Rare and Ultra Rare Diseases | GCT Breaks Through - 2021 World Medical Innovation Forum
May 2–4, 2022

Rare and Ultra Rare Diseases | GCT Breaks Through

Rare and Ultra Rare Diseases | GCT Breaks Through

Thursday, May 20, 2021

11:10 AM – 11:35 AM

One of the most innovative segments in all of healthcare is the development of GCT driven therapies for rare and ultra-rare diseases. Driven by a series of insights and tools and funded in part by disease focused foundations, philanthropists and abundant venture funding disease after disease is yielding to new GCT technology. These often become platforms to address more prevalent diseases. The goal of making these breakthroughs routine and affordable is challenged by a range of issues including clinical trial design and pricing.

  • What is driving the interest in rare diseases?
  • What are the biggest barriers to making breakthroughs ‘routine and affordable?’
  • What is the role of retrospective and prospective natural history studies in rare disease?  When does the expected value of retrospective disease history studies justify the cost?
  • Related to the first question, what is the FDA expecting as far as controls in clinical trials for rare diseases?  How does this impact the collection of natural history data?
Susan Slaugenhaupt, PhD
  • Scientific Director and Elizabeth G. Riley and Daniel E. Smith Jr., Endowed Chair, Mass General Research Institute
  • Professor, Neurology, HMS
Leah Bloom, PhD
  • SVP, External Innovation and Strategic Alliances, Novartis Gene Therapies
Bobby Gaspar, MD, PhD
  • CEO, Orchard Therapeutics
Emil Kakkis, MD, PhD
  • CEO, Ultragenyx
Stuart Peltz, PhD
  • CEO, PTC Therapeutics
  • Q&A

    11:40 AM – 11:55 AM

the Future of Medicine and AI

May 11–13, 2020 | Boston, MA