Foundation for Angelman Syndrome Therapeutics

What Foundation for Angelman Syndrome Therapeutics does

The Foundation for Angelman Syndrome Therapeutics (FAST) is a US 501(c)(3) patient advocacy organization founded in 2008 by Paula Evans and a group of parents of children with Angelman syndrome, with the singular mission of curing Angelman syndrome (AS), a rare neurodevelopmental disorder affecting approximately 1 in 12,000-20,000 people globally. The organization operates a five-pillar translational research strategy covering AAV gene therapy, hematopoietic stem cell gene therapy, antisense oligonucleotides (ASOs), CRISPR/base-editing, artificial transcription factors, and downstream pathway targets, and has become the largest non-governmental funder of AS research worldwide. FAST funds academic research, operates clinical trial readiness infrastructure including the Global Angelman Syndrome Registry (GASR), the 20-year AS Natural History Study (~500 enrolled patients), and the Angelman Syndrome Biomarker and Outcome Measure Consortium (A-BOM), and supports a network of country-level affiliates across nine-plus countries.

FAST's business model is donation- and grant-driven (charitable contributions, gala paddle raises, Team Kick-AS endurance events, and grants), with no commercial product revenue. The organization complements grant-making with direct biotech incubation: it founded GeneTx Biotherapeutics LLC in 2017 (later acquired by Ultragenyx in 2022 for $25M of prior investment), launched the AS²Bio therapeutic accelerator in 2024, and currently wholly-owns biotech subsidiaries MavriX Bio (AAV gene replacement therapy, first patient dosed November 2025), CourageAS Bio (non-viral CRISPR gene editing), and TransformaTx Biotherapeutics (HSC-GT). The organization is governed entirely by parents of AS children, holds Charity Navigator Four-Star and Candid Platinum Transparency recognitions, and operates virtually with staff distributed across the US and global affiliate countries.

Name

Foundation for Angelman Syndrome Therapeutics

Legal name

Foundation for Angelman Syndrome Therapeutics

Website

https://cureangelman.org

Company type

Private

Founded year

2008

Operating status

Operating

Headcount range

11–50 employees

Short description

The Foundation for Angelman Syndrome Therapeutics (FAST) is a 501(c)(3) patient advocacy organization founded in 2008 that funds and drives Angelman syndrome research across gene therapy, ASOs, CRISPR, and small molecules, operating biotech subsidiaries and a global affiliate network to accelerate curative treatments.

Ownership category

akta.pro rank

Product category

Rare Disease Patient Advocacy and Therapeutic Research

NAICS

Voluntary Health Organizations (813212), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)

akta.pro secondary industries

AAV Vector Gene Therapy Developers (HLAAACAA), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ), Clinical Trial Analytics, Risk-Based Monitoring (RBM) & Quality Management (HLACAOAI)

Keywords

• Patient advocacy foundation
• Rare disease research funding
• Gene therapy pipeline
• Clinical trial readiness
• Angelman syndrome therapeutics

Headquarters

HQ city

Illinois City

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B and B2C

Offering type

Services

Cost components

Others, Operations, Technology or R&D, Marketing or Sales, Personnel

Revenue model

1. Donations and Fundraising: FAST generates revenue through donations from individuals, families, and supporters. The organization conducts various fundraising activities including the annual Gala paddle raise, Team Kick-AS marathon fundraising, community events, and direct donation campaigns. All funds are used to advance Angelman syndrome research and support the drug development pipeline.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels9 records

Core offering

FAST is a nonprofit patient advocacy foundation that funds and drives translational research aimed at curing Angelman syndrome. It operates a five-pillar therapeutic roadmap covering gene therapy (AAV, HSC-GT), gene activation (ASOs, CRISPR/base-editing, ATF-ZF, RNA), and small molecule approaches, while managing clinical trial readiness through a global patient registry, natural history study, and A-BOM biomarker consortium. The organization runs fundraising events, congressional advocacy, and the annual Global Science Summit & Gala, and operates biotech subsidiaries (MavriX Bio, CourageAS Bio) through its AS²Bio therapeutic accelerator to advance investigational therapies into the clinic.

Product overview

Foundation for Angelman Syndrome Therapeutics (FAST) is a nonprofit patient advocacy organization that does not offer commercial products or services. Rather, FAST funds and accelerates Angelman syndrome research and drug development programs. Its primary activities include funding scientific research grants, operating a therapeutic development pipeline spanning multiple treatment modalities (AAV gene therapy, ASOs, CRISPR, small molecules), managing biotech subsidiaries (MavriX Bio and CourageAS Bio) launched to advance investigational therapies, maintaining patient registries and natural history studies, and hosting community events such as the annual Global Science Summit. FAST does not manufacture, sell, or license therapeutic products directly to patients.

