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Cell Therapy Bioprocessing

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Applying the Latest Scientific and Technical Innovations to Build the Bridge from Proof of Concept to Commercialization

September 15-16, 2014 · Sheraton Pentagon City Hotel · Arlington, VA

Conference Partner: International Society for Cellular Therapy (ISCT)

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Alternative Language Options:

  • Japanese
  • Korean
  • Taiwanese
  • Chinese
  • English

Agenda

Agenda

Sunday, September 14, 2014

3:00
Registration Opens

Welcome Networking Reception

4:00 pm – 6:00 pm

Products for Cell Therapy and Stem Cell Workflow - High-quality, Customizable Solutions You Can Trust
Timely and accurate information enables you to make high-confidence decision faster, offering the potential to reduce cycle time and production costs. Roche Custom Biotech recognizes the complex needs and challenges associated with product research, development, manufacturing, and ultimately achieving regulatory approval. We will highlight few reagents, instruments, and services that we offer that could meet your unique requirements from discovery through commercial production.
John Ogden, Ph.D., Senior Scientific Affairs Manager, Roche Diagnostics Corporation

Following Dr. Ogden's presentation will be a Welcome Networking Cocktail Reception open to all attendees

Monday, September 15, 2014

7:00
Registration and Coffee

8:00
Chairperson's Opening Remarks and Industry Update
Lee Buckler, Founder and Managing Director, Cell Therapy Group

Keynote Presentations

8:15
New Data
Developing GMP IPS Lines - Identifying and Eliminating Roadblocks
Current cell therapy models require reassessing standard manufacturing processes as well as taking into account the new changes in donor consent and tissue harvesting recently imposed while continuing to monitor cost of goods. I will use our example of manufacturing IPSC derived cell products for use in treating neural disorders and our solutions to mitigate cost as well as to develop a strategy that will be applicable for both autologous and allogeneic use models.
Mahendra Rao, M.D., Ph.D., Laboratory of Stem Cell Biology Chief, NIH

8:45
Impact of Technological Innovations on Cell Therapy Development
Cell therapy development is demanding with regard to cell number, process, regulatory requirements and cost effectiveness. Different approaches and their limitations will be discussed including the CliniMACS system, a fully automated cell processing system and a new microchip based flow sorting technology.
Stefan Miltenyi, Managing Director and Founder, Miltenyi Biotec GmbH, Germany

Lessons Learned & Future Considerations

9:15
New Data
Cell Therapy Biomanufacturing Risk Management
Cell therapy bioprocessing is replete with potential patient benefits; however, this is tempered by unique biomanufacturing risks and uncertainties. This presentation will discuss potential idiosyncratic challenges posed by cell therapies, and potential risk management tools available - including from other industries - to aid the effective translation of therapies from laboratory innovations into commercially viable patient benefits.
David Brindley, FRSA, FRI, MEng, Research Fellow, University of Oxford, CASMI, Harvard Stem Cell Institute

9:45
How Today's Manufacturing Innovations can be Leveraged by Tomorrow's Living Cell-Based Products
Technology is rapidly moving toward the integration of biologics into products like cell therapies, engineered tissues, bio-robotics, implantable devices, 3D printing, food, clothing, and even toys. This coming decade will see the incorporation of living cells into all these platforms and others not yet imagined. To expedite this biologics revolution, inventors, developers and suppliers will require a limitless, standardized, low-cost supply of cells - and today's biomanufacturing innovations are laying the groundwork to make this a reality.
Jon A. Rowley, Ph.D., Chief Executive & Technology Officer, RoosterBio Inc.

