About Event

What You Missed:

Collectively Changing the Paradigm for Rare Disease Treatment


As the industry-leading rare gene therapy meeting, the Gene Therapy for Rare Disorders is back at a landmark moment for the field.

Through a comprehensive program filled with keynote plenaries, interactive group sessions, and focused discussion days, all carefully co-curated with industry leading figures, no matter which challenges your team are currently grappling with, we’ve got you covered.

Across 4 days and 4 tracks, 70+ expert speakers will be sharing expertise and advances in:




Market Preparedness

After a successful year of approvals and with even more decisions anticipated for 2023, improving clarity over regulatory expectations and maintaining an open dialogue between industry and regulatory bodies remains paramount. We will:

  • Bring together multinational regulatory bodies, including the FDA, MHRA and PMDA, to better understand the global regulatory landscape
  • Enhance regulatory understanding to better inform clinical and manufacturing programmes
  • Navigate new regulatory designations and accelerated pathways

Over 1100 and 300 gene therapies are currently navigating preclinical and clinical development respectively. To help progress your product into and through the clinic, our program will dive into:

  • Designing and implementing safe and effective clinical trials, understanding the process behind defining primary endpoints
  • The operational intricacies behind rare gene therapy trials
  • Gearing up for registrational clinical trials

A common bottleneck during regulatory interactions is in the manufacturing side of gene therapy programs. Working towards robust and scalable manufacturing processes, this track will be dedicated to:


  • Leveraging innovative platforms, using the latest advances in technology to improve manufacturing efficiency
  • Navigating key bottlenecks in rare disease CMC to scale up effectively
  • Demonstrating comparability to regulatory bodies in light of process changes

As more gene therapy products progress through to pivotal trials and near approval, being prepared for launch and what lies ahead is now a nearing reality for many. Insights at the meeting will enable you to:


  • Define the value of your rare gene therapy
  • Effectively set up your cross-functional team to be prepared for launch
  • Ensure rare disease patients have access to gene therapies, and the practicalities surrounding early access