Cure MITO Foundation
- Company typePrivate
- Founded2018
- HeadquartersMckinney, United States
- Headcount1–10
- GTM typeB2C
- OfferingServices
Cure MITO Foundation firmographics
Firmographics- Name
- Cure MITO Foundation
- Legal name
- Cure Mito Foundation
- Website
- https://curemito.org
- Company type
- Private
- Founded year
- 2018
- Operating status
- Operating
- Headcount range
- 1–10 employees
- Ownership category
- akta.pro rank
Cure MITO Foundation industry classification
Industry- Product category
- Rare Disease Patient Advocacy
- NAICS
- Voluntary Health Organizations (813212), Grantmaking Foundations (813211)
- SIC
- Services-Social Services (8300), Services-Health Services (8000)
- akta.pro primary industry
- Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS) (BPAGACAA)
- akta.pro secondary industries
- Health & Medical Research Grantmaking Foundations (BPAGAKAL), Global Health Research, Evidence & Technical Assistance Organizations (HLAJAKAN)
Keywords
Where Cure MITO Foundation is headquartered
LocationHeadquarters
- HQ city
- Mckinney
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Cure MITO Foundation business model
Business model- GTM type
- B2C
- Offering type
- Services
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales
Revenue model
- Donations and Fundraising: The foundation operates as a 501(c)(3) nonprofit organization (EIN: 82-4665767) primarily funded through individual donations, grassroots fundraising campaigns such as the 'One Shot to Live' SURF1 gene therapy campaign targeting $2.5 million, merchandise sales through Amazon storefront and Bonfire store, and direct charitable contributions.
- Grants and Foundation Funding: Cure Mito receives grants from institutional funders including the Chan Zuckerberg Initiative (CZI) as a member of the Rare As One Network, RTW Foundation, Rare Village Foundation, and other research foundations supporting rare disease research.
Go-to-market motion1 record
Distribution channels4 records
Marketing channels6 records
Cure MITO Foundation product offering
Product offeringCore offering
Cure Mito Foundation is a patient-led 501(c)(3) nonprofit advocacy organization that accelerates research and treatments for Leigh syndrome and mitochondrial diseases. The foundation funds and supports research projects including gene therapy, drug repurposing, mitochondria transfer, and microbiome studies; operates the world's largest Leigh syndrome patient registry with 400+ participants from 40+ countries; and provides educational resources, family support, and community programs for affected families worldwide.
Product overview
Cure Mito Foundation operates as a patient-led advocacy organization focused on Leigh syndrome and mitochondrial diseases research. The organization offers a portfolio of interconnected programs including the Leigh Syndrome Global Patient Registry (the world's largest with 400+ participants from 40+ countries), the AboutLeighSyndrome.com educational resource, drug repurposing research initiatives in partnership with Perlara, Prigione Lab, Unravel Biosciences, and Transcripta Bio, and the SURF1 gene therapy development program. Additional offerings include an annual symposium, newsletter, patient support resources including free genetic testing, and merchandise through an Amazon storefront. The organization was founded in 2018 as Cure SURF1 Foundation and rebranded as Cure Mito Foundation in 2021 to expand efforts beyond SURF1 to other causes of Leigh syndrome.
Differentiator
Problem solved
Functional benefit
Products and services
- Leigh Syndrome Global Patient Registry The world's largest global patient registry for Leigh syndrome with 400+ participants from 40+ countries. Collects patient data to advance disease understanding, raise awareness for treatments, and connect participants with research and clinical trial opportunities. Registry data is aligned with CDISC regulatory submission standards and integrated into C-Path's RDCA-DAP platform. An AI-powered Registry Assistant was added in 2026.
- SURF1 Gene Therapy Development Program Gene replacement therapy development program for SURF1 Leigh syndrome using AAV delivery system. Since 2018, over $1.5 million has been funded toward this program. The therapy aims to deliver a healthy SURF1 gene to cells via intrathecal injection to restore energy production. Partnered with Dr. Steven Gray's lab at UT Southwestern Medical Center.
- Drug Repurposing Research Program Multi-pronged drug repurposing initiative spanning yeast-based screening with Perlara PBC, organoid and AI-enabled drug discovery with Prigione Lab, patient RNA-guided drug discovery with Unravel Biosciences (using nasal swabs and AI to create Living Molecular Twins simulations), and patient-derived brain cell drug screening with Transcripta Bio. Cure Mito has invested $225,000 in these projects. Identified losmapimod (FDA Rare Pediatric Disease Designation) and sildenafil (EMA Orphan Drug Designation) as promising candidates.