10:15
Networking Refreshment Break in Poster & Exhibit Hall

Track A

Analytical Methods & Metrology

Chairperson: Christopher A. Bravery, Ph.D., Director, Consulting on Advanced Biologicals Ltd., United Kingdom

10:45
New Data
Measurement Approaches for Assurance of Cell Therapy Products
Demonstration of a high degree of confidence in measurements is as critical for successful manufacturing and deployment of product as it is for regulatory approval. This talk will consider the characteristics of a validated assay, the challenge of developing appropriate reference materials, and how to address the issue of traceability of measurements over time and location.
Anne L. Plant, Ph.D., Chief, Biosystems and Biomaterials Division, Materials Measurement Laboratory, National Institute of Standards and Technology

11:15
New Data
Selecting Analytical Methods for Use in Cell Therapy Process Development and Product Characterization
Scientists engaged in assay development for cellular products are challenged to select the most appropriate methods for product release and adapt these assays to a quality environment. Types of assays used for product characterization and challenges associated with their validation will be discussed. QbD will be presented as a tool for characterizing complex methods towards validation.
Christopher Wiwi, Ph.D., Associate Director, Analytical R&D, Celgene Cellular Therapeutics

11:45
Ability to Detect Failure: Appropriateness and Capability of the Cedex Bio Analyzer in Media Optimization
Abstract not available at time of print.
David Scharp, MD, President and CEO, Prodo Laboratories, Inc.

Track B

Autologous Immunotherapies & Gene Therapies

Chairperson: Lee Buckler, Founder and Managing Director, Cell Therapy Group

10:45
Case StudyNew Data
Angiogenic Cell Precursors (ACP-01)-Development of an Autologous Cell Therapy Product
This presentation will focus on factors essential to the development of a quality cell therapy product, including: focus on the target product from the start of the development process, thorough understanding of the biological characteristics of the product, and tight control of the manufacturing process to ensure consistency of production.
Ina Sarel, Ph.D., Vice President, Research & Development, Hemostemix

11:15
New Data
Gene-Editing of Autologous Cells for the Treatment of Disease
Zinc-finger Nuclease-guided genomic editing is a highly specific and well tolerated methodology for modification of several primary cell types (T cells and Stem cells), as revealed by examination of the stability, growth, in vivo engraftment, and functional characteristics of the genome-modified sub-population, even in the absence of selection. This methodology is readily adaptable to a robust clinical scale cell processing system and applicable to a variety of disease indications.
Martin Giedlin, Ph.D., Vice President, Development, Sangamo Biosciences Inc.

11:45
New Data
New Tricks with Old Tools - Applying Cell Culture Engineering Tools for Cell Therapy Manufacture
Many existing tools and technologies from biopharma are applicable to cell therapy manufacture, but are often overlooked. This seems to be driven by a lack of familiarity or perceived complexity of application to cell therapies. In reality, these tools/technologies are necessary to the industrialization of cellular therapies, and with a bit of tweaking and insight, can be effectively adapted without re-inventing the wheel.
Nick Timmins, Ph.D., Director, Product and Process Development, Centre for Commercialization of Regenerative Medicine (CCRM)

12:15
Networking Luncheon in the Poster & Exhibit Hall

Track A

1:25
Chairperson's Remarks
Ron Fedechko, Associate Research Fellow, Pfizer

Quality Assurance & Control

1:30
Measurement Reliability Over the Product Lifecycle: The Need for Reference Materials
Successful demonstration of manufacturing and quality consistency is dependent on the use of often complex analytical methods, thus the risk of process and method drift over time is high. The use of reference materials is an established scientific principle and as such also a regulatory requirement; their various uses in the context of CTP manufacturing and quality will be discussed.
Christopher Bravery, Ph.D., Director, Consulting on Advanced Biologicals Ltd., United Kingdom

2:00
Case Study
Demonstrating Control of Autologous Cell Therapy Processes Throughout the Validation Lifecycle
Proving effective control of autologous cell therapy manufacturing processes is complicated by limitations in our ability to characterize such products. Furthermore, performing conventional Process Performance Qualification (PPQ) activities are difficult due to the inherent biologic variability and complexity of the incoming starting material and the resulting drug product. These challenges necessitate development of a new approach to demonstrate process control. This presentation will discuss a case study for a genetically modified autologous cell therapy product. The unique challenges and proposed validation strategy utilizing all three stages of the process validation lifecycle will be described.
Tristan Marshall, Biopharm Product Leader, Biopharm & Steriles Center of Excellence, GlaxoSmithKline

