Welcome To the Phacilitate Cell & Gene Therapy Forum 2010

Agenda | Day 1 Printer friendly version - full agenda

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DAY 1 \| Monday, January 25th 2010
7.30	Registration & buffet breakfast in the exhibition area
Morning plenary session How will cell therapies, gene therapies and tissue-engineered products fit in to a reforming healthcare picture? Examining the commercialization strategies of companies in pivotal trials • What degree of benefit will they need to deliver to secure reimbursement?
9.00	Chair's introduction Dr Andrew L. Pecora, Chairman of the Board, Amorcyte
9.10	Opening Address Assessing the feasibility of cell and gene therapies as commercial entities in the evolving healthcare environment What prices can traditionally high-value biologics expect to reach in the evolving healthcare marketplace, and what benefit level will be required to achieve them? Will functional improvement be enough to gain top prices, or will such therapies need to have an impact on mortality? How commercially feasible are 'personalized medicines' given ongoing healthcare reform? Eric Faulkner, Senior Director, US Market Access & Reimbursement, RTI Health Solutions
9.30	Questions & discussion
9.35	Phase III roundtable Representatives of companies with cell and gene therapies and tissue engineered products in Phase III trials will discuss how they are preparing for the commercialization of their products What are their experiences with navigating the BLA/MAA processes in the United States and Europe? How are they developing their pricing and reimbursement strategies? How do you value a one-stop treatment in an increasingly stringent healthcare climate? What preparations are they making for commercial-scale manufacture and delivery? How should companies in preclinical and early-phase clinical development be looking to build reimbursement considerations into their R&D strategy? What are the key considerations in preparing for the logistical side of commercialization of these products? Are the global supply and distribution logistics in place for the roll out of these therapies? If not, what preparations need to be made? Speakers: Dr Mitchell Gold, President & CEO, Dendreon Corporation Dr C. Randal Mills, President & CEO, Osiris Therapeutics, Inc Gil Beyen, Co-founder & Managing Director (CEO), TiGenix NV Dr Claudio Bordignon, President & CEO, MolMed SpA Kristin Comella, Vice President of Research & Corporate Development, Bioheart, Inc Panellist: Mike Rice, President, CEO & Chairman, BioLife Solutions, Inc
11.05	Morning coffee in the exhibition area
FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:

Focus session 1 An in-depth examination of autologous and allogeneic cell therapy business models: What are the keys to achieving commercial viability?
11.50	Moderator's introduction Solutions to challenges often encountered on the path to commercialization of cellular therapies Tissue procurement Oversee donor selection and screening and the tissue acquisition process Determine final donor eligibility Process/media development Media optimization (testing over 80 different basal media we manufacture) Scale-up from flasks to large vessels Determining optimal cell seeding density Optimizing feeding schedule and cell densities Development of commercial-scale cryopreservation methods cGMP manufacturing Autologous cells of varying shelf life for shipment to the USA and Europe Allogeneic cells, including sourcing, production of cell banks, and expansion to product quantities. Stem cell production via both master and feeder cell banks Freezing, packaging, shipping and distribution Delivering cells to clinical sites Managing large-scale distribution centers Regulatory services David Smith, Head of Global Therapeutic Cell Solutions, Lonza
12.05	Questions & discussion
Case studies What are the chief strategic considerations with, and limitations of, autologous and allogeneic cell therapy manufacturing processes? What have been our experiences in securing funding? What are investors' chief concerns with each model and how can they be assuaged? How does each business model impact on the R&D portfolio as a whole? How do you decide on the number of projects that can be advanced through R&D? How do the practical, regulatory and IP considerations in utilizing a device and/or involving surgical intervention impact on the business model? How will each model perform commercially on a global scale? Examining each model from the commercial perspective - what are the lessons to be learned by companies about to, or having recently entered the clinic?
12.10	Novel autologous cellular therapeutics addressing billion dollar markets and unmet clinical needs Compelling clinical data and well defined regulatory paths Established manufacturing infrastructure that can be scaled Debunking the myths of autologous therapies: Gross margins, logistics and adoption Ed Field, President & COO, Aldagen
12.30	Case study Unique opportunities for expanded autologous mixed cell products: Targeting unmet medical needs and solving the challenges of commercial-scale manufacturing Multiple chronic disease targets Scale out manufacturing and distribution logistics Reliability, efficiency and cost effectiveness of automated processes George W. Dunbar, Chairman of the Board, Aastrom Biosciences
12.50	Buffet lunch in the exhibition area
2.00	Case study Development and commercialization of a novel universal allogeneic cellular therapeutic Business considerations in developing an allogeneic cell therapy Manufacturing/distribution, clinical and regulatory issues Commercialization challenges: COGS, reimbursement and market adaptation Dr Ram Mandalam, President & CEO, Cellerant Therapeutics, Inc
2.20	Extending stability and maximizing yield of source material and finished products The critical role biopreservation plays in commercialization of new cell therapies Detrimental effects of delayed onset cell death Avoiding assay pitfalls Extending stability of source material and finished products Maximizing post-preservation viability and functional recovery of finished products Aby J. Mathew, PhD, Senior Director, Strategic Relations & Senior Scientist, BioLife Solutions, Inc
Case studies Centralized vs onsite cell therapy processing: What are the relative pros and cons of each option, and how does each fit into the business model?
2.40	Centralized Safety considerations Regulatory considerations Speed to market How are we each addressing the practical process/manufacturing/distribution issues with commercial scale in mind? (Eg. cell storage) Dr C. Randal Mills, President & CEO, Osiris Therapeutics, Inc
3.00	How do you begin to develop a model for point-of-care therapeutic delivery, given that the traditional manufacturing/delivery model is centralized? Cytori, as one of the first commercial-stage cell therapy companies, will be used as a case study to illustrate how to: Navigate the product design and development process to bring an autologous point-of-care device to market Commercialize, as well as train and educate the medical community in the practice of, cell-based therapies The following critical considerations will be addressed: Autologous or allogeneic: Commit to model ideally suited for cell source, its attributes, and anticipated costs for manufacturing and distribution Parallel product development, manufacturing, and regulatory strategies Corporate partners: The if, when, and how to manufacturing, development and commercialization collaborations Mark E. Saad, CFO, Cytori Therapeutics, Inc
3.20	Panel discussion How might evolving FDA guidelines impact business? How do autologous and allogeneic models compare with regard to their utility for cell-therapy-gene therapy combination approaches (ex vivo cell therapy) and with regard to therapy-device combinations? What will be the role of small molecules in cell therapy moving forward? What opportunities and challenges are there for the industry in this area? What are the pros and cons compared to existing autologous and allogeneic business models? Panellists: Dr Jon Rowley, Director of Cell Therapy R&D, Lonza Biosciences Dr Scott R. Burger, Principal, Advanced Cell & Gene Therapy
3.40	Moderator's closing summary
3.45	Close of session followed by afternoon tea in the exhibition area