Differentiator

Problem solved

Functional benefit

Products and services

• Global Angelman Syndrome Registry (GASR) Caregiver-reported patient registry providing demographic and disease data used by FAST and pharmaceutical partners to identify and recruit individuals living with Angelman syndrome for clinical trial participation.
• Angelman Syndrome Biomarker and Outcome Measure Consortium (A-BOM) Multi-stakeholder consortium developing validated biomarkers and clinical outcome measures required to accurately measure drug effects in Angelman syndrome clinical trials.
• Angelman Syndrome Natural History Study Longitudinal natural history study of nearly 500 individuals with Angelman syndrome providing outcome measure data, demographic and clinical characterization that informs clinical trial design and regulatory submissions.
• Global Science Summit & Gala Annual event convening the Angelman syndrome community with researchers, clinicians, and pharmaceutical industry partners for scientific updates, clinical trial information, networking, and fundraising.
• Congressional Advocacy Day Annual legislative advocacy event in which FAST mobilizes affected families to engage U.S. federal legislators on Angelman syndrome research funding and rare disease policy.
• AS²Bio Therapeutic Accelerator FAST's therapeutic accelerator that creates, funds, and manages wholly-owned biotechnology companies advancing Angelman syndrome therapeutics through research, development, and clinical translation.
• Team Kick-AS Endurance Fundraising Community endurance fundraising program in which FAST recruits supporters to run in major international marathons and raise donations for Angelman syndrome research.

Quantifiable outcome

• First patient dosed in first-in-human gene therapy trial (MVX-220) for Angelman syndrome in November 2025
• +5 more outcomes

Named customers1 record

Segments4 records

Ideal customer profiles4 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

25 partnerships are on record, tiered core, flagship and minor.