2:30
Overcoming Leachable/Extractable Issues, Lessons Learned and Examples from the Protein Manufacturing Arena how that Knowledge can be Applied to Cell Therapy
Whereas Extractables and Leachables (E&L) have been characterized for decades in support of therapeutic-protein manufacturing, the concepts are comparatively untapped in Cell Therapy. This presentation explores ways in which E&L concepts may be applied to Cell Therapy, by drawing analogy to the ways in which they are used to characterize product and process in therapeutic-protein manufacturing.
Jeffrey Carter, Ph.D., Strategic Projects Leader, Life Sciences, GE Healthcare

3:00
Target Product Profile(TPP) Tool to Drive Product/Process Development
The Target Product Profile (TPP) is the preferred document to capture the aspirational attributes of a therapeutic. It may be used both as a framework for regulatory discussions and as a guide to product development. The specifics of authoring a TPP for a cell or gene therapy will be presented in a hands-on manner.
Anthony Davies, Ph.D., President, Dark Horse Consulting, Inc.

Track B

1:25
Chairperson's Remarks
Jason N. Carstens, Ph.D., Director, Process Development, Fred Hutchinson Cancer Research Center

Autologous Immunotherapies & Gene Therapies (continued)

1:30
New Data
Chimeric Antigen Receptor T Cells Induces Durable and Complete Clinical Responses in B Cell Malignancies
Inserting nucleic acids encoding chimeric antigen receptors to T cells can redirect specificity to tumor antigens. Significant and durable clinical responses in leukemia have been observed in patients who are relapsed or refractory to all other available treatment, including patients who have failed allogeneic stem cell transplantation. Ex vivo modified cells can engraft, significantly expand in vivo by 1000 fold, and survive for years.
Bruce Levine, Ph.D., Barbara and Edward Netter Associate Professor in Cancer Gene Therapy, University of Pennsylvania

2:00
New Data
Issues Related to Commercial Scale Production and cGMP Compliance for Autologous Cell Therapies
Compared to traditional drugs where a bulk of drug substance is produced and dispersed to several individual patients, autologous cell therapy products are derived from the patient's own cells. With this, a unique manufacturing scenario exists where each patient equals one manufacturing batch. Thus, the commercialization strategy for such products needs to consider several unique issues. This presentation will discuss some of these issues in light of establishing a large market for an autologous therapy.
Knut Niss, Ph.D., Senior Technical Project Leader, Global Biopharm Operations, Novartis

2:30
New Data
Gene Modified T Cell Therapies for Lymphoma: A Bench to Bedside Approach
Lymphomas utilize multiple immune evasion strategies to evade host T-cell responses. These include limited expression of antigens and production of immunosuppressive cytokines. Using gene transfer strategies we have developed novel immunotherapeutic T-cell approaches. These therapies are well tolerated and can induce durable clinical responses. Given these promising results, research is now focused on broadening applicability and moving beyond phase I.
Catherine M. Bollard, MBChB, MD, FRACP, FRCPA, Senior Scientist, Center for Cancer and Immunology Research, Children's Research Institute

3:00
Spotlight Presentation to be Announced
For more information on spotlight presentations, please contact Kristen Schott at (508) 614-1239 or kschott@ibcusa.com

3:30
Networking Refreshment Break in Poster & Exhibit Hall

Process Development for Commercial Success

4:00
New Data
Process Development and Engineering Challenges to Commercializing Allogeneic Cell Therapies
The process development of cell-based therapy presents unique challenges compared to small molecules and traditional biologics. This talk will discuss some unique challenges, including: 1. Development of a robust bioreactor harvest process, 2. Particulates control and characterization of drug products, 3. Design of filling process for cryopreserved products at large scale.
Li Ren, Ph.D., Associate Director, Bioprocess Development, Celgene Cellular Therapeutics

4:30
New Data
Developing Immunotherapies: Where We Are Headed
Process development for cellular therapies is an iterative process that involves validations, qualifications, clinical trials, basic and translational research, and technological advances. This presentation uses individual examples to demonstrate how basic advances in cell manufacturing were translated to early phase clinical studies, and in some cases, beyond.
Patrick Hanley, Ph.D., Director of GMP for Immunotherapy, Assistant Professor, Program for Cell Enhancement and Technologies for Immunotherapy (CETI), Children's National Health System

