The Ryan Foundation

Name

The Ryan Foundation

Legal name

The Ryan Foundation

Website

https://ryanfoundation.org

Company type

Private

Founded year

1991

Operating status

Operating

Headcount range

1–10 employees

Ownership category

akta.pro rank

Headquarters

HQ city

Carrollton

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2C

Offering type

Services

Cost components

Technology or R&D, Marketing or Sales, Operations

Revenue model

1. Individual Donations: The foundation relies primarily on personal gifts from individual donors. They do not receive donations from large companies due to the rarity of diseases they support. Notable donation received: $200,000 from a single individual donor.
2. Fundraising Events: Revenue generated from community fundraising events including golf tournaments, fishing tournaments, raffles, bake sales, and other charitable events. First bake sale netted $342.

Go-to-market motion2 records

Distribution channels2 records

Marketing channels5 records

Core offering

The Ryan Foundation is a 501(c)(3) nonprofit organization that funds scientific research aimed at developing treatments and cures for rare genetic diseases, with specific focus on Mucopolysaccharidosis I (MPS I) and other lysosomal storage diseases (LSD). The foundation provides research grants to universities and medical centers, supports clinical trials, advocates for rare disease legislation, and connects affected families with research opportunities. Its funded research was decisive in the development and 2003 FDA approval of Aldurazyme (laronidase), the first enzyme replacement therapy for MPS I, now available in 75 countries.

Product overview

The Ryan Foundation is a 501(c)(3) nonprofit organization focused on funding scientific research to find treatments and cures for rare genetic diseases, with a specific emphasis on Mucopolysaccharidosis (MPS) and Lysosomal Storage Diseases (LSD). The foundation does not offer commercial products but instead operates as a research funding organization that supports clinical studies, gene therapy research, and enzyme replacement therapy development through partnerships with research institutions. Key activities include grant funding to universities and research facilities, publishing research findings, and raising awareness about rare diseases.

Differentiator

Problem solved

Functional benefit

Quantifiable outcome

• Aldurazyme (laronidase) approved by FDA in 2003 - first enzyme replacement therapy for MPS I
• +3 more outcomes

Named customers5 records

Segments2 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Ten partnerships are on record, tiered minor and core.

• Icahn School of Medicine at Mount SinaiminorStrategic or Co-development Partner · 1 January 2013Collaborating on clinical studies for mucopolysaccharidoses. Dr. Calogera Simonaro and Dr. Edward Schuchman are conducting research on pentosan polysulfate (PPS) treatment for MPS.
• Dr. Emil Kakkis (UCLA/LA BioMed)coreStrategic or Co-development Partner · 1 January 1992Dr. Emil Kakkis was conducting promising MPS I treatment research at UCLA when the Ryan Foundation found him. The foundation provided critical early funding that enabled Dr. Kakkis and his team to continue their research, eventually leading to the development of Aldurazyme, the first FDA-approved enzyme replacement therapy for MPS I. This partnership is considered the foundation's most significant achievement.
• Los Angeles Biomedical Research Institute at Harbor-UCLA Medical CentercoreStrategic or Co-development PartnerLA BioMed has been a critical research partner, conducting clinical trials for intrathecal enzyme replacement therapy for MPS I. The foundation has funded transportation for MPS I families to participate in clinical trials at this facility. Research at LA BioMed demonstrated that enzyme therapy can cross the blood-brain barrier.
• University of MinnesotacoreStrategic or Co-development PartnerThe foundation supports research at University of Minnesota exploring brain structure and function abnormalities in MPS disorders. Dr. Elsa Shapiro's research on neuroimaging techniques has been funded by the foundation and has led to government funding.
• Iowa State UniversitycoreStrategic or Co-development PartnerCollaborated on gene therapy research for lysosomal storage diseases in large animal models. Dr. N. Matthew Ellinwood has conducted MPS research funded by the foundation.
• Washington University School of MedicineminorStrategic or Co-development PartnerFoundation has funded research at Washington University focused on challenges in treating musculoskeletal disease in MPS.
• University of PennsylvaniaminorStrategic or Co-development PartnerResearch funding recipient for gene therapy for lysosomal storage diseases. Dr. Mark Haskins conducted research on canine models.
• University of UtahminorStrategic or Co-development PartnerOne of the research institutions receiving funding from the Ryan Foundation for rare disease research.
• National MPS SocietycoreStrategic or Co-development PartnerThe foundation works closely with the National MPS Society. Steve Holland, an MPS parent whose children benefited from foundation-funded research, serves as President of the National MPS Society Board of Directors. The foundation is recognized as the premier MPS 1 research foundation by the society.
• Bene Pharmachem GmbHminorStrategic or Co-development PartnerPartnering with Mount Sinai on clinical studies for mucopolysaccharidoses. Bene Pharmachem is involved in developing treatments for rare diseases.

