Welcome to the 4th Annual Gene Therapy for Rare Disorders Europe

Gene therapies are revolutionising rare disease treatment. However, there are still unique hurdles that must be overcome to bring these therapies to patients more quickly and effectively.

At this year's fully digital meeting, we are delivering three tracks of comprehensive content to support every stage of your gene therapy development:


As gene therapies continue to develop, the pressure on manufacturing to meet demand is more apparent than ever. This track will address key topics to overcome these manufacturing bottlenecks, including:

  • Scaling effectively to meet gene therapy demands
  • Developing effective characterisation and analytical tools to support development
  • Making strategic decisions within a rapidly evolving framework


More gene therapies are in the clinic than ever before. Given these exciting developments, this track will focus on key clinical issues in the gene therapy space for rare disease, including:

  • Optimising clinical trial design and recruitment
  • Navigating the fragmented European regulatory landscape
  • Developing patient-centric gene therapy programs


With more gene therapy approvals expected in 2020, questions surrounding pricing, reimbursement, payer perspectives and patient access in Europe will be central to the commercial track, with insights on:

  • Understanding the payer perspective to gene therapies
  • Exploring different pricing and reimbursement strategies
  • Breaking down market access barriers to bring therapies to patients