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31 October – 2 November , 2017

London, UK



Day One
Wednesday 1st November, 2017

Day Two
Thursday 2nd November, 2017

Chair’s Opening Remarks

Discussing Critical Factors Influencing Potential Reimbursement Approaches

Keynote: Establishing Value for Gene Therapy Products by Implementing Effective Reimbursement Strategies

  • Sven Kili VP, Head of Cell & Gene Therapy Development, GSK


  • Considering the patient and carers with broader implications, not just the therapy in reimbursement planning
  • Investigating new reimbursement models for gene therapy – strengths and weaknesses
  • Learning first hand from experiences in pricing Strimvelis in the European landscape

The Payer Perspective: Glybera Insights – What are the Key Takeaways?

  • Detlev Parow Head, Department of Care Management Development, DAK


  • Pricing of gene therapy products – is there a ceiling price and how can this be identified?
  • Assessing three different gene therapeutic approaches – key factors to consider e.g. treatment setting and regulatory framework
  • Evaluating the evidence required with limited patient populations in the rare disease space

Options for Managing Budget Impact of High Cost Gene Therapies


  • Understanding the challenges of reimbursement for high cost gene therapies
  • Investigating different approaches to manage risk – discussing the relative benefits and drawbacks of annualisation and amortisation strategies

Multi-Stakeholder Panel on Executing Effective Pricing, Reimbursement & Market Access Approaches

  • Detlev Parow Head, Department of Care Management Development, DAK
  • William Green Senior Research Consultant, York Health Economics Consortium
  • Sven Kili VP, Head of Cell & Gene Therapy Development, GSK
  • Diego Ardigo Project Leader - Advanced Therapy Medicinal Product, Biologics, Corporate Drug Development, Research & Development, Chiesi


  • Recognising how payers view value delivered and discussing what they are prepared to pay for
  • Identifying the full impacts of the therapy to ensure the full costs of the disease are recognised – such as hospital costs, ancillary costs, transport, impact on other family members – rather than simply the costs of the medicine
  • Considering the use and setting of the therapeutic and how this will impact on reimbursement

Morning Refreshments & Networking

Enhancing Process Development & Manufacturing to Prepare for Commercialisation

Optimising Vectorology, Process Development & Cross-Industry Standardisation


  • Establishing effective vector optimisation programs
  • Improving interactions between research, nonclinical development and process development teams
  • Discussing the necessity of cross-industry standardisation of development efforts in this space

Enhancing the Manufacturability of Gene Therapies

  • Joseph Earley Senior Scientist, Downstream Process Development, Allergan


  • Understanding the key challenges and issues to consider prior to scale-up
  • Evaluating the equipment requirements and how the manufacture of viruses contrasts with other fields
  • Investigating strategies to bypass the requirement of ultracentrifuges

Improving the Scalability of Gene Therapy Manufacturing


  • Overcoming challenges encountered in transitioning from lab scale to late-stage GMP commercial scale manufacturing
  • Examining the impact of vector choice on scalability – comparing serotypes and overcoming the challenges experienced in the scale up of self-complementary vectors
  • Controlling the cost of goods throughout scale up to guarantee cost-effectiveness

Lunch & Networking

Investigating the Funding Opportunities Available in Europe

Identifying Opportunities to Translate Gene Therapy in Academia & Key Factors to Address Using Case Study Examples


  • Understanding the de-risking and evidence required to support the commercial route forward
  • Analysing the capacity, capability and cost issues related to gene therapy development
  • Learning about the funding opportunities available in this field

Panel Discussion: How do Investors View the Gene Therapy Space?


  • How flexible are VCs in this space?
  • Do investors look for a focussed or broad pipeline when evaluating gene therapy companies?
  • Contrasting the funding scene in Europe compared to the US
  • Do VCs view gene therapy companies as platforms which can be adapted based on changes in gene of interest or project-based investments?
  • Understanding the scientific criteria investors use to determine funding decisions

Afternoon Refreshments & Networking

Strengthening Collaborations Between Academia & Industry

Uniting the Rare Disease Community Around Key Patient Affairs and Policy Objectives

  • Samantha Parker Chief Patient Affairs & Health Policies Officer, Lysogene


  • Understanding the specific and unique characteristics of the rare disease space
  • Learning how to best involve patients, industry leaders and regulators to map out policies and guidelines in this area
  • Sharing case studies investigating Lysogene’s work in this space

Panel Discussion: How can Organisations Forge Mutually Beneficial Alliances in the Gene Therapy Field?

  • Adrien Lemoine VP, Business Development & Operations, Orchard Therapeutics
  • Emily Culme-Seymour External Strategy Manager, Gene Therapy, Rare Diseases Unit, GSK
  • Michela Gabaldo Head, Alliance Management & Regulatory Affairs, TIGET
  • Magda Papadaki Head, Manufacturing Innovation, The Association of the British Pharmaceutical Industry (ABPI)


  • Evaluating the nature of deal structures and collaborations between biopharma companies, academic institutions and solution provider companies– how have they evolved and how can they be improved?
  • How can organisations in the field collaborate more effectively to bring therapies to patients more rapidly?

Chair’s Closing Remarks

Close of Conference