Welcome to the 5th Annual Gene Therapy for Rare Disorders

Gene therapies are redefining the treatment of rare diseases. However, a set of complex and unique challenges must be overcome for these therapeutics to achieve their full potential.

No matter which of these challenges you’re currently grappling with, the meeting will offer a wealth of relevant content for you:

Analytics & Process Development:

The ability to manufacture high quality products at a high yield are key to achieving robust and cost-effective manufacturing. This track will focus on highly technical content surrounding:

  • Designing appropriate analytical tools to measure product safety and efficacy
  • Optimizing upstream and downstream process development
  • Implementing higher quality, higher yielding manufacturing processes

Commercial Manufacturing:

With several companies in the midst of clinical trials and gearing up for commercial manufacturing, it’s vital to have robust and scalable manufacturing processes in place. This track will dedicate significant time to:

  • Transitioning from clinical to commercial manufacturing
  • Making strategic manufacturing decisions on capacity and capabilities
  • Addressing large-scale manufacturing bottlenecks


More gene therapies are in the clinic than ever before, so we’ll be addressing key issues across clinical development, including:

  • Addressing safety in clinical development
  • Building and implementing innovative clinical trials with meaning to patients and regulators
  • Incorporating patient perspectives across development


As regulators gear up for more gene therapy approvals on the horizon, it’s clear that a comprehensive insight into regulatory expectations is key to success. We will delve into:

  • Working towards global regulatory harmonization across agencies
  • Understanding current regulatory expectations on clinical trials and CMC submissions
  • Interacting with regulatory agencies to clarify timelines

Market Access:

The significant efforts being made to approve safe, effective gene therapies will be meaningless without a way to ensure market access of these transformative treatments. Insights at the meeting will enable you to:

  • Understand the payer perspective to access and affordability of gene therapies
  • Build innovative payment models based on gene therapy value
  • Navigate the practicalities of market access