Welcome to the 5th Annual Gene Therapy for Rare Disorders Europe

Gene therapies are revolutionising the treatment of a host of devastating diseases, giving hope to patients who previously had no therapeutic options. However, there are still unique hurdles that must be overcome to bring these therapies to patients more quickly and effectively.

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

Manufacturing

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:

  • Establishing effective analytical tools and improving characterisation of gene therapy products
  • Refining upstream development to increase yield and quality
  • Enhancing downstream purification to align with regulatory expectations and become more cost-effective
  • Making strategic decisions within a rapidly evolving framework

Clinical & Commercial 

More gene therapies are in the clinic than ever before, and several are heading towards approval in the coming 24 months. This track will focus on key issues across clinical and commercial development, including:

  • Navigating gene therapy safety in clinical development
  • Establishing novel payment models in a European market
  • Exploring market access strategies and building value early on
  • Building a global clinical trial for rare disease patients