Agenda

DAY 1 | Monday January 26th 2009
7.30	Registration & buffet breakfast in the exhibition area
Morning plenary session Maturing the cell, tissue and gene therapy sector - Creating and driving value along the R&D and commercialization path
Headline summary on status of approvals worldwide and on who is now closest to market Big pharma’s perspective on how the cell and gene therapy sector is reaching maturity and driving value What is encouraging our interest in the sector? Short case studies from cell and gene therapy product pioneers: How are we driving value for all stakeholders, including payers? What practical advice do we have for those following us? When should you go to the payers and with what data? What reimbursement systems can be used for these therapies in the absence of classification codes? A comparison with the commercialization path of monoclonal antibodies: What lessons can cell and gene therapy companies learn to avoid reinventing the wheel? What elements of the overall maturation cycle will be the same, and which ones are truly unique to cell and gene therapy? Panel discussion: Making money as a business and as an industry - How will value be driven? Therapies, tools and services: How is the sector developing and what needs to happen for all these layers to make money? Consolidation/mergers and acquisitions What is expected in 2009/2010? How are they an indication of market maturation? Investigating untapped synergies between regenerative medicine, tissue engineering, devices, and cell and gene therapy
FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:
Focus session 1 Manufacturing strategy: What roles are outsourcing, new platform technologies and process automation playing in achieving profitability?
How is supply and demand developing for cell and gene therapy contract production? Case studies: Making the decision to contract out manufacturing or retain in house How do you decide whether manufacturing is a core competence to keep in- house? Advantages/disadvantages to using a contract manufacturer instead of a partner pharma company Criteria for choosing a CMO: How do you assess the contactor’s competence? How to manage the relationship successfully? Deciding whether testing should be outsourced or not Panel discussion: Manufacturing autologous and allogeneic products - How is cost of goods being controlled and what impact is regional manufacture having? How can we drive COGs down to profitable levels? How to manufacture regionally: Addressing logistical, shelf-life, storage and import/export considerations, and their impact on COGs What are the commercialization platforms for success? Developing platform/enabling technologies: Which ones will be critical for the maturation of the sector by reducing cost of goods to make them reimbursable? What progress is being made on the development of enabling technologies tailored to the needs of cell and gene therapy products? Ensuring that they are incorporated early enough to be able to support pivotal trials right through to commercialization Case study: What are the financial and technical implications of investing in automation? Do we understand the biology and mechanism of action well enough to use it? Can you do GMP using automation? What about validation? What impact does it have on COGs? Panel discussion: Debating the role of systems and process engineering in the manufacture of cell and gene therapy products
OR | Focus session 2 Designing cell and gene therapy clinical trials • Taking a close look at each phase of development to identify the key inflexion points, assess risk:benefit, and plan for commercialization and reimbursement
Translating from preclinical to phase I Animal studies / toxicology modelling: What constitutes ‘enough’ animal testing? What is the relevance of animal models and what alternatives are there? In silico modelling to replace multi-species preclinical data requirement? Homologous recombination Drug screening Cell therapy case studies: Illustrating the key elements of trial design and the coordination between clinical, manufacturing and commercial at each stage of the development process Phase I/II trial design: Blinded trial design Safety studies Dosage Collecting the right data all through the stages Providing biological assurance – mechanism of action Phase III trial design: Adaptive trial design Critical mass of patients – what’s the minimum? Blinded trials / placebo / control mechanisms Realistic endpoints Qualification/validation of bioassays used to measure clinical endpoints Gene therapy case studies: Illustrating the key elements of trial design and the coordination between clinical, manufacturing and commercial at each stage of the development process Phase II trial design: What’s the critical mass of patients required to demonstrate efficacy and support the commercialization path? Dosage Collecting the right data for registration Efficacy: Demonstrating added value to patients and payers Link to commercialization and reimbursement Phase III trial design: Use of global trials Data collection, reporting and acceptance issues Link to commercialization and reimbursement Measuring outcomes / evidence-based medicine Panel discussion: Planning ahead for commercialization at every stage of the development process - What are the key criteria to consider at each inflexion point? Coordinating efforts across the organization to ensure that R&D, manufacturing and commercialization are fully supportive and in-step
OR | Workshop Latest results from the CNS area: how are cell and progressing in preclinical and clinical development?
(Highly interactive session for a maximum of 30 participants)
How are collaborations and partnerships with patient foundations and advocacy groups helping to drive these therapies into and through the clinic? Promising preclinical results: Latest breakthroughs and ongoing safety challenges Gene therapy case studies: Clinical results Cell therapy case studies: Clinical results Panel discussion: What is required to drive the CNS area forward to maturity? How can we make foetal tissue trials more robust? How can safety hurdles be addressed?
THEN | Afternoon plenary session What’s missing from the tool box? - Understanding which tailored enabling technologies cell and gene therapy companies need to help get into humans and progress to market?
