What are the next steps for the cell and gene therapy sector in terms of driving progress in product characterization?

11.40

Regulator's perspective
Knowledge is power - what are the next steps on the path to cell and gene therapy product characterization?

• Product characterization is critical to successful product development
• Minor components in a product can have a big impact
• Understanding product composition and product attributes related to potency are necessary parts of product characterization
• Developing the technology and/or other strategies to characterize products

Dr Denise K. Gavin, Division of Cellular & Gene Therapies (DCGT), Office of Cellular, Tissue & Gene Therapies (OCTGT), CBER, US FDA

12.00

Questions & discussion

12.05

Product characterization tools

• An overview of the growing array of technologies at therapeutic developers' disposal
• Cell differentiation, intracellular characterization, functional assays
• How such tool should be validated
• Tying characterization to mechanism of action

Dr Robert A. Preti, President & Chief Scientific Officer, Progenitor Cell Therapy, LLC

12.25

Questions & discussion

12.30

Cell therapy
Presentation & panel discussion
What is the impact of impurity on cellular product characterization and what can be done about it in practical terms?

• Exploring FDA requirements for cell purity and its impact on potency and identity of the actual therapeutic product
• What technologies are currently available to assist in further defining the therapeutic cell population?
• Given the current lack of standards in terms of cell therapy QC, what is the minimum that cell therapy companies can/should do with regard to QC and when?

Speaker:
Dr Joyce Frey-Vasconcells, Executive Director, PharmaNet

1.10

Buffet lunch in the exhibition area

OR | Lunch Briefing Sponsored by
How non-invasive, in vivo imaging of transplanted cells is expected to accelerate the pace of cell therapy development, generate critical safety data and improve clinical outcomes

This session will outline which cellular imaging technologies are currently available for pre-clinical and clinical use, the anticipated costs and benefits of incorporating an imaging protocol into scientific discovery and/or clinical trials, and the anticipated impact of clinical imaging data on discovery and preclinical research, regulatory submissions, and clinical protocols

Chair:
Charles F. O'Hanlon, President & CEO, Celsense, Inc
Speakers:
Robert Deans, PhD, Senior Vice President, Regenerative Medicine, Athersys, Inc
Eric T. Ahrens, PhD, Associate Professor. Deparment of Biological Sciences, Carnegie Mellon University
Lydia Martynec, MD, Medical Officer, Office of Cellular, Tissue, & Gene Therapies, Division of Clinical Evaluation & Pharm/Tox, CBER , FDA(For a maximum of 50 participants)

»Click here for more information

Cell and gene therapy industry case studies exploring challenges in the scaling of processes from GLP to GMP, and then from pilot to commercial scale

• What can/should you do to optimize your product as you progress through R&D? How do you keep your optimization moving forward as you advance through each stage?
• How to scale up clinical production to commercial scale whilst maintaining product comparability?
• Clarifying the differences between Phase I GMP requirements and those for later phases: How must you adapt?

2.20

Cell therapy
Production of cell therapies from clinical trial to commercial distribution

• Examples of how to establish GMP production, validation programs and quality testing for clinical trials using cellular therapies will be discussed
• Genzyme currently manufactures three cell therapies for commercial distribution worldwide, details will be given on the production and quality processes that need to be established will be reviewed

Leslie Wolfe, PhD, Vice President, Technology Development, Cellular Therapies, Genzyme

2.40

Questions & discussion

2.45

Gene therapy
Optimizing production of DNA nanoparticles for various stages of product development – taking the process from the research lab through early stage clinical to large scale clinical non-viral gene therapy applications

• Advantages of non-viral gene therapy manufacture process during the scale-up process
• Understanding production concerns at a research batch scale level
• Applying research batch scale knowledge to larger scale production suitable for early stage clinical trials
• Scaling up production via a continuous flow manufacturing system
• Closed system manufacturing versus open system manufacture for cGMP
• From grams to kilograms – can the same process work for vastly different scales?