5:00
New Data
New Cell Washing Concept Utilizing Spinning Membrane Device
Cell washing is an essential step for any cell therapy processing. A new cell washing concept is being introduced utilizing spinning membrane technology. Spinning membrane technology creates Taylor Vortex flow in the filtration area to enhance the membrane performance. An instrument that utilizes spinning membrane technology can replace the media and volume reduce the product efficiently. New data from this spinning membrane washing instrument will be discussed.
Augustin Min, Ph.D., Senior Director, Research and Development, Fresenius KABI

5:30
Poster & Exhibit Hall Reception Sponsored by

6:30
Evening Networking Reception Sponsored by

Tuesday, September 16, 2014

7:30
Coffee

8:00
Chairperson's Opening Remarks
Jon A. Rowley, Ph.D., Chief Executive & Technology Officer, RoosterBio Inc.

Featured Presentation

8:15
Case Study
World's First Approved Allogeneic Cell Product CARTISTEM®: Two Year Market Update and Global Perspective
CARTISTEM® was approved with a Biologics License Application(BLA) by Ministry of Food and Drug Safety(MFDS) Korea in 2012 as the world's first approved allogeneic cell product for the treatment of degenerative osteoarthritis. Since the product launch, over 1,000 patients have been treated on market in Korea. CARTISTEM®'s market experience and blobal commercialization efforts and strategy will be shared.
Anthonio (Tony) Lee, Chief Executive Officer & Managing Director, MEDIPOST America Inc.

Scale Up & Scale Out Case Studies

8:45
Case StudyNew Data
Manufacturing and Clinical Development of MultiStem - A Drug-Like Cell Therapeutic
During the past decade, the number of cell therapies under evaluation in clinical studies has increased dramatically. In many instances, the manufacturing process used early in clinical development is not feasible for commercial production, due to high cost of goods, lack of scalability, supply chain constraints, and regulatory requirements. In this presentation, key scale up considerations aimed at producing a commercially successful product will be discussed.
John Harrington, Ph.D., Chief Scientific Officer, Athersys, Inc.

9:15
Case Study
Generation of Therapeutically-Active Cells using Microcarrier-Based Expansion in Bioreactors
Microcarriers used in conjunction with bioreactors provide an ideal platform for the expansion of these stem cells. Their benefits include increased process control and reproducibility in bioreactors with a reduced footprint. In this presentation, feasibility studies demonstrating that large numbers of undifferentiated ASCs can be grown on SoloHill microcarriers under a variety of conditions and efficiently harvested will be discussed. Results from studies designed to scale processes to intermediate-sized reusable and single-use stirred-tank reactors will also be presented.
Mark Szczypka, Ph.D., Senior Director Applications and New product Development, Pall Life Sciences

9:45
Networking Refreshment Break in Poster & Exhibit Hall

Track A

Chairperson: Richard Grant, Senior Vice President, Cell Therapy, Invetech

Overcoming Manufacturing Challenges

10:15
Case Study
Commercial Scale Manufacturing; The Heartbeat of the UK Cell Therapy Catapult
For cell therapy to become a truly mature industry, the focus will need to shift from discovery science to manufacturing science. In addition to safety and efficacy considerations, the main driver behind process/manufacturing development should be based on Cost of Goods and price point. With changes in global reimbursement strategies Cost of Goods, overhead rate and failure rate are going to play a more important part in driving manufacturing science; this will mean changes in current clean room design, process efficiency, yield, product potency and end to end supply chain thinking. Manufacturing security is of even higher importance than in comparator biological industries. Details on the UK's strategic manufacturing capability investments shall be given.
Stephen Ward, Ph.D., Chief Operating Officer, Cell Therapy Catapult, United Kingdom

10:45
Case StudyNew Data
Overcoming Manufacturing Challenges for T Cell therapy
We are developing a product portfolio employing engineered autologous T cell therapy (eACT), designed to harness a patient's own immune system to eradicate cancer cells. Manufacturing success requires careful development of robust processes, complex relationships with dependable suppliers and well trained production organizations. As T cell therapies mature toward commercialization, cost-effective solutions must be developed to overcome challenging manufacturing barriers.
Marc Better, Ph.D., Vice President, Product Sciences, Kite Pharma, Inc.

