Cambridge Healthtech Institute’s 4th Annual
Cell Therapy Manufacturing, Operations and Logistics
Commercializing Cell Therapies
Part of CHI's Ninth Annual The Bioprocessing Summit

August 23-24, 2017 | Westin Copley Place | Boston, MA


CHI’s Cell Therapy Manufacturing, Operations and Logistics meeting takes an in-depth look at the practicalities of manufacturing autologous and allogenic cell therapies, focusing on key areas such as process development, scalability, bioreactors, next-generation production technologies, automation, closed systems, needle-to-needle logistics, supply chain and facility design. The meeting will feature extensive sessions on CAR-T and TCR production.

Final Agenda

Wednesday, August 23

7:00 am Registration Open and Morning Coffee

PROCESS DEVELOPMENT, SCALE-UP AND COMMERCIALIZATION STRATEGIES

8:05 Chairperson’s Opening Remarks

Knut Niss, Ph.D., Vice President, Program Management, Mustang Bio

8:15 Commercialization Challenges in Cell and Gene Therapy

Devyn_SmithDevyn Smith, Ph.D., COO, Sigilon

Differentiation, breakthrough, acceleration are the new buzzwords in the industry. How do we think about commercialization? Where are opportunities for new therapies? What are the challenges? There may be more answers than questions, but knowing the right questions to ask is important. This presentation will attempt to frame some of the key challenges and opportunities for new advanced therapies.

9:00 Making Cell Therapy Process Changes: Why, How, and When?

Greg_RussottiGreg Russotti, Ph.D., Vice President, Cell Therapy Development and Operations, Celgene Corporation

In order to make process changes for a cell therapy product, one must employ a combination of strong analytical tools, a thorough understanding of how the cell is intended to function as a therapy, detailed process characterization which elucidates how process parameters affect product attributes, and sound engineering principles to understand how the chemical and physical parameters of the process can be controlled to yield a comparable product after the process change. This presentation will discuss the reasons, methodologies, and timing for making process changes for cell therapy products.

9:30 FEATURED PRESENTATION: Gene Editing and Synthetic Biology Toolbox for Pluripotent Stem Cell Therapies

Robert_DeansRobert Deans, Ph.D., CTO, BlueRock Therapeutics

BlueRock Therapeutics is a next-generation regenerative medicine company that plans to develop best-in-class induced pluripotent stem cell (iPSC) therapies to cure a range of diseases using an industry-leading platform. This presentation will outline BlueRock’s strategy in cell therapy development based on latest stem cell technology in combination with superior cell differentiation protocols.

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

10:45 Commercial Assessments beyond COGS: Are Allogeneic Therapies a Better Business Model?

Knut_NissKnut Niss, Ph.D., Vice President, Program Management, Mustang Bio

In recent years, autologous cell therapies have come to the forefront of attention thanks to the emerging CAR-T technology. With the emergence of gene editing technologies, a new approach is being pursued in which T-cells are depleted of their TCR leading to “universal” T-cells that would not cause GvHD in an allogeneic recipient. This presentation will focus on some of the technical hurdles of generating allogeneic CAR-T cells as well as the potential challenges in the market place with such an approach.

11:15 Best Practice for a Viable Academic Cell and Gene Therapy Program

ChyAnn_Tran Chy-Anh Tran, Associate Director, Operations, Stanford Laboratory for Cell and Gene Medicine, Stanford School of Medicine

Stanford’s Laboratory for Cell and Gene Medicine engages a wide facet of therapies that treat genetic diseases, attacks cancers, and equips viruses to replace faulty genes with healthy, functional copies. We provide the roadmap to clinical applications and our model will de-risk and effectively translate research innovation into exciting clinical therapies that will have a profound impact on the medical, scientific, and patient communities.