• Oak Hill BiocoreStrategic or Co-development Partner · 1 April 2025Oak Hill Bio entered into an exclusive license agreement with Roche to obtain global rights for rugonersen, a Phase 3-ready ASO treatment for Angelman syndrome. In July 2025, Oak Hill Bio announced publication of rugonersen Phase 1 TANGELO study results in Nature Medicine, showing evidence of improvement in brain activity and developmental ability in children with AS.
• MavriX BioflagshipStrategic or Co-development Partner · 14 February 2025FAST launched MavriX Bio as a biotechnology company to advance the first AAV-delivered investigational gene replacement therapy for Angelman syndrome (MVX-220). The company builds on cutting-edge research from Dr. Jim Wilson's laboratory at the University of Pennsylvania and is managed through AS²Bio, FAST's therapeutic accelerator.
• CourageAS BioflagshipStrategic or Co-development Partner · 14 February 2025FAST launched CourageAS Bio to advance an investigational non-viral delivered gene-editing program for Angelman syndrome using CRISPR technology. The company is managed through AS²Bio and works with scientific leaders at Yale University, RUSH University, and Dr. Allyson Berent from FAST.
• UCLAcoreStrategic or Co-development Partner · 30 January 2025Dr. Roger Hollis from UCLA received a $5.8 million CIRM translational research award to further support development of hematopoietic stem cell gene therapy for Angelman syndrome. The program is funded by TransformaTx Biotherapeutics, a biotech company founded by FAST. Dr. Donald Kohn leads the research team.
• Brown UniversityminorStrategic or Co-development Partner · 1 March 2024FAST awarded a new FAST-TRAC grant collaboration with Dr. Alvin Huang at Brown University for $150,000 USD to address oligodendroglial dysfunction in Angelman syndrome by targeting estrogen receptor signaling in OPCs.
• AS²BiocoreStrategic or Co-development Partner · 1 January 2024AS²Bio is a therapeutic accelerator launched by FAST to support development of multiple treatment strategies for Angelman syndrome. By fostering and managing biotech companies focused on gene therapy, gene editing, and other innovative approaches, AS²Bio drives translation of scientific breakthroughs into clinical treatments.
• Yale UniversitycoreStrategic or Co-development Partner · 1 October 2023Yale University received a $40 million NIH Common Fund grant (Phase 1: $26.5M, Phase 2: $13.4M contingent) to advance a novel CRISPR-based gene-editing delivery platform (STEP) for Angelman syndrome. The team includes Drs. Yong-Hui Jiang and Jiangbing Zhou. FAST team led by Dr. Allyson Berent and Jennifer Panagoulias supports IND-enabling studies and regulatory strategy.
• Rush University Medical CentercoreStrategic or Co-development Partner · 1 January 2023The RUSH F.A.S.T. Center for Translational Research is a first-of-its-kind clinical trial and translational research center established via a $5 million gift from FAST. Directed by Dr. Elizabeth Berry-Kravis, it serves as the global headquarters for training physicians in neurogenetic clinical trials and delivering innovative interventional therapies. Human clinical trials for STEP will run at Rush.
• Ultragenyx PharmaceuticalflagshipStrategic or Co-development Partner · 18 July 2022Ultragenyx acquired GeneTx Biotherapeutics LLC (FAST's for-profit subsidiary) for the GTX-102 antisense oligonucleotide program. Ultragenyx invested $25 million in GeneTx and assumed complete control of the GTX-102 clinical development program, including ongoing Phase 1/2 and Phase 3 trials. FAST's Chief Science Officer Dr. Allyson Berent serves as a consultant to Ultragenyx.
• BIOM TherapeuticsminorStrategic or Co-development Partner · 1 March 2021BIOM Therapeutics received FDA Orphan Drug Status in March 2021 for BIO017, an investigational Cannabidiol (CBD) approach to treating Angelman syndrome. Orphan Drug Designation supports development for rare disorders.
• University of PennsylvaniacoreStrategic or Co-development Partner · 1 January 2021FAST entered into an exclusive global collaborative research and development agreement with UPenn to develop an investigational AAV gene therapy for Angelman syndrome. FAST has sponsored preclinical research studies at UPenn's Gene Therapy Program since 2017. FAST committed approximately $14 million for IND-enabling studies. Dr. Jim Wilson's team leads the research program.
• University of North CarolinacoreStrategic or Co-development Partner · 1 September 2019UNC received a $6.1 million NIH award to develop CRISPR/Cas9 gene therapy for Angelman syndrome in collaboration with the Philpot Lab. UNC is also exploring AAV gene therapy with Taysha Gene Therapies, small molecule paternal activation approaches, and CRISPR/base-editing strategies.
• Global Affiliates (Australia, Canada, Italy, France, LatAm, Poland, Spain, UK, Germany)coreStrategic or Co-development PartnerFAST has established a network of country and region-based affiliates all run by AS parents. Affiliates focus on helping bring effective therapeutics to those affected, representing the global footprint of AS, and supporting clinical trial readiness in their regions through education, research grant funding, translation, and natural history studies.
• Ionis PharmaceuticalscoreStrategic or Co-development PartnerIonis Pharmaceuticals is developing ION582 (obudanersen), an ASO for Angelman syndrome. ION582 received FDA Breakthrough Therapy designation in September 2025. The Phase 3 REVEAL study dosed first patient in June 2025. FAST supports the program through patient community engagement and clinical trial awareness.
• Angelman Syndrome Biomarker and Outcome Measure Consortium (A-BOM)coreStrategic or Co-development PartnerA-BOM brings together hundreds of individuals invested in Angelman syndrome including pharmaceutical companies, clinicians, translational research teams, regulatory experts, and patient advocacy groups to achieve advanced endpoints and biomarkers required to accurately measure drug effects in clinical trials.
• UC DaviscoreStrategic or Co-development PartnerUC Davis researchers, led by Dr. David Segal with collaborators Dr. Jill Silverman and Dr. Kyle Fink, are developing artificial transcription factors/zinc fingers (ATF-ZF) to unsilence the paternal UBE3A gene. FAST-funded research reported behavioral rescue in mouse models following ATF administration.
• Taysha Gene TherapiesminorStrategic or Co-development PartnerTaysha Gene Therapies collaborated with UNC to explore an investigational AAV gene therapy dual isoform approach for Angelman syndrome. Taysha published positive preclinical data in JCI Insight demonstrating therapeutic potential.
• Transformatx Biotherapeutics LLCcoreStrategic or Co-development PartnerTransformatx Biotherapeutics LLC is developing investigational hematopoietic stem cell gene therapy (HSC-GT) for Angelman syndrome. The company was founded by FAST and received funding support for the UCLA program that advanced with a $5.8M CIRM award.
• Encoded TherapeuticsminorStrategic or Co-development PartnerEncoded Therapeutics is exploring ETX201, an investigational microRNA knockdown approach to treating Angelman syndrome using AAV virus delivery to activate the silent paternal UBE3A gene.
• Arbor BiotechnologiesminorStrategic or Co-development PartnerArbor Biotechnologies is exploring a CRISPR approach to treating Angelman syndrome designed to cut the UBE3A-ATS and potentially allow the paternal UBE3A gene to be turned on. They presented data at ASGCT 28th Annual Meeting.
• Children's Hospital of Philadelphia (CHOP)minorStrategic or Co-development PartnerCHOP is exploring an investigational base editing strategy using mRNA-LNP (messenger RNA loaded lipid nanoparticles) to activate the paternal UBE3A gene as a potential treatment for Angelman syndrome.
• Keck Graduate InstituteminorStrategic or Co-development PartnerKGI is exploring Cell Penetrating Peptide (CPP) CRISPR approaches to treating Angelman syndrome. Dr. Barbara Bailus, FAST Scientific Advisory Board Chair, leads the KGI research team. KGI is also developing CPP-UBE3A enzyme replacement therapy.
• Mass General Hospital (MGH)minorStrategic or Co-development PartnerMGH is exploring an investigational di-siRNA (divalent small interfering RNA) approach to paternal gene activation as a potential treatment for Angelman syndrome. Dr. Mark Deehan leads the research team.
• Neuren PharmaceuticalsminorStrategic or Co-development PartnerNeuren Pharmaceuticals completed a Phase 2 clinical trial of NNZ-2591, a synthetic analog related to IGF-1, in children with Angelman syndrome. Results showed statistically significant improvements in CGI-I and CIC measures. The trial showed NNZ-2591 was safe and well tolerated.
• HealxminorStrategic or Co-development PartnerHealx is exploring HLX-0553, an investigational small molecule approach to treating Angelman syndrome by targeting downstream molecular pathways affected by missing UBE3A protein.