OR \| Focus session 2 Gene therapy clinical update: Analyzing recent clinical success stories in key therapeutic areas to inform your trial design strategy
11.45	Moderator's introduction Reviewing the gene therapy field: Who's still playing and which are now the most advanced product candidates on a global basis? What are the highlights from the past 12 months? Who's in the running to make a success with gene therapy? Which new products are the ones to watch? Is there a common theme emerging yet as to what will make a successful gene therapy product? Professor Alan Boyd, Honorary Professor, College of Medical & Dental Sciences, University of Birmingham Medical School & Managing Director, alan boyd consultants ltd
Case studies Updates on the latest clinical trial designs and data demonstrating clinical benefit for gene therapies in development How did we successfully translate from preclinical to clinical development? How are labelling and reimbursement considerations impacting trial designs?
12.00	Phase I trial of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of Adeno-Associated Virus gene vector One rare form of blindness among many - clinical and molecular perspective Pre-clinical proof-of concept Proving the human blindness has the defective pathway worth treating Understanding results of the trial required standard but also novel outcomes Dr Samuel G. Jacobson, Professor of Ophthalmology & Director, Center for Hereditary Retinal Degenerations & Retinal Function Department, University of Pennsylvania School of Medicine
12.20	Case study ZFP Therapeutics – diabetic neuropathy ZFPs operate at the DNA level to drive an array of unique therapeutic outcomes Transcription factor technology enables regulation of therapeutic target gene expression Sangamo’s lead ZFP Therapeutic candidate, SB-509, which is designed to upregulate VEGF-A, has demonstrated neuroprotective and neuroregenerative functions in two Phase II clinical studies in moderate and severe Diabetic Neuropathy (DN) Within DN, SB-509 represents a novel, first-in-class, disease-modifying therapy Edward Lanphier, President & CEO, Sangamo BioSciences, Inc
12.45	Questions & discussion
12.50	Buffet lunch in the exhibition area
2.00	AAV2-neurturin (CERE-120) for Parkinson’s disease Results of a Phase II controlled Parkinson’s disease clinical study; failure to meet primary endpoint Further analysis of the clinical data; statistically significant secondary endpoints Scientific rationale for the results; defects in retrograde transport Ways to overcome the issues; new targeting methods and mitigating the placebo effect Joao Siffert, MD, Chief Medical Officer, Ceregene
2.20	Questions & discussion
2.25	Case study Lessons learned from the AGENT trials: The future of cardiovascular biologics Cardium Therapeutics is conducting two of the most advanced gene therapy clinical trials in two indications with two different product candidates Therapeutic angiogenesis with Generx (Ad5FGF-4) for chronic myocardial ischemia The Phase II/III AGENT-3 and -4 trials with intracoronary application of Generx in patients with chronic myocardial ischemia (angina) provided important insights for the design of pivotal Phase III trials regarding the optimal patient population and clinical endpoints Recent preclinical studies support the use of certain small molecules aimed at optimizing transfection efficiency when used in combination with Generx Enhanced wound healing with Excellarate (Ad5PDGFB in Gene Activated Matrix) for non-healing diabetic foot ulcers Preclinical studies and a Phase I/II clinical trial provided evidence for the safety and efficacy of Ad5PDGF-B when applied in a Gene Activated Matrix (GAM) A Phase IIb clinical study (MATRIX) has just been completed, and the results will be presented at the meeting Dr Gabor M. Rubanyi, Chief Scientific Officer, Cardium Therapeutics
2.45	Questions & discussion
2.50	Oncology Dr Claudio Bordignon, President & CEO, MolMed SpA
3.10	Questions & discussion
3.15	Panel discussion Identifying and addressing the immunological barriers to the development and commercialization of gene therapies What are the potential solutions? What steps can be taken in R&D from the early stages?
3.40	Moderator's closing summary
3.45	Close of session followed by afternoon tea in the exhibition area

