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Key Sessions

Flemming Ornskov, M.D., M.P.H.

A Vision for Rare Disease: How We Can Champion Underserved Patients

Shire

James Collins, Ph.D.

Synthetic Biology: Biomedical Applications Come of Age

Massachusetts Institute of Technology

7:00am 8:10am (70 mins)

Main agenda

Coffee & Registration

8:10am 8:15am (5 mins)

Commercializing CAR-T Cell Treatments

Chairperson’s Remarks

  • David DiGiusto, Ph.D. - Executive Director, Stem Cells and Cellular Therapeutics Operations; Founding Director, Stanford Laboratory for Cell and Gene Medicine; Senior Research Scientist, Division of Pediatric Stem Transplantation and Regenerative Medicine, Stanford University

8:15am 8:45am (30 mins)

Commercializing CAR-T Cell Treatments

Manufacturing and Commercialization Strategies for CAR-T Cell Therapies: Novartis Case Study

8:45am 9:15am (30 mins)

Commercializing CAR-T Cell Treatments

Process Characterization and Comparability: Lessons for CAR T Cell Commercialization

The manufacturing process for engineering autologous T cell products is complicated by the inherent variability in cellular starting material, and ensuring manufacturing success requires careful development of a robust process and a well-trained production organization. Simple, robust manufacturing steps are designed to ensure that T cell products remain active and retain appropriate biologic activity.

  • John Tomtishen, Senior Scientist, BTDM Process Sciences- Cell & Gene Therapies

9:15am 9:45am (30 mins)

Commercializing CAR-T Cell Treatments

PANEL DISCUSSION: Cell-based Immunotherapy Manufacturers: Fast Followers and Next Generation Innovators

Following 30 second introductions on their platform, indication, stage of developments etc., a panel of speakers from a range of T-Cell based immunotherapies manufactures will be quizzed about their products, commercialization strategies, fears, concerns, experiences and visions for the future.

  • Moderator David DiGiusto, Ph.D. - Executive Director, Stem Cells and Cellular Therapeutics Operations; Founding Director, Stanford Laboratory for Cell and Gene Medicine; Senior Research Scientist, Division of Pediatric Stem Transplantation and Regenerative Medicine, Stanford University
  • Panelist Christian Homsy, M.D. - CEO, Celyad
  • Panelist Alan Smith, Ph.D. - Executive Vice President, Technical Operations, Bellicum Pharmaceuticals
  • Panelist Knut Niss, Ph.D. - VP Program Management, MustangBIO
  • Panelist Geoffrey Hodge - Chief Technical Officer, Unum Therapeutics
  • Panelist Laurence Cooper, Ph.D, M.D. - CEO, ZIOPHARM Oncology

9:45am 10:15am (30 mins)

Main agenda

Networking Refreshment Break

10:10am 10:15am (5 mins)

Bioprocessing

Chairperson’s Remarks

  • Rodney L. Rietze, Ph.D. - Bioprocess Science and Innovation, Exploratory Immuno-Oncology, Novartis Institutes for Biomedical Research

10:10am 10:15am (5 mins)

Commercialization

Chairperson’s Remarks

  • David Brindley, DPhil, MEng, FRSA - Managing Partner, Biolacuna & Senior Research Fellow, Nuf eld Department of Paediatrics, University of Oxford

10:10am 10:15am (5 mins)

Clinical

Chairperson’s Remarks

10:15am 10:45am (30 mins)

Clinical

Potency Testing for Expanded Stem/Progenitor Cell Product NLA101 - Can simpler be better?

  • Shelly Heimfield - Executive Vice President Manufacturing and Research, Nohla Therapeutics, USA

10:45am 11:15am (30 mins)

Clinical

T Cell Potency Assays-Honing in on Tumor Specificity

  • Linda Kelley - Senior Member & Cell Therapy Facility Director, Moffitt Cancer Center

11:15am 11:45am (30 mins)

Clinical

My Potency Assay is Quantitative; but is it Quantitative of Potency?

Potency assays should be quantitative, and in most cases the methods employed do quantify an analyte, e.g. (increasing) cell number, dead cells (cytotoxicity), cytokine release etc. However, when setting a specification for potency the questions become;

  • Is my assay quantitative for potency?
  • What is the threshold that assures the product is potent?
  • What data to I need to justification this specification?
  • Christopher Bravery, Ph.D. - Director, Consulting on Advanced Biologicals Ltd, Advanced Biologicals Ltd.

