Welcome to the 4th 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 commercial potential.

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


With larger populations and higher doses required, manufacturing issues are more prominent than ever. That’s why the 2020 agenda will devote two tracks solely to overcoming manufacturing issues, enabling you to:

  • Meet regulatory expectations for CMC and analytical packages
  • Implement robust and scalable manufacturing processes
  • Handle the rapidly evolving logistical and infrastructure challenges facing the space


More gene therapies are in the clinic than ever before. In the context of this unprecedented progress, the event will give significant attention to clinical issues including:

  • Maximizing meaningful output from trials through creative clinical trial design
  • Incorporating the patient voice into clinical development
  • Navigating the realities of the global regulatory landscape in light of recent approvals


How do you price gene therapies? This question and issues around reimbursement, payment models and the commercial realities of the space will form the basis of the commercial track, with insights that will enable you to:

  • Understand the payer perspective on balancing access and affordability
  • Contrast a variety of payment models including outcome- and performance-based payments
  • Evaluating the commercial realities of rare and ultra-rare disease business models