Millipore Sigma  11:45 Automated, Closed Manufacturing for Allogeneic Therapies 
Julie Murrell, Ph.D., Head of Cell Therapy Bioprocessing, MilliporeSigma
The use of closed, single-use expansion and harvest systems can robustly expand and recover a variety of novel therapies. The inclusion of reagents that are animal origin free can lead to better yields and are supplied with a strong quality package. We will present data regarding ease of use, yield, viability and characterization for full solution expansion and harvest of manufactured cell therapies which are key enabling technologies for success in commercializing cell therapies.

12:15 pm Luncheon Presentation: Next Generation Cell Expansion Technologies for Stem Cell Therapy

Ryann Russell, Development Associate, Corning

1:00 Session Break

AUTOMATION, CLOSED SYSTEMS

1:45 Chairperson’s Remarks

Ohad Karnieli, Ph.D., MBA, CEO and Co-Founder, Atvio Biotech Ltd, Chair, Process and Product Development Subcommittee, International Society of Cellular Therapies

1:50 Benefits and Challenges Associated with Automation for Autologous Cellular Therapies: Why One Size Does Not Fit All

Jeff_CramJeff Cram, Ph.D., Senior Scientist, Cellular Process Development and Manufacturing, bluebird bio

Early phase cell therapies rely on largely manual and partially closed systems that pose challenges to commercialization. Automating and closing systems is particularly challenging with autologous therapies due to small batch sizes and variable starting material. An appropriate automation strategy must also maintain manufacturing flexibility. I will review the pros and cons of some available cell processing devices as well as the benefits of a modular approach to automation.

2:20 Innovative Solutions to Scaling Up and Out, Case Studies and Out-of-the-Box Thinking

Ohad_KarnieliOhad Karnieli, Ph.D., MBA, CEO and Co-Founder, Atvio Biotech Ltd, Chair, Process and Product Development Subcommittee, International Society of Cellular Therapies

As science evolves and cell therapies move from the bench to the clinic, a need for efficient and scalable solutions emerges. The commonly seen paths in the industry seem to be heading into a dead end. The traditional culturing technologies were not designed nor intended to culture cells at scale and a new and innovative thinking must emerge. The talk will describe and give tools and examples to such optional solutions.

2:50 Approach to Late-Stage Process Development and Characterization of an Allogeneic Cell Therapy Product

Ravi_BhatiaRavinder Bhatia, Ph.D., Director, Pharmaceutical Development and Manufacturing Sciences, Johnson & Johnson

In this presentation, a case study will be presented on the considerations to select technologies to develop a robust, and scalable process for allogeneic cell therapy products. Also, a control strategy based on QbD principles to consistently meet product quality by controlling the raw materials attributes and process parameters will be discussed.

GMP REQUIREMENTS, PROCESS VALIDATION AND LIFECYCLE MANAGEMENT

3:20 GMP Considerations for Cell-Based Therapies

Trevor_DeeksTrevor Deeks, Ph.D., Consultant, Deeks Pharmaceutical Consulting Services LLC

This talk will consider the generic differences between the manufacturing processes of cell-based therapies and classical biotechnology processes and how these impact the GMP requirements, the quality systems and the process and final product controls. It will use examples and cases, both from cell therapy processes and from similar, more established biotechnology products, with similar challenges, such as live vaccines, to illustrate the challenges and how these can be overcome in practice.

3:50 Refreshment Break in the Exhibit Hall with Poster Viewing

4:45 PLENARY KEYNOTE PRESENTATION: Click here for details

6:00 Networking Reception in the Exhibit Hall with Poster Viewing

7:00 Close of Day

Thursday, August 24

8:00 am Registration Open and Morning Coffee

CAR-T MANUFACTURING AND CHARACTERIZATION STRATEGIES

8:25 Chairperson’s Remarks

Anthony Davies, Ph.D., Executive Chairman, Dark Horse Consulting

8:30 Challenges and Solutions for Academic to Industry Translation of Personalized Engineered T Cell Therapies

Bruce_LevineBruce Levine, Ph.D., Barbara & Edward Netter Professor, Cancer Gene Therapy, Perelman School of Medicine, University of Pennsylvania

CAR T cells have demonstrated robust and durable clinical responses in relapsed and refractory malignancies. CAR T cell design includes elements of the receptor design, gene delivery to T cells, as well as the sourcing, manufacture, testing, delivery, and administration of the final CAR T cell product followed by clinical management of the patient. CAR T cells targeting new targets in hematologic malignancies and in solid tumors are underway and provide demonstration that it is possible to design immunity at will for therapeutic application.