10:15am 10:45am (30 mins)

Commercialization

Enabling Greater Success of Cell Therapy Commercialization

Cell therapy is now entering an era where it has proven that it can succeed.  However, there are several opportunities for improvement to enable this success. This talk would highlight some of the challenges currently encountered across functions during scale-up and commercialization of cell therapy programs and present them as opportunities for improvement to enable better success of future programs.

  • Hari Kamaraju, MS, MBA - Senior Scientist, Janssen Research & Development

10:45am 11:15am (30 mins)

Commercialization

Planning for Commercialization: It All Starts (Really) Early!

With the advancement of the CAR-T and the emerging gene editing platforms, autologous cell therapies are on the frontier of commercialization. These complex therapies require careful planning early on in the lifecycle in order to avoid pitfalls towards commercial scale. This presentation will discuss these potential pitfalls and strategies to avoid them.

  • Knut Niss, Ph.D. - VP Program Management, MustangBIO

11:15am 11:45am (30 mins)

Commercialization

Converting a Research Paper / Process into a Clinical-Grade Cell Therapy

Converting an academic or research proof of concept (POC) study into a clinical grade cell therapy is a complex multi-step process. Successful transition from demonstrating POC to first in human clinical trials involves identifying and obtaining financial and technical support, mapping your process, and obtaining guidance for manufacturing, pre-clinical, and clinical studies.

  • Benjamin Fryer, Ph.D. - Team Leader / Cell Processing and Manufacturing, Heart Regeneration Program, University of Washington School of Medicine

10:15am 10:45am (30 mins)

Bioprocessing

Closing the Gaps in Cell Therapy Manufacturing Processes – The Adoption of Closed and Automated Systems into the Manufacturing Process

Early stage cell therapy production processes utilize open and manual manipulations from the cell isolation stage and up to final product filling. Open manipulations are operator depended and can increase the risk of contamination causing large gaps in the ability to develop a robust and cost effective product. This presentation will describe how adoption of closed system can change the “art” of growing cells into an industrial and reproducible process

  • Lior Raviv, MSc, Eng. - Vice President of Development, Pluristem Therapeutics Inc.

10:45am 11:15am (30 mins)

Bioprocessing

Adoption of an Automated Bioprocessing System

Dendritic cells play an important role in emerging cell-based cancer immunotherapies. However, their production from autologous monocytes is mostly manual and is low yielding and operator dependent. This presentation will describe a new alternative for automated dendritic cell production. The MicroDEN system employs a perfusion based approach that closely mimics the manual process but provides higher yield and consistency.

  • Sashi Murthy - Professor, Chemical Engineering, Northeastern University

11:15am 11:45am (30 mins)

Bioprocessing

Implementing Large Scale Data Processing and Analysis in Bioprocessing

In the world of bioprocessing of cell and gene therapies, numerous challenges exist. Choosing the optimal data processing and analysis technologies, and ensuring scientific leadership in this process, are both key to success. This presentation will share data strategies that not only enable leaders to take immediate action that can impact their current pipeline, but provide flexibility for the future.

  • Lisa Graham, Ph.D. - CEO and Founder, Alkemy Innovation Inc.

11:50am 12:20pm (30 mins)

Technology Workshop

The Development of Animal-Component-Free, Chemically-Defined Culture Media for Immune Cells

Cell-based immunotherapy is widely captivated due to its clinical potential. Generation of sufficient, desired cells is an essential for the success of any cell-based immunotherapy, which requires an effective ex vivo process with consistency. Case studies of how suitable chemically-defined, animal-component-free culture media for major immune cells were developed to foster a consistent bioproduction process will be shared.

  • Jessie Ni, Ph.D. - Chief Scientific Officer, Irvine Scientific

11:50am 12:20pm (30 mins)

Technology Workshop

Case Study about the Scale Up of Pluripotent Stem Cells in Single Use Bioreactors

Semma Therapeutics was founded to develop transformative therapies for patients with Type 1 diabetes. Recent work led to the discovery of a method to generate functional, insulin-producing beta cells in the laboratory. These cells develop in islet-like clusters grown from stem cells. Data for a scalable process using PBS SUBs for expansion &directed differention of pluripotent stem cells will be presented.