Scale indicators6 records

Recent moves5 records

Expansion highlights4 records

Broad incumbents

• Cystic Fibrosis Foundation: The Cystic Fibrosis Foundation is the leading model for disease-specific medical research foundations, having funded development of therapies that transformed CF from terminal to manageable. It is comparable as the gold standard of patient-founded, single-disease research philanthropy.
• National Organization for Rare Disorders (NORD): NORD is the largest US umbrella organization for rare diseases, advocating for the 7,000+ identified conditions and providing research grants and patient support. It is comparable as the broad incumbent in rare disease philanthropy that disease-specific foundations like Ryan Foundation operate within.

Direct peers

• National Tay-Sachs & Allied Diseases Association (NTSAD): NTSAD is a patient advocacy and research foundation for Tay-Sachs, Sandhoff, GM1, and Canavan diseases - several of which are lysosomal storage disorders. It is comparable as a small family-founded rare disease foundation funding translational research for inherited metabolic diseases.
• Cure SMA: Cure SMA funds spinal muscular atrophy research, supports patients, and advocated for newborn screening. Its trajectory from family-founded research funder to enabler of the first FDA-approved SMA gene therapy mirrors the Ryan Foundation's Aldurazyme story.
• Parent Project Muscular Dystrophy (PPMD): PPMD is a parent-founded nonprofit focused exclusively on Duchenne muscular dystrophy research, advocacy, and patient support. It is directly comparable as a family-driven rare disease research foundation with a similar operating model of funding academic labs and clinical translation.
• National MPS Society: The National MPS Society is a patient advocacy and support organization specifically for MPS and related diseases. It is the closest peer to the Ryan Foundation, working in tandem on family services, clinical trial connections, and MPS research, and has recognized Ryan Foundation as the premier MPS I research foundation.
• Kakkis EveryLife Foundation: Founded by Dr. Emil Kakkis, the researcher whose MPS I enzyme replacement therapy was funded by the Ryan Foundation. EveryLife advocates for rare disease drug development policy and is directly comparable as a researcher-led foundation operating in the same MPS/rare disease space.
• Acid Maltase Deficiency Association (AMDA / Pompe disease): AMDA is a patient-led foundation for Pompe disease, another lysosomal storage disorder. It is directly comparable as a small disease-specific nonprofit supporting research and patients for an LSD, sharing the same patient advocacy and research funding model.

Emerging players

• Global Genes: Global Genes is a rare disease patient advocacy organization that builds awareness, supports patient communities, and convenes the RARE Foundation Alliance. It overlaps with the Ryan Foundation on rare disease awareness and community-building missions.

Regional players

• Children's Hospital Foundation (Fort Worth / Cook Children's): Cook Children's Health Foundation is a regional health-focused philanthropy in the same North Texas geography as the Ryan Foundation. It is comparable as a regional nonprofit raising funds to support pediatric specialty care and research at a local children's hospital.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key highlights6 records

Customer concentration

• Facebookfacebook.com/The-Ryan-Foundation-100066501524665

Revenue estimate

Valuation estimate

Number of profiles

Profiles18 records

Funding overview

Funding rounds

Investors

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M&A

Investments

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Frequently asked questions about The Ryan Foundation