Developing Gene Therapies For Ultra-Rare Diseases

Time: 9:00 am - 12:00 pm
day: Workshop D


Several of the challenges in rare disease, including patient identification, trial design, and patient access to therapies are all exacerbated when looking to develop gene therapies for ultra-rare diseases. This workshop will delve into the challenges faced by ultra-rare gene therapy development to set up a process going forward to bring these transformative therapies more feasibly to patients in need.

Attend this workshop to learn about:

  • Exploring regulatory guidelines in the context of ultra-rare diseases
  • Utilizing natural history studies and designing trials for even smaller patient numbers
  • Understanding commercial viability for ultra-rare gene therapy development