  • Adam Roose - Senior Research Associate, Semma Therapeutics
  • Brian Lee - President, PBS Biotech Inc.

11:50am 12:20pm (30 mins)

Technology Workshop

The critical role of automation in CAR-T cell manufacturing

Automating the manufacturing process of a cell-based therapy is not a one-time event. Rather, it is a step-by-step, highly strategic process that is guided by a deep understanding of critical process parameters and the critical quality attributes of the resulting drug product. As understanding (and confidence in efficacy) evolves through positive clinical testing, so does confidence in receiving a positive return on investment. This presentation will describe the rationale and supporting data which guided the strategic automation of the CTL019 manufacturing process and associated analytics.

  • Rodney L. Rietze, Ph.D. - Bioprocess Science and Innovation, Exploratory Immuno-Oncology, Novartis Institutes for Biomedical Research

12:25pm 1:25pm (60 mins)

Main agenda

Luncheon Presentation Available

For more information, contact: Jennifer Wickett (Companies A-L): +1.857.504.6694 • Jennifer.Wickett@knect365.comKristen Schott (Companies M-Z): +1.857.504.6685 • Kristen.Schott@knect365.com

1:40pm 1:45pm (5 mins)

Bioprocessing

Chairperson’s Remarks

  • Alaina Schlinker, Ph.D. - Manager, Cell Therapy Application Support, Fresenius Kabi USA, LLC

1:40pm 1:45pm (5 mins)

Commercialization

Chairperson’s Remarks

  • Dominic Clarke, Ph.D. - Global Product Manager, Charter Medical

1:40pm 1:45pm (5 mins)

Clinical

Chairperson’s Remarks

1:45pm 2:15pm (30 mins)

Clinical

Potency Assay Discussion Panel

  • Similarities and differences between ex vivo and in vivo
  • Different types: Cytokines, cell based assays, profilin, cytometry
  • What type of profiling should be developed to ensure the cells have the right mode of action
  • How do you validate your assays
  • Shelly Heimfield - Executive Vice President Manufacturing and Research, Nohla Therapeutics, USA
  • Linda Kelley - Senior Member & Cell Therapy Facility Director, Moffitt Cancer Center
  • David DiGiusto, Ph.D. - Executive Director, Stem Cells and Cellular Therapeutics Operations; Founding Director, Stanford Laboratory for Cell and Gene Medicine; Senior Research Scientist, Division of Pediatric Stem Transplantation and Regenerative Medicine, Stanford University
  • Christopher Bravery, Ph.D. - Director, Consulting on Advanced Biologicals Ltd, Advanced Biologicals Ltd.

1:45pm 2:15pm (30 mins)

Commercialization

Establishing and Managing an Allogeneic Therapy Supply Chain

Allogenic cell therapies do not require the just-in-time supply chain design typically needed for autologous products. This allows for substantial flexibility in supply chain design, primarily because inventories can be built in anticipation of future demand. It is therefore critical to identify what options are available and what key trade-offs exist between allogeneic cell therapy supply chain designs.

  • Tom Bevington, MBA - Director of Supply Chain, Nohla Therapeutics, Inc.

2:15pm 2:45pm (30 mins)

Commercialization

Establishing and Managing an Autologous Therapy Supply Chain

The presentation will highlight key considerations and strategies for successful design, management and execution of the supply chain related to autologous or patient specific products. Various elements of the supply chain and associated requirements will be discussed for the full spectrum of the supply chain phases. Key strategies for risk management, reduction and for addressing regulatory requisites will be reviewed.

  • Alan Smith, Ph.D. - Executive Vice President, Technical Operations, Bellicum Pharmaceuticals

2:45pm 3:15pm (30 mins)

Commercialization

Automation and Process Management Technologies for the Clinic

Thawing is one of the most critical steps for “live” therapeutics and automated mobile thawing solutions are necessary for clinical scalability. MedCision’s digitally enabled ThawSTAR® Automated Cell Thawing Systems are designed to replace uncontrolled manual thawing methods, de-risking the therapeutic cell thawing process and optimizing therapeutic cell efficacy. An industry first, ThawSTAR customizable thawing systems advance near-patient drug management by providing controlled and scalable processing of temperature-sensitive biotherapeutic products.

