What FRAXA Research Foundation does

FRAXA Research Foundation is a 501(c)(3) nonprofit organization founded in 1994 and headquartered in Newburyport, Massachusetts, dedicated to finding treatments and a cure for Fragile X syndrome (FXS), the most common inherited cause of autism and intellectual disability. The foundation directly funds research grants and fellowships at universities worldwide, operating a portfolio of 700 grants to date representing $38.8 million in direct research investment, and supports an active network of approximately 41 research teams and 34 pharmaceutical and biomedical partners including Spinogenix, Mirum Pharmaceuticals, Shionogi, Servier, QurAlis, and CONNECTA Therapeutics. Core programmatic offerings include the FRAXA Research Grants Program, the FRAXA Drug Validation Initiative (FRAXA-DVI) — a standardized preclinical testing platform using validated Fragile X mouse models — and Clinical Trials Support covering site principal investigator grants and partner program coordination across Phase 2 and Phase 3 assets.

The organization's go-to-market is community-led rather than commercial: it raises capital through individual donations, monthly giving, corporate contributions, personal fundraising campaigns, and a portfolio of community events including World Fragile X Day (July 22), the Fragile X Poker Run, Patrick's PALS basketball tournament ($150K+/yr), Callum Cup soccer match, and the FRAXAMON online Pokemon tournament. As a nonprofit, FRAXA does not sell products or services in the traditional sense; its "revenue" is charitable contributions deployed as research grants, preclinical validation, and clinical trial infrastructure. Every staff member and Board director is a parent of one or more children with Fragile X, producing unusually high mission alignment. Recognition includes Candid Platinum Seal, Charity Navigator 4-Star rating, GreatNonprofits Top-Rated 2025, and the 2026 Kavli Prize in Neuroscience awarded to FRAXA-funded investigator Oswald Steward.

Name

FRAXA Research Foundation

Legal name

FRAXA Research Foundation

Website

https://fraxa.org

Company type

Private

Founded year

1994

Operating status

Operating

Headcount range

1–10 employees

Short description

FRAXA Research Foundation is a 501(c)(3) nonprofit founded in 1994 that funds Fragile X syndrome research through 700+ grants, a standardized preclinical testing platform (FRAXA-DVI), and partnerships with 34 pharma and biomedical companies, serving researchers, families, and drug developers.

Ownership category

akta.pro rank

Product category

Biomedical Research Grants

NAICS

Scientific Research and Development Services (5417), Voluntary Health Organizations (813212)

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

Health & Medical Research Grantmaking Foundations (BPAGAKAL), Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• Fragile x research
• Biomedical research grants
• Rare disease therapeutics
• Clinical trial support
• Nonprofit health research

Headquarters

HQ city

Newburyport

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

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

Revenue model

1. Donations and Charitable Contributions: FRAXA raises funds through individual donations, monthly giving programs, personal fundraising campaigns, and corporate giving programs. As a 501(c)(3) organization (Tax ID: 04-3222167), all contributions are tax-deductible.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels7 records

Core offering

FRAXA Research Foundation is a 501(c)(3) nonprofit organization that funds and conducts research aimed at finding effective treatments and a cure for Fragile X syndrome, the leading known genetic cause of autism. Founded in 1994 and headquartered in Newburyport, Massachusetts, the foundation awards biomedical research grants and supports preclinical and clinical drug development in partnership with pharmaceutical companies and academic researchers.

Product overview

FRAXA Research Foundation is a nonprofit organization (501(c)(3)) that operates as a research funding and advocacy organization rather than a traditional software product company. The foundation's primary offerings include: a research grants program funding investigations at universities worldwide, the FRAXA Drug Validation Initiative (FRAXA-DVI) providing preclinical compound testing services, clinical trial support and partnership programs, and community engagement initiatives including World Fragile X Day, the Annual Poker Run, Patrick's PALS basketball tournament, Callum Cup soccer match, and FRAXAMON Pokemon tournament. The foundation also produces educational resources including webinars on the Fragile X treatment pipeline. These programs work together to advance Fragile X research from discovery through clinical trials toward FDA-approved treatments.

Differentiator

Problem solved

Functional benefit

Brands

• FRAXA-DVI (FRAXA Drug Validation Initiative): FRAXA's preclinical drug testing platform that provides streamlined, cost-effective testing to evaluate investigational compounds for Fragile X syndrome.
• FRAXAMON

Products and services

• FRAXA Research Grant Program
• FRAXA-DVI Preclinical Testing Service
• Clinical Trial Support Program

Quantifiable outcome

• 700 Fragile X research grants awarded over 30+ years
• +3 more outcomes

Segments3 records

Ideal customer profiles3 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Partnerships

15 partnerships are on record, tiered core and minor.

