Changing What is Possible for Angelman Syndrome

FAST IMPACT AT A GLANCE

When FAST was founded in 2008, promising Angelman syndrome research faced significant barriers to becoming potential treatments.

Families understood that scientific promise alone would not lead to treatments. They created FAST to actively drive the work forward, bringing the urgency, resources, and determination needed to advance research toward therapeutic development.

Today, the landscape looks very different. FAST has helped advance therapeutic programs, create new pathways for drug development, and build the research tools, partnerships, and infrastructure needed to bring promising science closer to meaningful treatments.

How FAST is Changing Angelman Syndrome Research

FAST’s impact extends beyond funding scientific discoveries. From advancing therapeutic programs to building the tools needed for clinical trials, FAST has helped address critical gaps in research and development while ensuring families have a role in shaping what comes next.

Scientific Promise is Becoming Therapeutic Momentum

Guided by the Roadmap to a Cure and backed by more than $64M invested in research grants and mission-aligned investments, FAST helps promising discoveries take the critical steps toward therapeutic development.

First In-Human Progress

FAST-funded research helped bring two groundbreaking therapeutic approaches into human clinical trials for the first time, including the first investigational gene therapy.

A Broader Therapeutic Pipeline

The Angelman syndrome drug development has never had more momentum than it has today. →
The pipeline now includes 26 potential therapeutic programs, including 13 that are FAST-funded. Four investigational therapeutics are currently in ongoing clinical trials, with over 200 patients dosed to date.
See the pipeline

Leading Scientists Join the Work

Angelman syndrome now attracts leading researchers in genetics, gene therapy, and stem-cell science. →
FAST helps bring leading scientific minds into Angelman syndrome research, including Angelman syndrome genetics pioneers Art Beaudet (Baylor College of Medicine), Yong-Hui Jiang (Yale University), Jim Wilson (formerly University of Pennsylvania, now Gemma Biotherapeutics), Frances Arnold (Caltech), and Don Kohn (UCLA), while also supporting the next generation of Angelman syndrome researchers through ASGCT.
Explore FAST-funded research

A New Model for Therapeutic Development

FAST created AS²Bio to help promising gene-targeted therapies keep moving when traditional development paths are not ready. →
AS²Bio's portfolio includes MavriX Bio, whose MVX-220 is the first investigational AAV gene therapy for Angelman syndrome, and CourageAS Bio, which received major federal support through the NIH.
Learn about AS²Bio

Impact Beyond Angelman Syndrome

What began as narrow rare-disease research is now helping answer some of the hardest questions in brain medicine. →
FAST has helped move Angelman syndrome from a little-known rare disease to one of the most active and closely watched areas of brain-focused therapeutic development, with that same work now showing promise for autism, epilepsy, Alzheimer's, and Parkinson's disease.
Partner with us

The Field Can Measure, Study, and Prepare in Ways It Could Not Before

FAST has built the tools, data, and clinical capacity to design stronger studies and accelerate progress.

A Clearer Way to Measure Progress

Researchers now have stronger ways to measure whether a potential treatment is making a meaningful difference. →
A-BOM is helping develop and evaluate measures of meaningful change in Angelman syndrome, giving researchers better tools for clinical studies.
Learn more about A-BOM

A Brain Trust for Angelman Syndrome

Experts from across disciplines are working together on the scientific problems no single lab can solve alone. →
INSYNC-AS brings specialists together to tackle questions that can affect therapeutic development, from measurement to delivery and clinical readiness.

Long-Term Data for Better Studies

Two decades of natural history data give researchers a clearer view of how Angelman syndrome changes over time. →
The Angelman Syndrome Natural History Study helps inform study design and the evaluation of meaningful change across ages and genotypes.
Learn more about NHS

Families Have a Stronger Voice in What Comes Next

Families and caregivers understand what meaningful change could look like for their loved ones living with Angelman syndrome.

