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