What Cure Sanfilippo Foundation does

Cure Sanfilippo Foundation is a 501(c)(3)-style nonprofit foundation founded in 2013 and headquartered in Columbia, South Carolina, with a stated mission to advocate for and fund research directed toward a cure for children with Sanfilippo Syndrome, a rare terminal neurodegenerative disease affecting an estimated 15,000 children globally. It operates with a small staff (1-10 employees) and serves four principal constituencies: families of affected children, academic and clinical researchers, physicians diagnosing and managing Sanfilippo patients, and biotechnology and pharmaceutical companies developing therapies. Its core service areas are accelerating research, uniting and supporting families, driving collaboration across stakeholders, and raising disease awareness.

Name

Cure Sanfilippo Foundation

Legal name

Cure Sanfilippo Foundation

Website

https://curesanfilippofoundation.org

Company type

Private

Founded year

2013

Operating status

Operating

Headcount range

1–10 employees

Short description

Cure Sanfilippo Foundation is a nonprofit foundation funding research toward a cure for Sanfilippo Syndrome, serving affected families, researchers, clinicians, and biotech companies through research grants, the global SanfilippoLINK registry, the ADVANCE conference, the Aurora grief support program, and FDA-facing caregiver preference research.

Ownership category

akta.pro rank

Product category

Nonprofit Rare Disease Patient Advocacy

NAICS

Voluntary Health Organizations (813212), Grantmaking and Giving Services (8132)

SIC

Services-Health Services (8000), Services-Social Services (8300)

akta.pro primary industry

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

akta.pro secondary industry

Grantmaking & Philanthropic Funds (Institutional Donors) (BPADAOAB)

Keywords

• Rare disease research funding
• Patient advocacy foundation
• Clinical research registry
• Pediatric neurodegenerative disease
• Family support services

Headquarters

HQ city

Scarborough

HQ country

Canada

HQ region

North America

Offices1 record

Markets served

GTM type

B2B and B2C

Offering type

Services

Cost components

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

Revenue model

1. Individual Donations: One-time and recurring donations from individuals supporting the mission to cure Sanfilippo Syndrome. Donors can give once or set up monthly recurring donations.
2. Corporate Giving & Matching: Corporate giving programs and employer donation matching programs for employees who donate.
3. Cryptocurrency Donations: Acceptance of cryptocurrency donations.
4. IRA, Stocks & Bequests: Planned giving options including IRA distributions, stock donations, and bequests.
5. Online Store / Merchandise: Sale of branded merchandise including t-shirts, hats, and family boutiques where supporters can purchase family-specific fundraising items.
6. Event Fundraising: Virtual and in-person events including 5K runs, streaming fundraisers through Tiltify, and hosted events.

Go-to-market motion3 records

Distribution channels4 records

Marketing channels8 records

Core offering

Cure Sanfilippo Foundation is a nonprofit patient advocacy organization that funds research toward treatments and a cure for Sanfilippo Syndrome, a rare terminal neurodegenerative childhood disease. It operates SanfilippoLINK, a global patient registry and clinical research platform, has funded 50+ research projects and multiple clinical trials, and provides counselor-led family support, educational webinars, clinical care guidelines, and the annual ADVANCE conference connecting families, researchers, clinicians, and biotechs.

Product overview

Cure Sanfilippo Foundation operates as a patient advocacy and research organization rather than a traditional product company. Its core offerings include SanfilippoLINK, a global patient registry platform for clinical research; ADVANCE, an annual virtual conference connecting families and researchers; the Aurora Program providing counselor-led grief support groups; Sanfilippo Speak webinar series for family education; Clinical Care Guidelines for disease management; and a Caregiver Preference Study. The organization also operates an online store for awareness merchandise and family boutiques. All offerings are oriented toward accelerating research, supporting affected families, and raising awareness for Sanfilippo Syndrome.

Differentiator

Problem solved

Functional benefit

Products and services

• SanfilippoLINK A global clinical research and patient registry platform dedicated to all forms of Sanfilippo syndrome, open to participants worldwide to advance understanding and treatments. It supports researchers and biotech companies with patient recruitment, natural history data, and clinical trial design insights.
• ADVANCE Conference An annual virtual Sanfilippo Community Conference bringing together families, caregivers, scientists, researchers, clinicians, therapists, advocates, biotechs, and supporters for collaboration, information sharing, and Sanfilippo-specific learning sessions.
• Aurora Program A counselor-led group support program providing grief processing and resilience building for families of children with Sanfilippo Syndrome. Includes Dads' Group, Daytime Open Group, Evening Open Group, and closed counseling groups of 8-10 parents meeting bi-weekly for six months; provided at no cost to partner families.
• Sanfilippo Speak A free family support webinar series providing insights and discussions around topics specific to navigating life with Sanfilippo Syndrome, available to families, caregivers, therapists, educators, and clinicians.
• Clinical Care Guidelines

Quantifiable outcome

• $20 million raised since 2013
• +4 more outcomes

Named customers3 records

Segments4 records

Ideal customer profiles4 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Twelve partnerships are on record, tiered core and minor.

