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October 22-24, 2018
London, UK

 

 

 

Conference Day One
Tuesday 23rd October, 2018

Conference Day Two
Wednesday, 24th October, 2018

08.50
Chair’s Opening Remarks

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

09.00
Scaling Manufacturing to Deal with the Demands of Commercial Gene Therapy Products

Synopsis

  • 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

09.30
Developing Effective Process Characterisation & Analytical Tools

Synopsis

  • 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

10.00
Finding the Right Dose From Mice to Human: A Complex Equation

Synopsis

  • 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

10.30
Morning Refreshments & Networking

11.00
Navigating the Capacity Bottleneck – Contrasting In-House and External Manufacturing Options & Understanding the Shire Approach

Synopsis

  • 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

11.30
Panel Discussion: The Next Step – Pioneering Gene Therapies in More Common Diseases

Synopsis

  • 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

12.15
Lunch & Networking

13.15
Presentation Details To Be Confirmed

Navigating the Complex European Regulatory Landscape

13.45
Engaging European Regulators Around Key Issues Throughout the Gene Therapy Development Process

  • Robert Morgan Executive Director, Regulatory Affairs, Nightstar Therapeutics

Synopsis

  • 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

14.15
Afternoon Refreshments & Networking

14.45
Tackling the Additional Complexities of GMO Applications to Save Crucial Development Time

Synopsis

  • 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

15.15
Managing Late-Stage Gene Therapy Development in Preparation for MA/BLA Applications

Synopsis

  • 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

15.45
Chair’s Closing Remarks

16.30
Close of Conference