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31 October – 2 November , 2017

London, UK



Day One
Wednesday 1st November, 2017

Day Two
Thursday 2nd November, 2017

Registration, Coffee & Networking

Chair’s Opening Remarks

Keynote: Investigating Critical Challenges in the Gene Therapy Field – Mapping out the Landscape & Discussing Future Strategies


  • Understanding factors contributing to the clinical efficacy of gene therapies and investigating strategies to enhance this efficacy
  • Outlining the case for different reimbursement models and discussing potential future approaches
  • Improving the safety of gene therapies and investigating novel technologies and platforms to reduce immunogenicity while maintaining efficacy

Keynote Panel Discussion – The Future of Gene Therapies for Rare Disease


After decades of unfulfilled potential, gene therapies are gaining momentum and hold genuine promise in treating a series of previously incurable diseases. Clearly a significant amount of progress has been made in this field in Europe, but how can we ensure that future gene therapies do not encounter the same commercial hurdles that have been experienced in the past?

  • Assessing the unique considerations for development in the rare and ultra-rare disease space – how can companies meet these demands?
  • Bridging the gap between academia and early proof-of-concept studies and commercialisation
  • Considering approaches to create value in clinical programs from the early stages

Investigating Novel Vector Technology with the Promise of Enhancing the Efficacy of Gene Therapy


  • Learning about the limitations of current vector technology – where can improvements be made regarding expected benefits?
  • Strategies to enhance the tissue targeting of gene therapies of in vivo AAV– how to deliver a greater quantity of therapeutic to a specific tissue
  • Minimising the complications associated with safety and potential re-dosing by examining novel delivery technologies

Speed Networking & Morning Refreshments

Enhancing the Speed & Robustness of Clinical Development

The Selection of Endpoints for Ophthalmology Gene Therapy Trials in the Rare Disease Space


  • What are the key endpoints being explored at the moment and in the future?
  • What endpoints are realistically measurable in the timescale?
  • Investigating the regulators’ feedback  relating to these endpoints

Discussing how we can Define and Maintain Clinical Standards – Is Gene Therapy a Special Case?

  • Wim Scheele Executive Director, Rare Disease Clinical Development & Operations, Pfizer


  • Introducing the protocols and clinical practices that need to be in place to avert safety concerns in gene therapy trials
  • Understanding the importance of per-patient verification and requirements for clinical development
  • Setting the standard – introducing the ICH guidelines are understanding how they can be adhered to correctly in the gene therapy field

Lunch & Networking

Interactive Roundtable Discussions: Drilling into Key Areas of the Gene Therapy Space


Drive your own learning and crowd-source ideas and discover multiple perspectives on the key issues affecting your development efforts in the gene therapy space. Join roundtable discussions that have been specifically designed to enable you to leave with insights that you can immediately implement into your own drug development programs.

  1. Enhancing manufacturing practices
  2. Optimising clinical development
  3. Gaining clarity of the gene therapy regulatory framework
  4. Executing effective reimbursement and pricing strategies

Following discussions in the intimate and open roundtable format, the table leaders will form a panel to feed back to the entire audience on the key topics that were discussed in the session. Form a comprehensive understanding of the gene therapy development and commercialisation process and the strategies that are employed to overcome challenges at every point along the way.

Afternoon Refreshments & Networking

Navigating the Complex Regulatory Landscape

MHRA Perspective on Gene Therapies for Rare Disorders

  • Christiane Niederlaender CAT Member, Senior Quality Assessor, UK Medicines and Healthcare Products Regulatory Agency (MHRA)


  • Learning what the regulators are looking for in this field
  • Understanding how to engage the regulators most effectively at critical time points in the development of gene therapy products
  • How regulatory guidelines impact on every stage of the gene therapy development process

Registering a Cell-Based Gene Therapy Product – CMC Considerations


  • Understanding the guidance, legislation and other regulatory requirements you should consider
  • Overcoming practical CMC challenges for autologous cell-based products
  • Sharing CMC lessons learnt from the Strimvelis MAA

Investigating the Regulatory Perspective on Clinical Development, Manufacturing & Market Approval

  • Michela Gabaldo Head, Alliance Management & Regulatory Affairs, TIGET
  • Christiane Niederlaender CAT Member, Senior Quality Assessor, UK Medicines and Healthcare Products Regulatory Agency (MHRA)
  • Gopalan Narayanan VP, Disruptive Biologics, Voisin Consulting
  • Magda Papadaki Head, Manufacturing Innovation, The Association of the British Pharmaceutical Industry (ABPI)


  • Understanding the variations in clinical trial application of regulations across Europe
  • What do you need to demonstrate in order to gain accelerated approval?
  • Learning about the benefits and drawbacks of conducting commercial reviews with the regulators compared to national health bodies in various EU countries

Chair’s Closing Remarks

End of Conference Day One