• SpinogenixcoreStrategic or Co-development Partner · 2 March 2026FRAXA Research Foundation provided grant support to site principal investigators for Spinogenix's Phase 2b trial of SPG601, a first-in-class BK channel modulator for Fragile X syndrome. SPG601 has received FDA Orphan Drug and Fast Track designations. The therapy showed compelling Phase 2a results including improvements in gamma band activity and measures of attention and inhibitory control.
• NospharmaminorStrategic or Co-development Partner · 4 February 2026FRAXA partnered with Nospharma to conduct pre-clinical testing of NOS-01 in Fragile X mouse models. The partnership utilizes FRAXA's standardized preclinical testing platform (FRAXA-DVI) to validate NOS-01's efficacy and accelerate development.
• Quiver BiosciencescoreStrategic or Co-development PartnerFRAXA funds Quiver Biosciences to validate a novel antisense oligonucleotide (ASO) therapy targeting the root cause of Fragile X syndrome.
• Mirum PharmaceuticalscoreStrategic or Co-development PartnerFRAXA-funded PDE4D research led to Mirum's MRM-3379 Phase 2 trial for Fragile X syndrome, now enrolling males ages 13-45 with FXS.
• ShionogicoreStrategic or Co-development PartnerShionogi's EXPERIENCE Phase 3 clinical trial of zatolmilast in Fragile X syndrome is supported by FRAXA-funded research on PDE4D inhibition.
• ServiercoreStrategic or Co-development PartnerServier's BK channel program (KER-0193) for Fragile X syndrome is being advanced with FRAXA support; Phase 2 study expected to begin in adults in Europe and US.
• QurAlis CorporationcoreStrategic or Co-development PartnerQurAlis is developing ASO/splicing strategy for Fragile X focused on restoring FMRP production in individuals with Fragile X who produce specific FMR1-related RNA. UMass Chan Medical School licensed RNA-based therapy to QurAlis.
• CONNECTA TherapeuticsminorStrategic or Co-development PartnerCONNECTA's Phase 2 trial of CTH120, a first-in-class neuroplasticity modulator, has begun in Spain with trial sites in Barcelona area.
• King Abdullah University of Science and Technology (KAUST)coreStrategic or Co-development PartnerFRAXA funds research at KAUST, including Leena Ibrahim and Deyl S. Djama's project on sensory processing difficulties in Fragile X syndrome ($100,000 grant for 2026-2027).
• University of GothenburgminorStrategic or Co-development PartnerFRAXA-funded researchers at Gothenburg University study whether activating TFEB can restore dopamine function and improve flexibility in Fragile X syndrome.
• Cincinnati Children's HospitalcoreStrategic or Co-development PartnerFRAXA grant of $100,000 to Cincinnati Children's Hospital supports mobile EEG research to simplify and test EEG technology for home use, improving clinical trial accessibility.
• Italian Institute of TechnologyminorStrategic or Co-development PartnerFRAXA funds Laura Cancedda's research on advanced preclinical testing of NKCC1 inhibitors supporting Phase 2 trials in Fragile X.
• UMass Chan Medical SchoolcoreStrategic or Co-development PartnerFRAXA funds Joel Richter and Sneha Shah's ASO rescue of FMR1 mis-splicing research. The school licensed RNA-based therapy to QurAlis for Fragile X treatment development.
• Harvard Medical SchoolcoreStrategic or Co-development PartnerFRAXA funds Emily Osterweil's research identifying new therapeutic approaches using human Fragile X models. Dr. Jeannie Lee received $1M Blavatnik Award for gene reactivation therapy research supported by FRAXA.
• University of PaduaminorStrategic or Co-development PartnerNicola Elvassore's team at University of Padua conducts FRAXA-funded research on cell type-specific protein dysregulation in Fragile X brain organoids.

Scale indicators5 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• National Fragile X Foundation: The other major U.S. nonprofit focused on Fragile X syndrome, providing community support, advocacy, and clinical resources. Operates in the same disease space with overlapping donor and family constituencies, making it the most directly comparable peer to FRAXA.
• Tuberous Sclerosis Alliance: Disease-specific nonprofit funding research, providing family support, and advocating for treatments for tuberous sclerosis complex — a similar single-gene rare neurological disorder with comparable grantmaking, clinical trial support, and community-advocacy model.
• CureSMA: Spinal muscular atrophy-focused nonprofit that funds research and supports clinical trials; structurally analogous to FRAXA as a parent-founded, single-disease research foundation that has helped bring disease-modifying therapies (Spinraza, Zolgensma, Evrysdi) to market.
• Parent Project Muscular Dystrophy: Parent-led nonprofit driving Duchenne muscular dystrophy research funding and clinical trial support — a near-identical operating model to FRAXA, with deep pharma partnerships and a track record of moving multiple therapies through FDA review.
• COMBINEDBrain: Nonprofit consortium for neurodevelopmental disorders that FRAXA explicitly collaborates with on Fragile X biomarker research; operates a comparable translational research and clinical-trial-enabling model across related rare genetic conditions.

Broad incumbents

• ALS Association: Disease-specific nonprofit funding ALS research, advocacy, and clinical care. Comparable scale and structure to FRAXA, with a similarly broad pharma and academic partner network and reliance on grassroots fundraising events.
• National Organization for Rare Disorders (NORD): Umbrella advocacy and research-funding organization for the rare-disease community. FRAXA engages with NORD-style coalitions for policy and orphan-drug advocacy, making it a broader-incumbent peer in the same ecosystem.
• Cystic Fibrosis Foundation: The archetypal disease-specific research foundation that helped fund development of Trikafta — a gold-standard model FRAXA aspires to replicate. Highly comparable operating model and revenue scale (multi-hundred-million-dollar fundraising), but with a far larger employee base and broader clinical-care footprint.
• EveryLife Foundation for Rare Diseases: Rare-disease policy and advocacy nonprofit that amplifies the work of disease-specific foundations like FRAXA. Operates in the same funding and advocacy ecosystem and provides policy infrastructure FRAXA benefits from but does not directly replicate.

Emerging players

• Autism Science Foundation: Nonprofit funding autism-related research, including work that intersects with Fragile X (the most common monogenic cause of autism). Comparable scientific scope, though smaller scale and broader indication focus than FRAXA.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat6 records

Key risks5 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/fraxa-research-foundation
• Xx.com/fraxaresearch
• Facebookfacebook.com/fraxaresearch

Revenue estimate

Valuation estimate

Number of profiles

Profiles2 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about FRAXA Research Foundation