At FAST, it’s our job to push forward all promising Angelman syndrome (AS) programs, and to ensure that the community has a birds-eye view of the entire landscape. Many of these programs have been, or still are, funded by FAST, but this chart also includes those that aren’t (but are disclosed). The progress made is truly promising.
This pipeline is a snapshot in time as of July 2026, please click on the Live Progress Updates below for the most recent pipeline progress..
Following Ultragenyx’s announcement that its Phase 3 Aspire trial of GTX-102 did not meet its primary or key secondary endpoints, the Angelman Syndrome Foundation (ASF)
MavriX Bio announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) to its investigational gene therapy, MVX-220. Rare Pediatric Disease Designation is granted
Ionis has shared an important update with the Angelman syndrome community about the design of CHAMPION, its Phase 3 clinical trial evaluating ION582 (obudanersen). The
We found solace and purpose through FAST. Their relentless pursuit of a cure resonated with us, and we started our fundraising journey with them.