FAST helps bring that experience into research, regulatory, and policy conversations while giving families trusted guidance as the science moves forward.

Daily Life Becomes Regulatory Evidence

Family experience is helping define what meaningful change looks like in Angelman syndrome. →
FAST and the Angelman Syndrome Foundation hosted an Externally-Led Patient-Focused Drug Development meeting that led to a Voice of the Patient Report submitted to the FDA. The report captures how Angelman syndrome affects daily life and what families consider meaningful change, creating a formal resource for regulators and therapy developers.
Learn more

Angelman Syndrome Has a Voice on Capitol Hill

Families are bringing Angelman syndrome directly to federal lawmakers. →
Through Congressional Advocacy Day, FAST and the Angelman Syndrome Foundation help advocates meet with policymakers and raise priorities affecting research, rare disease policy, and the Angelman syndrome community.
Learn more

A Stronger Voice in Regulatory Decisions

Angelman syndrome is represented in regulatory conversations that can shape how potential treatments are developed and evaluated. →
FAST engages with the FDA on issues affecting rare disease therapeutic development and brings the realities and priorities of the Angelman syndrome community into those discussions.
Learn more

Angelman Syndrome Progress Is Now Globally Connected

FAST helps connect families, affiliates, researchers, advocates, fundraisers, and partners across borders so Angelman syndrome progress is not limited by geography.

A Growing Global Network

Newly diagnosed families used to navigate this alone. Now, FAST affiliates connect Angelman syndrome communities across 22 countries. →
FAST’s nine global affiliates help expand local education, advocacy, fundraising, and family connection while linking communities to a broader international network.
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A Global Voice in What Comes Next

Family perspectives are reaching international conversations about research, policy, and future access to treatments. →
Through efforts connected to EURORDIS and the Angelman Syndrome Global Community Advisory Board, FAST helps ensure that the experiences and priorities of families are represented in conversations about research, policy, and access beyond the United States.

Global Family Data for Research

Families around the world are contributing data that is strengthening Angelman syndrome research. →
No single country ever had enough data to answer Angelman syndrome's hardest questions. Now, the Global Angelman Syndrome Registry, Led by FAST Australia, brings together data from more than 2,000 families across nearly 100 countries.
Learn more

The Reason This Work Matters

Behind every program, tool, partnership, and investment is a family waiting for progress to reach the people they love.

For families facing Angelman syndrome, research is not abstract. It is connected to communication, sleep, seizures, movement, learning, independence, and quality of life. It is connected to the hope that tomorrow can look different than today.

That is why FAST’s work is built around urgency. Not urgency for its own sake, but urgency in service of the people and families waiting for meaningful progress.

From Progress to Possibility

Behind every milestone is a family waiting for progress to become something real.

FAST’s impact is measured by what that progress makes possible: more therapeutic paths, stronger research tools, deeper clinical readiness, greater visibility with decision-makers, and a global community moving with urgency and purpose.

There is still more work ahead. But Angelman syndrome research is no longer where it once was, and FAST is working to keep that progress moving.

Find Your Role in What Comes Next

Different people come to FAST for different reasons. Whether you are here to give, learn, connect, or partner, your next step can help keep Angelman syndrome progress moving.

If you are considering a gift

Your support helps fund the research, tools, data, infrastructure, and partnerships needed to move promising science forward.

Donate

If you are newly diagnosed

You are not doing this alone. FAST can help you find trusted information, connect with other families, and understand the research progress shaping the future of Angelman syndrome.

Get Connected

If you are here for the science

Angelman syndrome research is moving faster than ever. See where the field stands today and how FAST helps turn scientific promise into therapeutic momentum.

Explore the Pipeline Read Our Research

If you are a researcher, clinician, or industry partner

FAST helps build the tools, data, funding pathways, and global connections that reduce uncertainty and strengthen decision-making across Angelman syndrome research.

Partner with FAST View Funded Research

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