Welcome To the Phacilitate Cell & Gene Therapy Forum 2010

Agenda | Day 3 Printer friendly version - full agenda

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DAY 3 \| Wednesday, January 27th 2010
7.30	Registration & buffet breakfast in the exhibition area
OR	Breakfast workshop sponsored by (90-minute optional session for a maximum of 50 attendees) Cell therapy industry seminar: Partnering for regulatory success The ability to overcome regulatory hurdles is one of the most common concerns cited by senior executives of cell therapy companies and their investors. While the underlying biology of a cell therapy intervention contributes significantly to clearance outcomes, many factors associated with regulatory success are in fact independent of the therapeutic platform and therefore controllable from a management standpoint. In this panel session, presenters will focus on how companies leverage partners to give them better control of their regulatory outcomes and then answer questions from the audience on areas within their domain expertise. Role of FDA Cell Tissue and Gene Therapy Advisory Committee and what it can do for you How best to leverage regulatory consultants Reducing the regulatory burden through tools and technology provider partnering Moderator: Dr Paul Pickering, General Manager, Cell Therapy Systems, Life Technologies Panellists: Dr Mahendra Rao, Vice President, R&D, Primary & Stem Cells, Life Technologies, former Chair & current industry representative, FDA Cell Tissue & Gene Therapy Advisory Committee (CTGTAC) Dr Joyce Frey-Vasconcells, Executive Director, PharmaNet, former Deputy Director, Office of Cellular, Tissue and Gene Therapies, CBER Kelli Tanzella, Head of Americas Regulatory Affairs, Life Technologies *» Click here for more information*
Morning plenary session Picking the winners: Assessing the prospects of - and progress with - emerging technologies • What are the keys to accessing Government funding in order to help realize their potential?
9.00	Chair's introduction Leslie Wolfe, PhD, Vice President, Technology Development, Cellular Therapies, Genzyme
Emerging technologies and cell & gene therapy candidates Picking winners from early stage cell and gene therapy pipelines: What novel technologies/indications/therapeutic areas are showing particular promise?
9.05	Presentation & panel discussion Induced pluripotent stem cells (iPS cells): What progress is being made in industry and academia in this field? Debating pathways forward for this, and other such novel technologies of great promise: How can the errors made in the early days of gene therapy be avoided? Moderator: Dr John McNeish, Executive Director, Regenerative Medicine, Pfizer, Inc Panellists: Dr E. Edward Baetge, Senior Vice President & CSO, Novocell James M. Wilson, MD, PhD, Head, Gene Therapy Program, Department of Pathology & Laboratory Medicine, University of Pennsylvania, School of Medicine Dr Philip D. Gregory, D. Phil, Chief Scientific Officer, Sangamo BioSciences, Inc
Next-generation gene therapy technologies: How are they performing in early-stage clinical trials?
9.40	Case study ZFP Therapeutics - exploiting DNA as a platform for drug development ZFPs operate at the DNA level to drive an array of unique therapeutic outcomes Transcription factor technology enables regulation of therapeutic target gene expression ZFN mediated genome editing can add, correct or delete target genes across transformed, primary, stem cells and transgenic models Multiple Phase 1 and Phase 2 programs highlight novelty, flexibility and speed of ZFP Therapeutic drug development Dr Philip D. Gregory, D. Phil, Chief Scientific Officer, Sangamo BioSciences, Inc
10.05	Questions & discussion
10.10	Solutions to overcome barriers encountered in gene therapy trials using first generation technology Review immune barriers to in vivo gene therapy Discuss issues that should be considered in the design of clinical trials Present strategies and data to improve vector technology James M. Wilson, MD, PhD, Head, Gene Therapy Program, Department of Pathology & Laboratory Medicine, University of Pennsylvania, School of Medicine
10.25	Questions & discussion
Next-generation cell therapy technologies: How are they performing in early-stage clinical trials?
10.30	Case study Development of human embryonic stem cells for therapeutic applications Production requirements Preclinical study requirements Delivery requirements Clinical study design Dr Jane Lebkowski, Senior Vice President & CSO, Regenerative Medicine, Geron Corporation
10.45	Questions & discussion
10.50	Cardiac cell therapy: Current status, challenges, and future directions Lessons from current clinical trials Challenges of autologous cell manufacturing, homing and engraftment for optimum therapy New directions to optimize efficacy for cardiac repair through pharmacological treatment and scaffolding of cells Jai Pal Singh, PhD, Chief Scientific Officer, Vice President of Research, St Joseph’s Translational Research Institute
11.05	Questions & discussion
11.10	Roundtable discussion Examining stem cell R&D funding opportunities at State level: What are the keys to securing this funding? How is this funding helping the services sector to develop in step with the industry? What is the real opportunity in terms of the potential for industry to gain funding for the clinical development of stem cell therapeutics? Panellists: Elona Baum, Esq, General Counsel, California Institute for Regenerative Medicine (CIRM) Dr Dan Gincel, Director, Maryland Stem Cell Research Fund, Maryland Technology Development Corporation (TEDCO) Dr Debra S. Grega, Executive Director, Center for Stem Cell & Regenerative Medicine, National Center for Regenerative Medicine
11.40	Morning coffee in the exhibition area
FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:

Focus session 1 Delivering strategic solutions to the key cost control issues and risk management conundrums with cell and gene therapy products • How to minimize Cost of Goods (CoG)? • Build or buy? • How to efficiently source both capacity and materials?
12.20	Moderator's introduction What will be the main drivers for Cost of Gods at commercial scale for cell and gene therapies, and what can be done to affect them? How to scale up to commercial levels whilst controlling Cost of Goods and reducing cost-per-unit sufficiently to make the product viable? How far down can the sector realistically drive CoG? Weighing up the various options to access sufficient quantities of donor cells for a scaled-up allogeneic cell therapy process How to minimize the impact on your CoG Dr Scott R. Burger, Principal, Advanced Cell & Gene Therapy
12.45	Questions & discussion
12.50	Exploring the impact of CoG at commercial scale on pricing, reimbursement and marketing/supply strategies How do you develop a sales forecast related to commercial scale GMP manufacturing? Sheila A. Mikhail, Chief Executive Officer, NanoCor Therapeutics, Inc
1.10	Questions & discussion
1.15	Buffet lunch in the exhibition area
2.25	Quality and qualification of raw materials - impact of legacy materials Jack Harvey, MPH, Director of Manufacturing, Pervasis Therapeutics, Inc
2.45	Questions & discussion
2.50	Addressing the challenges for gene therapy companies in accessing capacity/technology for large scale gene therapy production What are the lessons to be learned from the recent establishment of a state-of-the-art commercial gene therapy manufacturing facility? Is the gene therapy field as a whole ready to take the step up to commercial scale in the same way that monoclonals did when they broke onto the market? What are the advancements in terms of developing gene therapy manufacturing standards and how much work still needs to be done? Robert Shaw, Technical Director, Ark Therapeutics
3.10	Questions & discussion
3.15	Panel discussion How should you plan in advance to avoid issues with the availability and supply of raw materials as you scale up? How must industry and the services sector collaborate to address the current shortfall of raw materials supply at commercial scale for both cell and gene therapy products? Panellist: Dr Robert A. Preti, President & Chief Scientific Officer, Progenitor Cell Therapy, LLC
3.35	Moderator's closing summary
3.40	Close of session and of the Phacilitate Cell & Gene Therapy Forum 2010, followed by afternoon tea

OR \| Focus session 2 Stem cell therapy clinical strategy and data update • How are leading candidates performing in the clinic? • Addressing key fundamental questions regarding the sourcing and delivery of stem cells
12.20	Moderator's introduction Dr Debra S. Grega, Executive Director, Center for Stem Cell & Regenerative Medicine, National Center for Regenerative Medicine
Case studies Delivering the latest clinical trial designs and data from MSC-based cell therapies in development
12.25	MSCs for immune modulation/suppression A look at Mesenchymal Stem Cells (MSCs) as potential cellular immune therapeutics to treat autoimmune disease Objective: The immunomodulatory properties of human and murine bone marrow – derived mesenchymal stem cells (MSCs) include suppression of T cell responses and reduction of key inflammatory mediators, such as IFN-_ and TNF_.We postulated that MSC mediated immune suppression may provide benefit in the treatment of spontaneous T cell-mediated autoimmune diseases, such as type 1 diabetes Research designs and methods: Allogeneic murine MSCs were administered intravenously in the non-obese diabetic (NOD) model of spontaneous diabetes, either prior to (preventative protocol) or at the time of disease onset (therapeutic protocol) in order to study the amelioration of disease Results: Prophylactic delivery of allogeneic MSCs to pre-diabetic NOD mice delayed the onset of disease. Therapeutic treatment at the time of disease onset was effective in reversing disease, as measured by restoration of blood glucose levels to the normal range. These data suggest that MSCs are able to modulate an ongoing autoreactive immune response Benefits: MSCs can effectively alter an autoimmune response MSCs can ameliorate ongoing diabetes Impact of MSC therapy on other auto-immune diseases Speaker to be announced
12.45	Questions & discussion
12.50	Mesenchymal stem cells in cardiac repair: Translational development and Mechanisms of Action Discuss the translational development of cell-based therapy for human heart disease Discuss mechanism of data of cell-based therapy from large animal models Discuss clinical trial design of cell based therapeutics for human heart disease Dr Joshua M. Hare, Professor of Medicine, Director of the Interdisciplinary Stem Cell Institute, Miller School of Medicine, University of Miami
1.10	Questions & discussion
1.15	Buffet lunch in the exhibition area
2.25	Case study When and why does cell source matter? Adipose derived stem cells in the cure of peri-anal fistula Choice of cell types: Mechanism of Action Choice of cell types: Autologous versus allogeneic use Choice of cell types: EMEA/FDA Choice of cell types: Manufacturing and scale of economy Choice of cell types: FTO versus own patents Dr Dirk Büscher, Vice President of Research & Development, Cellerix
2.45	Challenges in delivering cells to the heart The challenges of delivery to the heart The challenges of clinical trial design for cardiovascular cell therapy The NHLBI-funded cell cardiovascular therapy network The new NHLBI Cell Consortium Dr Sonia Skarlatos, Deputy Director, Division of Cardiovascular Sciences, National Heart, Lung & Blood Institute, NIH
3.05	Short presentation & panel discussion Clarification of the latest developments with the stem cell patent environment What are the repercussions for your IP portfolio and how can you strengthen you position in this regard? Because of the complexity of the regulatory pathway, data exclusivity will be an important aspect as well as patent protection Cross-licensing third party IP is likely necessary Dr Liz Bui, Director, Intellectual Property & Corporate Development, Novocell
3.35	Moderator's closing summary
3.40	Close of session and of the Phacilitate Cell & Gene Therapy Forum 2010, followed by afternoon tea