Robert C. Moen, MD, PhD, President & CEO, Copernicus Therapeutics

3.05

Questions & discussion

3.10

Considerations and examples for automation and commercial scale-out of patient-specific cell therapy manufacturing

• Scale-out strategy and contrast to multiple patient process
• Automation drivers and contrast to conventional drug/biologic process
• Choices for automation
• Example approach
• Financial and regulatory considerations
  

Brian S. Hampson, Senior Engineering Fellow, Aastrom Biosciences, Inc

3.30

Questions & discussion

3.35

Moderator’s closing summary

3.40

Close of session followed by afternoon tea in the exhibition area

11.45

The essentials of commercializing a cell therapy: Start at the end and work your way back

• The importance of connecting the dots (define molecular through clinical pathology, define Mechanism of Action, define therapeutic, and define distribution and reimbursement)
• Clinical trial development (the importance of early assessment of efficacy)
• Commercialization (who will use it, how will they use it, who will make it and how will they distribute it, and who will pay for it and why)

Dr Andrew L. Pecora, Chairman of the Board, Amorcyte

12.10

Questions & discussion

Case studies
Cell therapy trial designs and strategies to identify and demonstrate potency and Mechanism of Action at each phase of clinical development

• What technologies are out there that can improve the efficacy of a product candidate in terms of driving our understanding of its MoA?
• How to gauge what the regulator wants/needs to see in terms of MoA data and then build this into your clinical trial design

12.15

Case study
Harnessing dendritic cells for cancer vaccination

• Dendritic cells can be derived in vitro from peripheral blood leucapheresis products or from human embryonic stem cells
• Electroporation of dendritic cells with RNA can create antigen presenting cells capable of raising a peptide-specific immune response
• Vaccination of patients with manipulated (electroporated) dendritic cells may induce low-level yet potentially clinical significant immune responses in vivo
• Questions still exist around specification for release of cellular therapies for human administration, the dose required for therapeutic effect, choice of antigen, and monitoring of responses
  

Stephen M. Kelsey, MD, Executive Vice President & Chief Medical Officer, Oncology, Geron Corporation

12.40

Questions & discussion

12.45

Case study
Explaining Phase I and II clinical observations: Utilizing product characterization and laboratory assays to develop potency assays and formulate Mechanisms of Action

• Product characterization from preclinical to Phase I
• Correlating product characterization, and biological activity with proposed Mechanisms of Action
• Selecting potency assay candidates from Phase I and Phase II data
• MoA and potency - addressing licensing and Phase III needs

Speaker to be announced

1.05

1.10

Buffet lunch in the exhibition area

OR | Lunch Briefing Sponsored by
How non-invasive, in vivo imaging of transplanted cells is expected to accelerate the pace of cell therapy development, generate critical safety data and improve clinical outcomes

This session will outline which cellular imaging technologies are currently available for pre-clinical and clinical use, the anticipated costs and benefits of incorporating an imaging protocol into scientific discovery and/or clinical trials, and the anticipated impact of clinical imaging data on discovery and preclinical research, regulatory submissions, and clinical protocols

Chair:
Charles F. O'Hanlon, President & CEO, Celsense, Inc
Speakers:
Robert Deans, PhD, Senior Vice President, Regenerative Medicine, Athersys, Inc
Eric T. Ahrens, PhD, Associate Professor. Deparment of Biological Sciences, Carnegie Mellon University
Lydia Martynec, MD, Medical Officer, Office of Cellular, Tissue, & Gene Therapies, Division of Clinical Evaluation & Pharm/Tox, CBER , FDA(For a maximum of 50 participants)

»Click here for more information

2.20

Case study
Phase III case history: Development of Sipuleucel-T, an active cellular immunotherapy for prostate cancer

• Introduction to Sipuleucel-T
• Characterization of the product and development of the potency assay
• Clinical outcomes and correlation with product parameters

Dr David Urdal, Chief Scientific Officer, Dendreon Corporation

2.40

Questions & discussion

2.45

Presentation & panel discussion
Defining the preclinical and clinical utility of the latest cell labelling and tracking technologies

• What technologies are available?
• What are their relative pros and cons in terms of cost and potential benefit in providing biodistribution and cell fate data?
• In what indications are they being implemented, and with what effect on preclinical and clinical development timelines and on clinical endpoints?

