offer only a low level of cell culture monitoring and control.  Here we demonstrate the utility of the ambr250 modular and the newly designed unbaffled single impeller vessel as a process evaluation tool for the expansion of CAR-T cells.   

    11:45 Q&A, Session Wrap-up

    12:00 Lunch Break - Come view our virtual Exhibit Hall

     

    PLENARY SESSION:

    NEXT-GENERATION PROCESSES AND PRODUCTS

    12:25 Chairperson’s Remarks

    To be Confirmed

    12:30 Continuous Processing for Vaccine Manufacturing: Challenges and Opportunities

    Yang-PingYangYan-Ping Yang, PhD, Head of Bioprocess Research & Development, North America, Sanofi Pasteur

    Over the last decade, there have been significant investments in continuous manufacturing in the pharmaceutical industry, as it holds great promise to lead the reduction of process steps, smaller footprint, higher product quality, and better pharmaceuticals for patients. While it’s still in its early stage, the vaccine industry has embraced this concept and is ready to explore the full advantages associated with this approach. This presentation explores the challenges and opportunities to make continuous vaccine manufacturing a reality.

    12:55 Gene Therapy Manufacturing and Technical Development

    Blumenthal_DianeDiane 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.

    13:20 Q&A, Session Wrap-up, Host intro to special virtual features

    13:00 End of Cell Therapy CMC and Manufacturing

    BPDE Brochure RC