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October 22-24, 2018
London, UK




Conference Day One
Tuesday 23rd October, 2018

Conference Day Two
Wednesday, 24th October, 2018

Registration, Coffee & Networking

Chair’s Opening Remarks

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

Establishing Value for Gene Therapy Products: Discussing Reimbursement, Market Access & Pricing Considerations

How Will Value Frameworks and Budget Impact Analysis be Used to Evaluate Gene Therapies?


  • Understanding the challenges of reimbursement for high cost gene therapies
  • Balancing near-term budget impact with long-term value – what mechanisms are in place to evaluate this balance?
  • Investigating various approaches to manage risk – discussing the relative benefits and drawbacks of annualisation and amortisation strategies

The Payer Perspective: Investigating how Gene Therapies Require a Fundamental Change in Healthcare Delivery

  • Detlev Parow Head, Pharmaceuticals Department , DAK-Gesundheit


  • Contrasting gene therapies with existing therapeutics – what are the key differences and how will European healthcare systems need to adapt?
  • Understanding the key aspects involved in the establishment of treatment centres
  • Is this a sustainable model and what are the alternatives?


Panel Discussion: Evaluating Value-Based Pricing Models for Gene Therapy Products


  • Investigating real-world learnings from European pricing models to incorporate into program designs
  • Beyond the overview – examining at what threshold the budget impact is it even useful or meaningful examine payment over time and performance based models rather than a one-off payment
  • Investigating the challenges in reducing payment models to practice, in terms of:
    • Data collection
    • Patient tracking
    • US versus EU experiences
    • Single payer versus multi payer systems

Setting the Scene: Regulatory Insights & Nuances


This presentation will use a past experience and a recent experience of product development through to authorisation, focusing on:

  • Challenges of clinical studies with rare diseases
  • Impact of indication changes during review

What does a global approach look like?

Speed Networking & Morning Refreshments

A Call to Arms for Standardisation

Establishing Effective Manufacturing & Regulatory Standards

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


  • Defining the elements of gene therapy development that need to be standardised
  • Investigating strategies to overcome analytical limitations to make comparability easier
  • Establishing effective comparability practices
  • Call to arms for regenerative medicine community

Defining & Executing Robust Clincal Development Strategies

Incorporating Patient Input & Patient Reported Outcome Measures: A Fabry Clinical Trial Example


  • Considerations for use of PRO Measures in clinical trials
  • Example of utilising a PRO Measure designed for one disease in another disease
  • Collaboration with patient groups regarding PRO Measures

Developing & Commercializing Novel One-Time Treatments for Patients Suffering from Rare Inherited Retinal Diseases that would Otherwise Progress To Blindness – the Nightstar Approach

  • Samantha Vieira Senior Director, Programme Management, Nightstar Therapeutics


  • Nightstar is gene therapy company solely focused on treating inherited retinal diseases that would otherwise lead to blindness
  • Lead clinical candidate, NSR-REP1 being studied in Phase 3 registrational trial for Choroideremia with one year data expected in 2020
  • Second clinical candidate, NSR-RPGR, being studied in Phase 1/2 trial with preliminary dose escalation data expected Q3’2018

Lunch & Networking

Investigating the Funding Opportunities Available in Europe

Panel Discussion: Gaining Insights into How Investors View the Gene Therapy Space


  • Contrasting the funding scenes in Europe compared to the US
  • What do VCs like to see and what resonates with them in their investment strategy or do they appraise programs on an individual basis?
  • Understanding whether investors see gene therapies as a platform play or more project based
  • How flexible are VCs and do they look for a focused or broad pipeline?

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

Establishing Mutually Beneficial Partnerships to Achieve Commercial Potential

Panel Discussion: Understanding Unique Challenges Involved in Developing Gene Therapies in a Biotech Environment


  • Understanding how gene therapy biotechs can work effectively with academic institutions to drive progress
  • How can small companies without a global reach approach issues like patient recruitment across the world and cross-border legislation?
  • Do companies need to change their hiring strategies to deal with the requirements of the gene therapy space?
  • What are the new types of infrastructures required?

Panel Discussion: Strategic Partnerships to Achieve Commercialisation: What are the Trends & What Does the Future Look Like?


  • Gaining strategic insights from recent partnering and acquisitions in the field
  • Understanding how pharma and biotech companies can work effectively together to develop gene therapies on a global scale
  • Pros and cons of going it alone versus partnering
  • Investigating the value that needs to be demonstrated to attract interest from larger entities

Chair’s Closing Remarks

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

End of Conference Day One