• Sangrail BiologicscoreStrategic or Co-development Partner · 5 May 2026Clinical-stage gene therapy company that picked up Sanfilippo Type B AAV9 gene therapy program (SNG-101, formerly ABO-101 under Abeona Therapeutics). Launched company May 5, 2026.
• UT Southwestern Children's Medical CentercoreStrategic or Co-development PartnerConducts Natural History Study of Sanfilippo Syndrome Type IIIC (C-RARE), a remote prospective observational study of participants with MPS IIIC.
• Hospices Civils De LyoncoreStrategic or Co-development PartnerConducts natural history study of Sanfilippo Syndrome Type IIIC in a multi-center, 2-year observational study.
• Ultragenyx PharmaceuticalcoreStrategic or Co-development PartnerDeveloping UX111 AAV9 gene therapy for Sanfilippo Type A. Submitted to FDA for accelerated approval. Foundation has been actively advocating for FDA action on this therapy.
• Denali TherapeuticscoreStrategic or Co-development PartnerDNL126 (MPS IIIA) program selected for FDA's START pilot program, intended to improve efficiency of drug development.
• FDA (U.S. Food & Drug Administration)coreOthersFoundation presented Caregiver Preference Study to FDA in March 2020, advocating for non-cognitive based endpoints when evaluating therapies. FDA has engaged on surrogate endpoint agreement for UX111.
• NORD (National Organization for Rare Disorders)minorStrategic or Co-development PartnerNORD's Caregiver Respite Program provides financial assistance to enable caregivers a break from caregiving responsibilities.
• MPS SocietyminorStrategic or Co-development PartnerPartner organization providing family support programs including conference scholarships, bereavement expenses, equipment or medical aids, and travel assistance.
• The BLAIR ConnectionminorStrategic or Co-development PartnerSupport site for siblings of terminally-ill children, created by Grey Chapin whose sister Blair passed away from Sanfilippo in 2017.
• Courageous Parents NetworkminorStrategic or Co-development PartnerParent-created network providing wisdom from fellow parents and pediatric care providers to support families caring for seriously-ill children.
• Angel Flight NEminorStrategic or Co-development PartnerProvides free flights and ground transportation for patients to access life-saving medical care, including travel to Sanfilippo-related treatments.
• TSA CaresminorOthersTSA program assisting travelers with disabilities and medical conditions through Passenger Support Specialists who can provide assistance through security screening.

Scale indicators6 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Parent Project Muscular Dystrophy (PPMD): Parent-led nonprofit focused on Duchenne muscular dystrophy that funds research, advocates at FDA, and operates family support programs. Highly comparable operating model to Cure Sanfilippo's parent-driven, disease-specific structure.
• Cure SMA: Spinal muscular atrophy nonprofit that funded research leading to approved gene therapies (Spinraza, Zolgensma, Evrysdi). Closely comparable playbook: rare pediatric neuro disease, parent-founded, FDA advocacy, and direct biotech partnerships.
• Alex's Lemonade Stand Foundation: Childhood cancer research nonprofit combining research funding, family support, and broad donor engagement. Operates at a larger scale but follows a similar disease-specific research accelerator model Cure Sanfilippo employs for a rare pediatric disease.
• Cystic Fibrosis Foundation: Patient-founded, disease-specific nonprofit that funds research, runs a patient registry, and supports families for a single rare disease (CF). Cure Sanfilippo mirrors this model at a smaller scale, including direct FDA engagement, clinical care guidelines, and biotech partnerships.
• National MPS Society: Nonprofit supporting all mucopolysaccharidosis (MPS) and related lysosomal storage diseases, including Sanfilippo (MPS III). Provides family support, research funding, and conferences; directly relevant as an adjacent MPS umbrella organization.

Broad incumbents

• National Organization for Rare Disorders (NORD): Umbrella advocacy organization supporting all rare diseases, including operating the Caregiver Respite Program that partners with Cure Sanfilippo. Comparable as a federal-policy and cross-disease advocacy leader, but operates at a different scale and scope.
• EveryLife Foundation for Rare Diseases: Public policy advocacy nonprofit focused on accelerating biotech innovation for rare diseases. Comparable to Cure Sanfilippo in FDA/regulatory engagement mission but at an industry-wide rather than disease-specific level.
• ALS Association: Larger disease-specific nonprofit funding ALS research, supporting families, and advocating at FDA. Demonstrates a more mature version of the model Cure Sanfilippo is building, including clinical care guidelines and biotech partnerships.
• Global Genes: Rare disease patient advocacy organization focused on education, awareness, and community-building across many conditions. Overlaps with Cure Sanfilippo's awareness and family-support programming but serves a broader constituency.

Others

• Abeona Therapeutics: Clinical-stage gene therapy company that previously led ABO-101 (Sanfilippo Type B) before transferring the program to Sangrail. Relevant ecosystem participant as a former biotech partner rather than a comparable peer.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat5 records

Key risks5 records

Key highlights6 records

Customer concentration

• Xx.com/curesanfilippof
• Facebookfacebook.com/CureSanfilippoFoundation
• LinkedInlinkedin.com/company/33298970
• Instagraminstagram.com/curesanfilippofnd
• TikToktiktok.com/@curesanfilippo

Revenue estimate

Valuation estimate

Number of profiles

Profiles1 record

Funding overview

Funding rounds1 record

Investors1 record

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments1 record

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Cure Sanfilippo Foundation