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

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

Phase 3 Aspire trial of gene therapy GTX-102 doses 1st patient

31/01/2025 No Comments

New trial, Aurora, to test GTX-102 in other Angelman genotypes, age groups

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.

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Foundation For Angelman Syndrome Therapeutics UK Charity 1167984 ICO Registered ZA672029

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