OR \| Workshop Overcoming the remaining challenges with viral and non-viral delivery platforms for gene and RNAi therapies
(Highly interactive session for a maximum of 30 participants)
Case studies Examining the latest safety and efficacy data for varying viral and non-viral vector platforms in clinical development: How are they performing and what issues remain?
12.20	Moderator's introduction Professor Sander Van Deventer, CSO, AMT BV
12.25	Overcoming (at least some of) the remaining challenges with viral and non-viral delivery platforms for gene therapy Recent apparent successes Teaching vectors new tricks Immunology considerations Manufacturing – as always From niche to larger applications Choosing the right tool for the job at hand Dr Sam Wadsworth, Group Vice President, Translational Research, Genzyme Corporation
12.45	Questions & discussion
Case studies Examining the latest safety and efficacy data for varying viral and non-viral vector platforms in clinical development: How are they performing and what issues remain?
12.50	Adenoviral/ AAV vectors Long-term expression and therapeutic benefit of alipogene tiparvovec (Glybera‰) One time intramuscular administration of alipogen tiparvovec (Glybera‰) results in long-term expression and a significant therapeutic benefit in patients with LPL deficiency The AAV-LPL manufacturing process has been optimized, is fully scalable and cGMP compliant. The mode of action will be discussed Long-term safety (up to 4 years of follow-up), biodistribution and vector shedding data are presented Professor Sander Van Deventer, CSO, AMT BV
1.15	Buffet lunch in the exhibition area
2.25	Lentiviral vectors - safety profile Analyzing recent instances of lentiviral vector integration in patients: What are the most recent developments with these and how can the safety profile of lentiviral vectors be improved moving forward? Safety data base on patients treated in VIRxSYS Phase I and II clinical trials Total number of autologous CD4+ cells administered to patients Total number of lentiviral copies administered to patients Number of patients' years of safety Total number of lentiviral vectors produced under cGMP for these trials Integration profiles of lentiviral vectors in patients Dr Gary J. McGarrity, Executive Vice President of Scientific & Clinical Affairs, VIRxSYS Corporation
2.45	Questions & discussion
2.50	HGF plasmid: Efficacy and safety from early phase trials in Critical Limb Ischemia The current management of Critical limb ischemia The properties of HGF plasmid and the role of angiogenesis Efficacy and Safety data from early phase HGF trials Future challenges Prannath Marrott, FRACP, MRCP, Senior Vice President, Clinical Development & Regulatory Affairs, AnGes, Inc
3.10	Questions & discussion
3.15	Panel discussion What is the optimal R&D strategy for ex-vivo cell therapies? How are delivery platforms impacting both safety and efficacy and how can challenges with both aspects be addressed through clinical trial design and technology development/application? What constitutes the actual 'product' in the realm of ex vivo cell therapy? Is it the cell? Is it the gene? Is it the process? What are the repercussions for the clinical and business models?
3.35	Moderator's closing summary
3.40	Close of session and of the Phacilitate Cell & Gene Therapy Forum 2010, followed by afternoon tea

Gold Passes
Would you like to attend sessions during both the Cell & Gene Therapy Forum and the co-located Washington Vaccine Forum? E-mail team@phacilitate.co.uk or call +44 (0)20 7839 6137 for more information on our Gold Passes.

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