Cambridge Healthtech Institute’s Inaugural
Cell Therapy Bioproduction
Addressing the Development, Manufacturing and Scale Up/Out Dilemma
Part of CHI's 6th Annual The Bioprocessing Summit
August 21-22, 2014 | Renaissance Waterfront Hotel | Boston, Massachusetts
Day 1 | Day 2 | Short Courses | Download Brochure | Speaker Bios
Friday, August 22
8:00 am Registration and Morning Coffee
8:25 Chairperson’s Remarks
Mark Angelino, Ph.D., Vice President, Pharmaceutical Sciences, bluebird Bio
8:30 Translating a Research Methodology into a Mechanism of Action Based Validated Potency Assay
Sagi Nahum, Ph.D., QC Manager, Pluristem Therapeutics, Inc.
Validated potency assays aims to measure or predict the expected therapeutic mechanism of action (MOA). Clinical data may be used to establish a correlation between potency assays allowing lot release and stability. Most of the potency assays emerge from research and academy transforming to potency assays after a long journey of validation. The talk will describe the path and challenges of bioassay development from research to QC based on Pluristem Therapeutics experience.
9:00 Autologous Lots of Cell Therapy Products: Potency Defined by Commonality
Don Healey, Ph.D., CSO, Opexa Therapeutics
Autologous lots of cell therapy products invariably display differences in either phenotype and/or genotype, as may be a requirement to meet their intended mechanism of action on a per patient basis. Nevertheless, potency can be defined based on biological features that must be held ‘in common’ between products that achieve the intended clinical benefit. The development of potency assays should be multi-factorial in the first instance, and should be initiated early in the process development timeline.
9:30 Critical Quality Attributes (CQA) Identification for a Cell-Based Gene Therapy Product, Approaches to Selection and Validation of Release Test Methods
Bernadette Keane, BSc., Vice President, Quality, Bluebird bio
In the emerging fields of cellular and gene therapy, control strategies are not yet well defined and pose their own set of challenges due to inherent variability of living systems. Using examples from autologous cell based gene therapy products, the presenter will discuss approaches to validation of test methods employed in the release of cell based gene therapy products.
10:00 Mid-Morning Snack in the Exhibit Hall with Poster Viewing
10:45 Rapid Microbiological Methods to Enhance Safety Profile of Cell-Based Therapeutics
Gary C. du Moulin, Ph.D., MPH, RAC, former Senior Director, Quality Aseptic Controls, Genzyme (a Sanofi Company)
Cell-based therapeutics has accelerated Rapid Microbiological Methods implementation. In 2004, the United States Food and Drug Administration (FDA) approved an RMM for lot release of Genzyme’s cell therapy product, Carticel®. Globalization of pharmaceutical quality systems, which emphasizes risk assessment and continual improvement of manufacturing processes has further accelerated acceptance of these technologies. RMM have the ability to identify microbiological risks, monitor critical control points in real time thus enhancing the safety profile of cell therapy products.
11:15 Scale-Up and Optimization of an Allogeneic Cell Therapy Process
Hari Kamaraju, Ph.D., Senior Associate Scientist, Pharmaceutical Development & Manufacturing Sciences, Janssen Research & Development
11:45 Optimization of T Cell Production Process for Adoptive T Cell Therapy
Pranay Khare, Ph.D., Director, Research, Cancer Immunotherapy and Gene Therapy cGMP Facility, Roger Williams Medical Center
Adaptive T cell therapy has showed promising results in several clinical trials for leukemia, but limited success has been achieved in solid tumors. Almost all clinical studies have used interleukin-2 as primary cytokine for the T cell production and expansion process. This talk will focus on the optimization protocol for T cell production process using other common gamma-chain family cytokines of T cell, and explore the therapeutic response of T cells for solid tumors using adoptive T cell therapy approach.
12:15 pm Q&A with Speakers
12:30 Luncheon Presentation: Single-Use Expansion and Harvest of Adult Stem Cells Supports Large-Scale Manufacturing
Julie R. Murrell, Ph.D., Program Manager, Collaborations; R&D Manager, EMD Millipore Corporation
As more stem cell therapeutics progress through clinical testing, current in vitro culture methods are cumbersome to scale. Here we describe a case study for full expansion, harvest and characterization system for hMSCs. In this work, we verified that cells expanded in the single-use stirred tank bioreactor and subsequently harvested were identical in phenotypic and genotypic profile in comparison to flat culture and maintained the desired cell characteristics of hMSCs, thereby confirming the consistency, quality and reproducibility of large-scale in vitro systems for stem cell expansion.
1:55 Chairperson’s Remarks
Ravinder Bhatia, Associate Director, Pharmaceutical Development and Manufacturing Sciences, Johnson & Johnson
2:00 Strategy to Commercialize Autologous Cell Therapies
Knut Niss, Ph.D., Senior Technical Project Leader, Novartis Pharmaceuticals Corp.
Commercialization of an autologous cell therapy product requires consideration of several unique issues. In particular, logistics of product distribution, manufacturing strategy and supply chain requirements are critically different from traditional drug development or allogeneic cell therapies. This presentation will discuss some of these issues in light of establishing a large market for an autologous therapy.
2:30 From Development through Approval of An Autologous Cell Therapy
Stephen J. Duguay, Ph.D., Associate Director, Process Development, Aastrom Biosciences
3:00 Refreshment Break
3:15 The Road Not Taken… Moving Cell Therapy from Benchtop to an Industry
Ohad Karnieli, Ph.D., MBA, Vice President, Development and Manufacturing, Pluristem Therapeutics, Israel
The need for large quantities of cells with high quality becomes crucial as product candidates advance into clinical trials. Technologies are evolving to allow production of large quantities. Nevertheless, high quantities of cells opens new questions and challenges of cell quality, identity, reproducibility and cost. The talk will describe the development of the GMP manufacturing technology for PLX (PLacental eXpanded) cell product candidates and some of the bottlenecks encountered in Pluristem’s pilot and manufacturing facilities.
3:45 Towards Bioengineered Control of Cell Fate Post Transplantation
Jeffrey M. Karp, Ph.D., Associate Professor of Medicine, Brigham and Women’s Hospital, Harvard Medical School
Control of cell fate and its extracellular environment following transplantation is critical for maximizing efficacy of cell based therapy. This talk will explore methods to enhance the engraftment and tracking of systemically infused stem cells through engineering the cell surface and through functionalizing cells with contrast agents and depots containing phenotype altering agents.
4:15 End of Conference
Day 1 | Day 2 | Short Courses | Download Brochure | Speaker Bios