11:15
Case StudyNew Data
The Road Not Taken…. Moving Cell Therapy from the Benchtop to an Industry
The need for large quantities of cells with high quality becomes crucial as more therapies move in to clinics. High quantities of cells opens new questions and challenges of cell quality, identity, reproducibility and cost. Bioreactor technology brings to the cell therapy industry the opportunity to manufacture large quantities of cells in a tightly controlled and monitored manner, lowering relatively the cost of goods manufactured resulting in a product and process for cell therapy commercialization. The talk will describe some of the main challenges accounted during the development of the GMP manufacturing technology for PLX (PLacental eXpanded) cell product candidates and some of the bottlenecks opening methods developed in Pluristem's pilot and manufacturing facilities.
Ohad Karnieli, Ph.D., MBA, Vice President, Development & Manufacturing, Pluristem Therapeutics, Inc.

11:45
Late-Stage Clinical Trials: Activities to Consider When Preparing for Commercial Launch
Over the last few years, several cell therapy developers have been challenged with the successful commercialization of their products. In recognizing these challenges and leveraging our experience in the biologics and small molecule fields, Lonza has identified key gaps in cell therapy development that should be addressed prior to commercial launch. Supply chain, logistics, manufacturing strategies, and analytical development will be discussed.
Jessica Carmen, Ph.D., Business Development Manager, Cell Therapeutic Services, Lonza Walkersville, Inc.

Track B

Chairperson: Jon A. Rowley, Ph.D., Chief Executive & Technology Officer, RoosterBio Inc.

Bioengineering, Tissue Engineering & Bioprinting

10:15
New Data
Engineering Stem Cell Microenvironments for Scalable Manufacturing
Stem cell technologies represent potent regenerative therapies and novel platforms for drug discovery and development if scalable methods of controlling stem cell differentiation can be achieved and broadly implemented. Biochemical and biophysical properties of stem cell environments regulate cell fate decisions, thus engineering 3D environmental parameters affords a robust approach to direct differentiation efficiently and effectively.
Todd C. McDevitt, Ph.D., Professor, Biomedical Engineering; Director, Stem Cell Engineering Center, Georgia Institute of Technology

10:45
The Path to Large Scale Biomanufacturing of Cultured Leather
Abstract not available at time of print.
Hemanthram Varadaraju, Process Engineer, Modern Meadow, Inc.

11:15
New Data
Bio-Pick, Place and Perfuse: A New Instrument for Layer by Layer 3D Tissue Engineering
By adapting the principles of pick and place machines used in the high speed assembly of electronics, we've developed a new instrument that picks up large multi-cellular building parts, stacks the parts and perfuses the stack of parts. Scaffold-free multi-cellular building parts are self-assembled within 24 hours in nonadhesive micro-molds that dictate part shape (spheroids toroids, honeycombs). Assembled parts fuse within 48 hours to form a large contiguous tissue construct.
Jeff Morgan, Ph.D., Professor of Medical Science and Engineering, Brown University

11:45
New Data
Jackin' up MSCs! - Engineered Control of MSC Fate and Function Post Translation
This talk will explore technologies that are currently being developed to control cell fate and function following transplantation. Namely, an mRNA transfection strategy using cells for targeted delivery of biologics will be discussed and approaches will be covered to engineer cells with an intracellular depots of phenotype altering agents that can be used for drug delivery or programming cell fate via both intracrine-, paracrine-, and endocrine-like mechanisms.
Jeffrey M. Karp, Ph.D., Associate Professor, Brigham and Women's Hospital

12:15
Networking Luncheon in the Poster & Exhibit Hall

1:25
Chairperson's Remarks
Aby J. Mathew, Ph.D., Senior Vice President & Chief Technology Officer, BioLife Solutions, Inc.

