Our Strategic Approach
To achieve a future with multiple approved therapeutics, we need as many shots on goal as possible. Because each of our loved ones living with Angelman syndrome (AS) is different, and each may respond differently to different treatment options, we need a robust and diverse pipeline.
To help guide this work, FAST has organized its efforts into five key pillars – our Roadmap to a Cure. The Roadmap sets a clear and coordinated strategy to drive global research forward, keeping the focus on what matters most: delivering effective and transformative treatments for every person living with Angelman syndrome.
The 5 Pillars of Our Roadmap to a Cure
Pillar 1: Replace Mom’s UBE3A
Pillar 1 consists of therapeutic programs that focus on replacing the missing or non-functional maternal copy of the UBE3A gene or protein in neurons of the brain.
Pillar 2: Turn on Dad’s UBE3A
Pillar 2 consists of therapeutic programs that focus on activating the silent copy of the paternal UBE3A gene in the brain.
Pillar 3: Explore Adjacent Genes
Pillar 3 consists of research on genes nearby UBE3A on chromosome 15 to understand their contribution to Angelman syndrome characteristics and identify additional opportunities for treatment in the genotypes that have other genes impacted (deletion, UPD/ICD).
Pillar 4: Target Downstream Pathways
Pillar 4 consists of therapeutic programs that focus on different molecular pathways and proteins impacted by the missing UBE3A protein.
Pillar 5: Accelerating and Preparing for Clinical Trials
Pillar 5 consists of initiatives that support necessary research tools, clinical activities, and community efforts to prepare and advance the landscape for drug discovery, clinical trials, and drug approvals
The Drug Development Pipeline for Angelman Syndrome
Therapeutic approaches spanning across each Pillar
The field of AS is showcasing what becomes possible when families, researchers, clinicians, and industry unite around a shared goal: moving from understanding a disorder to developing meaningful treatments.
Investigational programs
FAST-funded
Potential therapeutics with ongoing clinical trials
Tools & Resources
Bioresources
Research tools globally available that are geared towards accelerating clinical trial readiness and designing the most robust clinical protocols.
DART E-Journal
An e-journal dedicated to publishing negative or null data has the potential to revolutionize how the scientific community engages with AS research.
Explore More
Many of these programs have been, or still are, funded by FAST, but the pipeline also includes those that aren’t (but are disclosed). The progress made is truly promising.
Learn about the Angelman syndrome therapeutic pipeline, development landscape, and roadmap to future treatments.
FAST advances Angelman syndrome research by funding critical gaps, building shared tools, and accelerating promising science toward treatment.