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DAY 1 | Monday January 26th 2009
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| 7.30 |
Registration & buffet breakfast in the exhibition area |
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Morning plenary session
Maturing the cell, tissue and gene therapy sector
- Creating and driving value along the R&D and commercialization path
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- Headline summary on status of approvals worldwide and on who is now closest to market
- Big pharmas 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

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FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:
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Focus session 1
Manufacturing strategy: What roles are outsourcing, new platform technologies and process automation playing in achieving profitability?
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- 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 contactors 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

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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
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- 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 whats 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: Whats 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

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OR | Workshop
Latest results from the CNS area: how are cell and progressing in preclinical and clinical development?
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(Highly interactive session for a maximum of 30 participants)
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- 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?

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THEN | Afternoon plenary session
Whats 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?
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- 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

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DAY 2 | Tuesday January 27th 2009
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| 7.30 |
Registration & buffet breakfast in the exhibition area |
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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?
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- 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?

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FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:
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Focus session 1
Rigorous characterization of cell therapy products through the phases: Ensuring comparability
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- 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?

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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
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- 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
- 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?
- Whats 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?

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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
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(Highly interactive session for a maximum of 30 participants)
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- 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

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THEN | Afternoon plenary session
Ongoing opportunities for - and barriers to - cell, gene and tissue therapy
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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?
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DAY 3 | Wednesday January 28th 2009
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| 7.30 |
Registration & buffet breakfast in the exhibition area |
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Morning plenary session
Gaining an insight into big pharma and big biotechs current and future plans for cell, gene and tissue regeneration therapies
- Analyzing internal and external investment, and collaborative moves
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- Recent case studies of companies buying, investing in and partnering with cell, gene and tissue engineering companies
- Panel discussion: How is big pharma/big biotechs 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?

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FOLLOWED BY YOUR CHOICE OF 3 PARALLEL BREAKOUT SESSIONS:
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Focus session 1
Viral and non-viral vectors for the targeted delivery of gene-based therapies: Advances in safety, regulation, manufacturing and scale-up
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- 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?

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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?
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- 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?

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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?
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(Highly interactive session for a maximum of 30 participants)
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- 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?
- 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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