9:00 Product Characterization Options for Cell and Gene Therapies

Sadik_KassimSadik H. Kassim, Ph.D., Vice President and Head, Cell Therapy Process and Analytical Development, Mustang Bio

As “living drugs”, cell therapies pose unique analytical characterization challenges; these challenges extend from the research bench to the cGMP manufacturing setting. This talk will review common analytical methods for cell therapies, specifically CAR T- and TCR-based therapies, with a specific emphasis on the limitations of current methods and desired areas of improvement.

MaxCyte 9:30 Bench to Bedside Manufacturing: Implications of Choosing the Right Cell Engineering Platform  

James Brady, Ph.D., Vice President of Technical Applications and Customer Support, MaxCyte

Autologous, cell-based treatments have shown tremendous potential to address the unmet medical needs of thousands of people. Advancement of cellular therapies depends on the ability of researchers and manufacturers to deliver engineered cell-based therapeutic products that are safe and exhibit enhanced potency with resulting clinical efficacy. The ability to engineer such enhanced potency using non-viral, cGMP-compliant, automated and closed system manufacturing processes will represent a significant advantage. To outline how such a process might work, we will summarize the application of a scalable, cGMP-compliant, electroporation platform for engineering dendritic cells (DCs), NK cells and T cells for development of cellular immunotherapies targeting a range of malignancies and other disease applications.

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

10:45 Process Development of CAR-T towards Commercialization

Marvin_LinMarvin Lin, Ph.D., Principal Scientist, Cell & Gene Therapy and Process Sciences, Novartis Pharmaceuticals Corporation


11:05 Capacity Management Paradigms in CAR T Cell Therapy Manufacturing

Jon_GuntherJon C. Gunther, Ph.D., Senior Engineer, Technical R&D, Juno Therapeutics

Juno Therapeutics has developed a plant simulation of its global manufacturing network, and has the ability to modify various aspects of its manufacturing operations, such as process design, staffing totals, shift schedules, facility configuration, and activity prioritization. An overview of Juno’s simulation and optimization methodology is presented here.

11:25 Leveraging TALEN, Gene Edited CAR-T Platform to Industrialize Off-the-Shelf Allogeneic Cell-Based Pharmaceuticals

David_SourdiveDavid Sourdive, Ph.D., Executive Vice President, Technical Operations, Cellectis

TALEN®-mediated gene editing can turn CAR-T cell therapies into off-the-shelf pharmaceutical products widely and readily available to large patient populations. It is also the key to expand the use of CAR-T cells beyond the few targets and indications they have been successfully used so far. With the first gene-edited CAR-T product candidates now in clinic, and the first allogeneic CAR-T application, Cellectis is at the forefront of this endeavor.

11:45 Manufacturing of Clinical Grade CAR-T NKR-2 THINK, Celyad First-in-Class Product Targeting Both Hematological and Solid Tumors

Sylvain_ArnouldSylvain Arnould, Ph.D., Director, Manufacturing and Production, Celyad

Celyad is developing a unique CAR-T cell platform, using Natural Killer Receptor (NKR) transduced on to T lymphocytes. Platform targets a wide range of solid and hematological tumors. Celyad’s lead candidate, CAR-T NKR-2, a CAR-T-Cell engineered express human NKR, NKG2D, and triggers cell killing through NKG2D binding to any of eight naturally occurring ligands overexpressed on more than 80% of tumors.

12:15 pm Enjoy Lunch on Your Own

1:15 Dessert Refreshment Break in the Exhibit Hall and Last Chance for Poster Viewing

1:55 Close of Conference

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