Dr Dara Kraitchman, Associate Professor, Russell H. Morgan Department of Radiology & Radiological Science, Johns Hopkins School of Medicine

3.35

Moderator’s closing summary

3.40

Close of session followed by afternoon tea in the exhibition area

11.35

Workshop 1: Preparing for 'first in man' studies on gene and cell therapy products: Quality and nonclinical requirements for a Phase I Clinical Trial Application

• Regulations and guidelines: Which are helpful and what needs to be taken into account?
• Interacting with regulators at an early stage to validate quality and nonclinical requirements
• Where to conduct the 'first in man study', the US, Europe or elsewhere?

1.05

Moderator’s closing summary

1.10

Buffet lunch in the exhibition area

OR | Lunch Briefing Sponsored by
How non-invasive, in vivo imaging of transplanted cells is expected to accelerate the pace of cell therapy development, generate critical safety data and improve clinical outcomes

This session will outline which cellular imaging technologies are currently available for pre-clinical and clinical use, the anticipated costs and benefits of incorporating an imaging protocol into scientific discovery and/or clinical trials, and the anticipated impact of clinical imaging data on discovery and preclinical research, regulatory submissions, and clinical protocols

Chair:
Charles F. O'Hanlon, President & CEO, Celsense, Inc
Speakers:
Robert Deans, PhD, Senior Vice President, Regenerative Medicine, Athersys, Inc
Eric T. Ahrens, PhD, Associate Professor. Deparment of Biological Sciences, Carnegie Mellon University
Lydia Martynec, MD, Medical Officer, Office of Cellular, Tissue, & Gene Therapies, Division of Clinical Evaluation & Pharm/Tox, CBER , FDA(For a maximum of 50 participants)

»Click here for more information

2.20

Workshop 2: Beyond Phase I: How to prepare for a successful clinical trial programme on a gene or cell therapy product

• Regulations and guidelines: What is applicable for a gene or cell therapy product, especially targeted at an 'unmet clinical need'?
• Interacting with regulators beyond Phase I to develop a successful clinical trial program
• Towards a marketing authorization application: Examining successes and failures (case studies)

3.35

Moderator’s closing summary

3.40

Close of session followed by afternoon tea in the exhibition area

4.25

US FDA perspective
FDA regulation of cellular, tissue, and gene therapies

• Overview of Office and regulation
• Update on recent guidances and meetings
• Challenges in regulation of cellular, tissue, and gene therapies

Dr Celia M. Witten, Director, Office of Cellular, Tissue and Gene Therapies (OCTGT), CBER, US FDA

Update on the European Committee for Advanced Therapies (CAT) on its first birthday: What are the experiences to date from both CAT and industry perspectives?

4.45

Regulator's perspective
First experiences from the CAT's work

• How the CAT operates
• Challenges with Advanced Therapie
• How the CAT interacts with stakeholders

Dr Christian K. Schneider, Head of Division of EU Cooperation/Microbiology, Paul-Ehrlich-Institut & Chair, EMEA Committee for Advanced Therapies (CAT)

5.00

Industry perspective
EU approval of ChondroCelect - meeting the regulatory requirements for a cell- based product

• EU regulatory framework
• Challenges in meeting the classical CMC requirements
• Clinical trial design
• Benefit – Risk assessment

Dr Wilfried Dalemans, CTO & Vice President, Regulatory Affairs, TiGenix NV

5.10

Panel discussion

• New EU post-market safety surveillance legislation: What are the chief considerations for cell therapy, gene therapy and tissue engineered product companies?
• What are the key areas of disharmonization between North American and European cell and gene therapy regulatory processes and standards?
• Clarifying the roles moving forward of non-regulator overview authorities such as the RAC and their processes by which they interface and harmonize with the regulators
  • What do the RAC see as their role as more gene therapy clinical trials move to Phase III?

Panellists:
Michael J. Werner, Partner, Holland & Knight
Dr Jacqueline Corrigan-Curay, Acting Director, Office of Biotechnology Activities, National Institutes of Health

5.50

End of day 2 followed by a themed cocktail reception in the exhibition area