  • Rolf Ehrhardt, M.D., Ph.D. - President and CEO, MedCision

1:45pm 2:15pm (30 mins)

Bioprocessing

Investing in Process Development for Increased MSC Production in Stirred Tank Bioreactors

In the first case study, the impact that different gassing methods have on DO and how hypoxic growth conditions affect MSC function are examined. The second case study demonstrates the application of Zwietering’s equation for suspension of solids in stirred tanks. Identifying optimal control strategies for microcarrier-based bioreactor expansion of adherent cells is paramount for the development of a robust cell therapy manufacturing platform.

  • Julie Murelle, Ph.D. - Head of Cell Therapy Bioprocessing, MilliporeSigma

2:15pm 2:45pm (30 mins)

Bioprocessing

PANEL DISCUSSION: We Need to Talk about Particulates, Leachables and Extractables

  • Discussing strategies used for the identification and control of particulates, leachables and extractables
  • Evaluating the need for new technologies, methods and strategies
  • Addressing particulates, leachables and extractables given a lack of regulatory guidance / specifications
  • Assessing the importance come the clinic
  • Moderator Ohad Karnieli, Ph.D., MBA - CEO, Atvio Biotech
  • Panelist Jean Stanton, MSc - Director, Regulatory Compliance, Johnson & Johnson
  • Panelist Carl Burke, Ph.D. - New Platform Integration, Pharmaceutical Development & Manufacturing Sciences, Janssen
  • Panelist Jean-Sébastien Parisse, MBA - Head of Sales, Aseptic Technologies
  • Dominic Clarke, Ph.D. - Global Product Manager, Charter Medical

2:45pm 3:15pm (30 mins)

Bioprocessing

Processing Non-Mobilized Leukapheresis Products for Upstream Cell Therapy Manufacturing

Leukapheresis products often have a high platelet content which poses challenges for upstream processing steps. New data will be presented for efficiently removing platelets from non-mobilized leukapheresis products,while achieving high lymphocyte recovery and formulating the cells in the appropriate volume for immunomagnetic selection.

  • Alaina Schlinker, Ph.D. - Manager, Cell Therapy Application Support, Fresenius Kabi USA, LLC

2:45pm 3:15pm (30 mins)

Clinical

Please move to one of the other streams

3:15pm 3:45pm (30 mins)

Main agenda

Grand Opening of the BWB Exhibit & Poster Hall and Refreshment Break

3:40pm 3:45pm (5 mins)

Main agenda

Chairperson’s Remarks

3:45pm 4:15pm (30 mins)

Main agenda

Improving Global Access to Biotherapeutics Through Molecule, Process and Manufacturing Design

This presentation will discuss the application of innovative technologies designed to expand global access to biotherapeutics. These integrated technologies will assist biotherapeutic discovery through the application of molecular design tools to improve candidate molecule developability, improve process design through intensification efforts, and reduce fixed costs associated with manufacturing facility construction and operation through the design of facilities with flexible, low cost features.

  • Dean Pettit, Ph.D. - Chief Scientific Officer and Founding Partner, Just Biotherapeutics

4:15pm 4:45pm (30 mins)

Main agenda

A Vision for Rare Disease: How We Can Champion Underserved Patients

Although each rare disease affects a relatively small number of patients, together they are one of the largest under-served patient populations in the world. To make sustained progress for rare disease patients, the biopharma industry must continue to innovate and embrace new technologies to drive advances that result in faster diagnosis, better access and more effective treatments.

  • Flemming Ornskov, M.D., M.P.H. - Chief Executive Officer, Shire

4:45pm 5:15pm (30 mins)

Main agenda

Synthetic Biology: Biomedical Applications Come of Age

Synthetic biology is bringing together engineers, physicists and biologists to model, design and construct biological circuits out of proteins, genes and other bits of DNA, and to use these circuits to rewire and reprogram organisms. These re-engineered organisms are going to change our lives in the coming years, leading to cheaper drugs, rapid diagnostic tests, and synthetic probiotics to treat infections and a range of complex diseases. In this talk, we highlight recent efforts to create synthetic gene networks and programmable cells, and discuss a variety of synthetic biology applications in biotechnology and biomedicine.

  • James Collins, Ph.D. - Termeer Professor of Medical Engineering & Science and Professor, Department of Biological Engineering, Massachusetts Institute of Technology

5:15pm 7:00pm (105 mins)

Main agenda

Opening Night Cocktail Reception in the BWB Exhibit & Poster Hall