Biopreservation and Product Stability Considerations for GMP

1:30
Fundamentals of Biopreservation - Stability Issues for Cryopreservation/Cryobiology
Abstract not available at time of print.
Shelly Heimfeld, Fred Hutchinson Cancer Research Center

2:00
Case StudyNew Data
Cell Cryopreservation and Reconditioning for Clinical Application
Cardio3 BioSciences (C3BS) is a Belgian biotechnology company that had developed a GMP therapy based on autologous bone marrow-derived and cardiogenically oriented mesenchymal stem cells. In order to administrate to the patient the final cell suspension product, Cardio3 BioSciences developed a cryopreservation-reconditioning process, allowing to extended shelf-life, eliminate logistics problems and make commercialization success far more attainable.
Peter de Waele, Ph.D., Vice President R&D, Cardio3 BioSciences, Belgium

2:30
Case StudyNew Data
Cost-Effective Standardizing Cryopreservation of Cell Therapy Products
Successful cryopreservation depends upon the proper rate of temperature decrease, followed by long-term storage in liquid nitrogen. When cryopreservation steps are applied in cell-based clinical studies, any variability in this process may have severe downstream consequences to the clinical outcome. We describe a cost effective, scalable alternative to the programmable freezer and demonstrate its successful adoption into a clinical cell therapy trial.
Rolf Ehrhardt, Chief Executive Officer, BioCision

3:00
Biopreservation and Biologistics - Points of Risk in the Cell/Tissue Life Cycle
The clinical and commercial success of cellular therapies and regenerative medicine products is potentially impacted by stability limitations, which include transport of the source material and biopreservation of the final cell or tissue product (either frozen or non-frozen). This discussion will offer methods for optimizing stability/shelf-life, and explore the critical role and impact of biopreservation in the development and commercialization of regenerative medicine products, as well as discuss points of risk within a GMP clinical manufacturing and product delivery process including biologistics.
Aby J. Mathew, Ph.D., Senior Vice President & Chief Technology Officer, BioLife Solutions, Inc.

3:30
Networking Refreshment Break in Poster & Exhibit Hall

Ensuring Control, Safety and Supply of Raw Materials

4:00
Building an Effective Control Strategy for Raw Materials Used in Cellular Therapy Manufacturing
This presentation will give an insight into how such a control strategy can be developed specifically for raw material selection and use, including what must be achieved to reach the desired end, how to ensure the desired end is reached, tools used during the assessment phase of risk management, and control choices that can be made during the management of risk.
Jean Stanton, M.S., Director, Regulatory Compliance, Johnson and Johnson

4:30
Challenges and Solutions for Sourcing Human Serum or Animal Serum
Dealing with Supply Challenges... ask your team lots of questions. Minimum and maximum amount of serum needed? Recruiting and screening human donors - Ins & Outs of a Plasma Collection Center, policies and procedures for the collection facility, including the safety components. Overview of the Human & Bovine Serum Manufacturing Process. Ensuring adequate future supply - Partner with your supplier. Share your expected needs.
Tom Pecora, Eastern U.S. Sales Director, Gemini Bio-Products

5:00
Close of Conference

Workshop and Innovation Session

Filling the Gap in Cell Therapy Manufacturing Technologies

1:30 pm – 3:30 pm

New on the agenda this year, is an industry workshop that provides the opportunity to attendees to roll-up their sleeves and interact with their peers to generate discussions and new ideas to well-known challenges in the CT manufacturing. Skilled Invetech facilitators will work with the participants to identify gaps in process technologies and generate novel ideas and equipment concepts to address these challenges. The workshop goal is to create concepts with realizable potential while attendees also gain an introduction to some of Invetech's innovation tools and processes.

Workshop learnings include:

  • Identifying translational process gaps
  • Requirements generation
  • Idea generation
  • Concept development
  • Concept evaluation/trade off
  • Understanding of development speed and cost

Invetech Facilitators:
Richard Grant, Senior Vice President, Cell Therapy, Invetech
Geoff Ball, Program Manager, Cell Therapy, Invetech
Brian Hanrahan, Program Manager, Cell Therapy, Invetech
Bob Speziale, Vice President, Business Development, Invetech

Workshop participation is by enrollment and is strictly limited to 48 attendees.