Workshop Providing practical insights into how to build a commercially focused cell/gene therapy or tissue engineered product company from the ground up • What are the key business challenges you will face at each stage of R&D, and how can you prepare to meet them
(Highly interactive session for a maximum of 30 participants)
11.45	Moderator's introduction Paul J. Schmitt, Managing Director, Novitas Capital
11.50	What fundamental business, clinical, regulatory and operational infrastructure is required, and at what stage should each aspect be developed/introduced? How do you decide which aspects will be outsourced and which will remain core competencies as your organization starts to develop? How do you assess whether a potential outsourcing partner has the specific technical expertise required for novel cell and gene therapy technologies? Staffing issues: Understanding whom you will need, when you will need them, and how to source them Kristin Comella, Vice President of Research & Corporate Development, Bioheart, Inc
12.10	Questions & discussion
12.20	Preparing for the regulator: What are the keys to successfully navigating the regulatory process? Understanding the logistics of commercial delivery and the regulatory implications involved How to build these considerations in to early-stage R&D How can QC considerations be pushed further upstream in cell therapy R&D? Assessing various technology approaches with the potential to deliver the data that regulators and CMOs alike will require at later stages Dr Wilfried Dalemans, CTO & Vice President, Regulatory Affairs, TiGenix NV
12.40	Questions & discussion
12.50	Buffet lunch in the exhibition area
2.00	Understanding the pathway to the clinic: How to match research data with clinical targets? What data should you have before moving to the next stage? Rigorous product characterization at each stage of development At what stage in the pathway to the clinic should you create the infrastructure to develop a commercially viable manufacturing process? Managing risk and cost while moving into clinical development Dr Janneke Meulenberg, CEO, ORCA Therapeutics
2.20	Questions & discussion
2.30	The importance of defining your product at an early stage: How can/should you build dosing, labelling and reimbursement strategies into your early-stage R&D decision-making processes and study designs? Cell therapy production protocols must optimise among product quality/potency, clinical convenience, regulatory approval and reimbursement objectives Personal cell therapies frequently involve highly focussed applications – is off-label use possible when the cell therapy manufacturer has direct knowledge of each patient? Seeing the big picture for reimbursement – the cell therapy is often only a part of an overall procedure which needs reimbursement approval Gregg Sando, CEO, Cell Medica Limited
2.50	Questions & discussion
3.00	Entering and progressing through the clinic: What are the keys to controlling costs and cash-burn rate at each phase? How much do you know about your product? How much should you know about your product at each stage? The biotech casino: Resource allocation, doing more with less, and risk assessment Partnering is good but can be stressful Setting goals and hitting them is a cash flow issue Dr Doug Jolly, Executive Vice President of Research & Product Development, Tocagen, Inc
3.20	Questions & discussion
3.30	Questions, discussion & Moderator's closing summary
3.45	Close of session followed by afternoon tea in the exhibition area

THEN \| Afternoon plenary session What traditional and alternative funding options are now open to academics and companies seeking to translate preclinical cell/gene therapy and tissue engineered product candidates to Phase IIa and beyond?
4.25	Chair's introduction Dr Alex Barkas, Managing Director, Prospect Venture Partners
4.30	Roundtable discussion What funding is available in the translational phase? Where are the key inflexion points for value creation and how can biotech companies progress through them in the current financial climate? VC perspectives: What specific aspects of a cell therapy, gene therapy or tissue engineered product add value in our eyes currently? What is the relative value of product scalability versus cell source at the moment? What are the opportunities for working with academia to bridge the translational 'valley of death'? What alternative funding options are out there? What are the pros and cons of receiving orphan drug designation for a cell or gene therapy product and how must one adapt the business model accordingly? What opportunities are consortia providing to bridge the translational funding gap? What novel funding and cost-saving initiatives are available through the EU/EMEA How can companies from all regions of the world capitalize on them? Panellists: George W. Dunbar, Chairman of the Board, Aastrom Biosciences Dr Brian A. Meltzer, Senior Director, RedScript Ventures, Johnson & Johnson Corporate Development Paul J. Schmitt, Managing Director, Novitas Capital Gary J. Kurtzman, MD, Vice President & Managing Director, Life Sciences Kenneth C. Aldrich, Chairman & CEO, International Stem Cell Corporation & Managing Director, Convergent Investors, LLC Dr Geert-Jan Mulder, General Partner, Forbion Capital Partners Michael J. Werner, Partner, Holland & Knight Dr Ram Mandalam, President & CEO, Cellerant Therapeutics, Inc Dr Samuel G. Jacobson, Professor of Ophthalmology & Director, Center for Hereditary Retinal Degenerations & Retinal Function Department, University of Pennsylvania School of Medicine
5.40	End of day 1 followed by a themed cocktail reception in the exhibition area

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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