LinkedIn Icon
October 22-24, 2018
London, UK




Conference Day One
Tuesday 23rd October, 2018

Conference Day Two
Wednesday, 24th October, 2018

Chair’s Opening Remarks

Supporting Pioneering Gene Therapy Development by Enhancing Manufacturability, Dose Selection & Adding to the Analytical Toolbox

Scaling Manufacturing to Deal with the Demands of Commercial Gene Therapy Products


  • Establishing robust and scalable manufacturing processes early in development
  • Investigating novel manufacturing technologies to improve efficiency at scale
  • Examining the impact of vector choice on scalability – comparing serotypes and overcoming the challenges experienced in the scale up of self-complementary vectors
  • Understanding how to control cost of goods throughout scale-up

Developing Effective Process Characterisation & Analytical Tools


  • Improving the analytical tool box: Understanding the most effective analytical approaches for gene therapy products
  • Understanding analytical challenges specific to the rare disease space
  • Refining processes to deal with low concentrations

Finding the Right Dose From Mice to Human: A Complex Equation


  • Complexity of first in human with therapeutic objectives versus safety
  • How to calculate the dose and translation from mice to monkey and then human
  • Impact of pre-existing immunity?
  • Perspectives for re-dosing?
  • Regulatory perspectives

Morning Refreshments & Networking

Navigating the Capacity Bottleneck – Contrasting In-House and External Manufacturing Options & Understanding the Shire Approach


  • Evaluating the long-term strategic impacts of internal vs external manufacturing decisions
  • Case studies outlining the various advantages and disadvantages of internal and external manufacturing
  • Complimenting drug development activities with in-house manufacturing to drive forward the gene therapy field as a whole

Investigating the Indications that Will Allow Gene Therapies to Achieve their Full Potential

Panel Discussion: The Next Step – Pioneering Gene Therapies in More Common Diseases


  • Understanding the challenges involved in transitioning from rare to more common disease indications
  • Will we soon run out of economically viable monogenic diseases to treat with gene therapy?
  • Highlighting the incentives to build gene therapy platforms in rare indications but looking towards the future treatment of more common diseases
    • The investor view – giving a long-term objective to initial financing
    • The pharma view – demonstrating platform capabilities
  • Investigating how gene therapies would potentially compete with small molecule and antibody approaches in more mainstream indications

Lunch & Networking

Presentation Details To Be Confirmed

Navigating the Complex European Regulatory Landscape

Engaging European Regulators Around Key Issues Throughout the Gene Therapy Development Process

  • Robert Morgan Executive Director, Regulatory Affairs, Nightstar Therapeutics


  • Importance of partnering with regulators throughout the development pathway for gene therapy products
  • Understanding how and when to engage the Authorities most effectively, with objective of agreeing a harmonised global approach
  • How regulatory guidelines impact throughout the development pathway

Afternoon Refreshments & Networking

Tackling the Additional Complexities of GMO Applications to Save Crucial Development Time


  • Investigating differences in GMO applications in different member states
  • Understanding what can be done in parallel and what can be done sequentially
  • Broadening initial GMO submissions to expand beyond small-scale phase 1 studies into later stages of development
  • Navigating cross-border legislation and differences in regulatory landscape across Europe and tapping into local knowledge to select the best locations for trials

Managing Late-Stage Gene Therapy Development in Preparation for MA/BLA Applications


  • Contrasting late-stage regulatory approaches to consider in the US vs European landscapes
  • Analysing features unique to the rare disease space
  • Integrating manufacturing and clinical development in regulatory strategies

Chair’s Closing Remarks

Close of Conference