Panel discussion: Company perspectives - Which tailored enabling tools are missing and what would be first on the wish list? What types of matrix / scaffold would be valuable? What are the limitations of flow cytometry and how can we overcome them? High quality imaging to track vectors and cells Validated PCR to monitor cells – DNA & RNA Closed systems for cell extensions, cell separation and cell processing Use of small molecules in stem cell differentiation Potency assays & biomarkers Automation Adaptive trial design Development of full GMP (eg big-pharma level quality) Fostering coordination between companies and tool/service providers to enable a better understanding of the needs of the cell and gene therapy sector

DAY 2 | Tuesday January 27th 2009
7.30	Registration & buffet breakfast in the exhibition area
Morning plenary session Analyzing the latest interpretation of the US, EU and Asian regulatory environments for cell, gene and tissue therapies - How are they impacting global R&D and commercialization strategies?
A comparison with the regulatory pathways for other biologics: Monoclonal antibodies, vaccines and RNAi What are the key similarities and differences? Regulators from US, EU and Japan will discuss how regulatory pathways are evolving with respect to: Differences of approach to autologous/allogeneic cell therapies and to different cell sources – how to determine the potency of a stem cell Regulatory differences between ex vivo and in vivo gene therapy Relevance and requirement for animal models MOA characterization Regulations concerning long-term follow up – especially for vectors Regulation of combination cell/gene therapy products and devices Regulation of disease modification therapies/curative therapies Environmental risk/viral shedding: ICH harmonization International harmonization for import/export of cell and gene therapy products Paediatrics: What are the appropriate safeguards for doing clinical trials in fatal paediatric conditions? Update on the obligation to insert a paediatric plan in your marketing authorization application What is the likelihood of regulation of rogue clinics, especially in Asia?
FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:
Focus session 1 Rigorous characterization of cell therapy products through the phases: Ensuring comparability
series of case studies illustrating how comparability can be ensured as manufacturing scales up Clarifying how the regulatory/GMP requirements are met at each stage: What constitutes a change (or not) in a product? Phase 1/II – case study 1 Sourcing raw materials Generating new lines and cultures Ancilliary materials Cryo-preservatives Preparing to move to the next development stage - Phase 1/II – case study 2 Process characterization and comparability Release assays Potency assays Purity and functional assays Preparing to move to the next development stage Phase I/II – case study 3 Quality systems Creative ways of doing release testing and how to manage this in real time Preparing to move to the next development stage Phase III – case study 1 Commercial scale CMC When should you start producing the commercial batches and generating data? Product characterization Process characterization Process validation Phase III – case study 2 Facility & process integrity What do you need to show to be able to switch from one facility to another? Multi-product manufacturing Protection from cross-contamination Controls for autologous products Chain of custody and identity Control for starting materials Control for product Transport validation and supply chain IT support versus manual Data management & integrity Case study and panel discussion: Planning well ahead for regional / global manufacturing and scale-up - How to ensure consistency and comparability What is required to gain a license to manufacture at a specific facility?
OR | Focus session 2 Designing cell and gene therapy clinical trials • Taking a close look at each phase of development to identify the key inflexion points, assess risk:benefit, and plan for commercialization and reimbursement
What is the commercial potential of cell, gene and tissue therapies in combination with devices, with small molecules and with each other? Which disease areas will be most suited to their application Orthopaedic? Cancer? Which combinations are most likely to improve delivery for cell and gene therapies? Matrixes and scaffolds? Drugs? Other biologics? What product life extension opportunities are emerging as a result of these combinations? How is the regulator addressing and preparing for the complexity of assessing the various combinations cell and gene therapies with other products? How is coordination being enabled between departments to streamline regulatory oversight? What studies will be required for combinations of cell/gene therapies with drugs that are on the market already? How will the safety and quality of the matrix or scaffold be assessed when used in combination with cell/gene therapy? How are these regulations likely to evolve as combination products become more prevalent? Case studies illustrating the decisions taken in selecting a particular combination, and how the development and regulatory hurdles are being overcome How and when are the products combined? Specific issues when designing clinical trials How to dose? What’s in the regulatory package? How do requirements between US and EU vary? How will they be reimbursed? Panel discussion: Is everyone doing enough to prepare for the growth of combination product opportunities? How will levels of risk be assessed for combination products?