Scale indicators9 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• CureSMA: CureSMA is a disease-specific patient advocacy foundation that funded and drove development of multiple SMA therapeutics including Spinraza/Zolgensma, using a venture-philanthropy model with biotech partnerships and equity-like upside — directly analogous to FAST's Angelman syndrome strategy.
• Parent Project Muscular Dystrophy: PPMD is a Duchenne muscular dystrophy patient advocacy foundation that funds gene therapy, exon-skipping, and other translational research, operates a patient registry, and has driven multiple programs into clinical trials — highly comparable structure and modality focus.
• Rett Syndrome Research Trust: RSRT is a rare neurodevelopmental disorder research foundation focused on reversing Rett syndrome through gene therapy, gene editing, and RNA approaches — directly comparable patient advocacy, single-gene CNS focus, and venture-philanthropy biotech incubation model.
• Friedreich's Ataxia Research Alliance: FARA is a rare-disease patient-driven research foundation that funds translational research, manages a patient registry, and partners with biotech/pharma on therapeutics development for Friedreich's ataxia — directly comparable to FAST's operational model.
• National Fragile X Foundation: The National Fragile X Foundation is a rare neurodevelopmental disorder advocacy organization that funds research, supports families, and engages in clinical trial readiness — comparable patient population (developmental disability) and operational approach to FAST.
• Charcot-Marie-Tooth Association: CMTA is a disease-specific patient advocacy foundation that funds translational research and partners with biotech on gene therapy approaches for Charcot-Marie-Tooth disease — comparable single-disease nonprofit with active therapeutic development pipeline.
• Spinal Muscular Atrophy Foundation: The SMA Foundation funds translational research and partners with pharma on SMA therapeutics development — directly analogous disease-specific, rare-genetic, CNS-focused advocacy and research funding model to FAST.

Broad incumbents

• Cystic Fibrosis Foundation: CFF pioneered the venture-philanthropy model for rare disease therapeutics (funding Kalydeco/Trikafta development and earning billions in royalties) — a much larger and more established organization in the same broad category, with a directly analogous funding-plus-equity strategy.
• Muscular Dystrophy Association: MDA is a larger, multi-disease neuromuscular disorder advocacy foundation that funds research across multiple disease programs, operates care centers, and supports clinical trials — broader than FAST but operating with similar funding and registry infrastructure.

Others

• Epilepsy Foundation: The Epilepsy Foundation is a large patient advocacy organization that funds research and supports clinical trials for seizure-related disorders — Angelman syndrome involves seizures and overlaps with epilepsy research/patient communities, making it a thematically related peer.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xx.com/CureAngelman
• YouTubeyoutube.com/@CureangelmanOrg
• Facebookfacebook.com/cureangelman.org
• LinkedInlinkedin.com/company/foundation-for-angelman-syndrome-therapeutics
• Instagraminstagram.com/cureangelman
• TikToktiktok.com/@cureangelman

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Subsidiaries5 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about Foundation for Angelman Syndrome Therapeutics