Drug Development Pipeline

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 January 2025, please view the cureangelman.org website for the most recent pipeline progress..

Scientific News

Oak Hill Bio has shared a community letter to the Angelman syndrome community

28/05/2026 No Comments

Oak Hill Bio has shared a community letter to the Angelman syndrome community with an important update on the next steps of the development of

MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome

05/12/2025 No Comments

MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the U.S. Food

FAST Global Search & Rescue Initiative Joins the Global Angelman Syndrome Registry

03/03/2025 No Comments

A Unified Effort to Connect Families, Accelerate Research, and Find a Cure for Angelman Syndrome

Heather Brady

We found solace and purpose through FAST. Their relentless pursuit of a cure resonated with us, and we started our fundraising journey with them.

Drug Development Pipeline

Scientific News

Oak Hill Bio has shared a community letter to the Angelman syndrome community

MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome

FAST Global Search & Rescue Initiative Joins the Global Angelman Syndrome Registry

Heather Brady

Learn about Angelman Syndrome

Make a Dontation Now

Start a Fundraising Campaign