OR | Workshop The EU regulatory framework for Advanced Therapy Medicinal Products: Practical case studies illustrating the realities of its implementation and the differences between member states’ interpretation
(Highly interactive session for a maximum of 30 participants)
What impact has the framework had on procedures to date? Clarifying the different roles of CHMP, CAT, Innovation Task force (ITF) Specifying the peculiarities about how it is functioning: What do you need to be aware of? Allowance for certification of quality of non-clinical data for SMEs Traceability and pharmacovigilance: Scope and harmonization globally Hospital exemption
THEN | Afternoon plenary session Ongoing opportunities for - and barriers to - cell, gene and tissue therapy
Which diseases are now considered most amenable to their application? How will they fare against the competition? Panel discussion OPPORTUNITIES: What makes a good cell or gene therapy target? Developing a framework to enable the evaluation of indications and assess where strengths and weaknesses lie How do safety, benefit:risk, and time to market compare? BARRIERS: Countering the negative impact of ‘rogue’ clinics and medical tourism – how can the opportunities and value be retained in the west? What should clinicians be telling their patients about the value of these therapies? Harvesting the human clinical data that is going to waste How will cell, gene and tissue therapies fare against competition from cocktails of small molecules and growth factors?

DAY 3 | Wednesday January 28th 2009
7.30	Registration & buffet breakfast in the exhibition area
Morning plenary session Gaining an insight into big pharma and big biotech’s current and future plans for cell, gene and tissue regeneration therapies - Analyzing internal and external investment, and collaborative moves
Recent case studies of companies buying, investing in and partnering with cell, gene and tissue engineering companies Panel discussion: How is big pharma/big biotech’s interest in the cell, gene and tissue regeneration sector now manifesting itself? What is our ongoing strategy for involvement in the West and the East? How are companies ramping up their internal capabilities in the cell, gene and tissue regeneration sectors? How are Asian subsidiaries approaching these new therapies? What does the product profile need to look like to turn them into fundable technologies?
FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:
Focus session 1 Viral and non-viral vectors for the targeted delivery of gene-based therapies: Advances in safety, regulation, manufacturing and scale-up
What is the latest regulatory stance on vector safety and production? Clarifying regulators current concerns around viral vector production How does localized versus systemic delivery impact our decisions? Case studies: Viral and non-viral delivery mechanisms in clinical trials – integrating each specialised development function into one cohesive, synchronized plan Toxicity Specificity Durability Host responses Re-administration/immunogenicity issues Regulatory interaction Manufacturing hurdles Timelines for commercial adoption Planning worldwide product launch RNA therapy roundtable: What promising technologies are coming out of research and how are they progressing in the clinic? How will different therapeutic options be regulated and how will this differ from "standard" gene therapies? How will this impact their development path and progress? What advances are being made in specificity and delivery of RNA therapies?
OR | Focus session 2 Assessing the therapeutic performance of stem cells from different sources - How is a better understanding of mechanism of action impacting their application to particular disease environments?
Alternative sources of stem cells – natural and synthetic: Which have real potential? Which are ethically acceptable? Case studies: Taking three different sources of stem cells to examine the capabilities and benefits of each How thorough is the understanding of the mechanism of action? What are the specific toxicity and safety issues for each? How do you determine the potency of a stem cell? What might the regulatory pathway look like? How would they fare in the clinic? Can they yield enough to be cost effective? What are the manufacturing and scale-up issues? Case study 1 – embryonic stem cells What are their terms of acceptability to the regulator: Is it a raw material or is it an intermediate that does have to be made under GMP? Case study 2 - induced pluripotent stem cells Would they be considered genetically modified? What do these genetic differences mean for industry? Is it a gene or a cell therapy? How will they be classified and how will they be regulated? Case study 3 – mesenchymal stem cells Immunogenicity of stem cells: What is the regulator looking for? What types of assays are required? Correlating immunogenicity of cells with your therapeutic results Clarifying how the tests are being interpreted What might the regulatory pathways be for embryonic stem cells and IPS? Directed differentiation: what factors affect it and what impact is it having on therapeutic performance of stem cells? How do you define an efficient differentiation? Comparing one cell lineage vs another How do you deal with undifferentiated cells How is the field being pursued? Panel discussion: Which cell type is the right one to use? What questions should companies ask in order to select one cell type over another and minimize investment risk? What other sources of stem cells are being evaluated and with what results? How can cell companies prove that theirs is better? There are too many unknowns – what are we doing to address this?
OR | Workshop Financing global growth in the cell, gene and tissue therapy sector What sources of funding are now being exploited? Are investors evaluating these opportunities appropriately?
(Highly interactive session for a maximum of 30 participants)
What are the alternatives to VC funding? Summary of recent major financing events What impact is the increasing interest from big pharma having on the funding environment? Case studies demonstrating the application of different sources of funding, how the companies attracted the capital, and how the investors evaluated the risk:reward Partnerships with research tools companies Corporate venture financing Foundations / philanthropic investors Government money Equity funding – IPOs Best practice due diligence for the cell and gene therapy sector: What criteria should investors be evaluating when making investment decisions, and how can companies better prepare for this? IP due diligence Panel discussion: VC response What do we see as core evaluation criteria for the cell and gene therapy sector? To what extent does the product vs service debate apply to cell and gene therapies, and how does it influence financing options and investment cycles?

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