- Annual Leigh Syndrome Symposium Annual virtual conference branded 'Empower and Inspire' that unites families, clinicians, researchers, and industry to drive progress in Leigh syndrome research and community connection. The 2022 conference had 228 participants from 34 countries and it has continued as an annual event through 2025 and beyond.
- Patient Support Resources Comprehensive support services for Leigh syndrome families including a medical provider directory, free genetic testing partnership with Probably Genetic, family planning guidance, newly diagnosed resources, and a glossary of terms. Targeted at newly diagnosed families and families navigating the disease.
- AboutLeighSyndrome.com First-of-its-kind comprehensive online educational resource dedicated to Leigh syndrome for both patients and physicians, covering education, research, support, and clinical information. Sponsored by PTC Therapeutics.
- Patient Fibroblast Collection Biospecimen resource created through partnership with Coriell Institute for Medical Research. Skin samples collected from Leigh syndrome patients to create fibroblast cell lines stored at Coriell, enabling researchers worldwide to study the disease through patient-derived cell models.
- Amazon Merchandise Storefront Online storefront selling branded merchandise including shirts, hoodies, and awareness items at amazon.com/shop/curemitofoundation, with proceeds supporting Leigh syndrome research and the foundation's mission.
Quantifiable outcome
- 400+ patients enrolled in global registry from 40+ countries
- +4 more outcomes
Companies that use Cure MITO Foundation
Customer profileNamed customers6 records
Segments3 records
Ideal customer profiles3 records
Cure MITO Foundation technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
AI capability5 records
Cure MITO Foundation partnerships and signals
Strategic signalPartnerships
18 partnerships are on record, tiered minor, major and core.
- Hope for PDCD FoundationminorPatient registry collaboration for the 25-35% of patients who have both Leigh syndrome and PDCD, aligning registries on Sanford Health CoRDS platform.
- PTC Therapeutics, Astellas Pharma US, Saol Therapeutics, Standigm, Abliva ABmajorCorporate Advisory Council (CAC) formed with five prominent rare disease companies to provide strategic guidance and industry expertise to Cure Mito Foundation.
- Critical Path Institute (C-Path)coreData sharing agreement to incorporate Leigh syndrome patient registry data into C-Path's Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) to accelerate treatment development.
- AllStripesmajorPartnership with MitoAction and AllStripes for Leigh syndrome research. AllStripes provides platform to accelerate research for rare disease patients and inform natural history understanding.
- Chan Zuckerberg Initiative (CZI)coreCure Mito is a member of the Rare As One Network, supported by a grant from CZI. This partnership provides funding and network support for patient-led rare disease research initiatives.
- UT Southwestern Medical CentercorePrimary research partner for SURF1 gene therapy development. Dr. Steven Gray's lab at UT Southwestern has received over $1 million in funding from Cure Mito since 2018 to develop gene replacement therapy for SURF1 Leigh syndrome.
- Perlara PBCcoreBiotechnology partnership for yeast-based drug repurposing screens. Testing thousands of existing drugs in yeast models of Leigh syndrome to identify candidates for clinical studies. Identified losmapimod with FDA Rare Pediatric Disease Designation.
- Prigione Lab (Heinrich Heine University)coreResearch collaboration on organoids and AI-enabled drug repurposing. Identified azole compounds and sildenafil as promising candidates; sildenafil received EMA Orphan Drug Designation for MT-ATP6 Leigh syndrome.
- Unravel BiosciencescorePatient RNA-guided drug discovery partnership using nasal swab collection and AI-driven analysis to predict which drugs could help across Leigh syndrome population. Created 'Living Molecular Twins' digital simulations.
- Transcripta BiomajorDrug screening collaboration in patient-derived brain cells. Screened thousands of FDA-approved drugs in neurons derived from Leigh syndrome patients; identified omaveloxolone as promising candidate.
- Coriell Institute for Medical ResearchmajorPatient fibroblast cell lines created and stored at Coriell Institute. Cell models from Leigh syndrome patients stored and shared with researchers for disease study.
- Sanford Research / CoRDScoreLeigh Syndrome Global Patient Registry hosted on Sanford Health CoRDS platform, enabling global patient enrollment and data collection for research purposes.
- Taysha Gene TherapiesmajorBiotech partnership to advance SURF1 gene therapy toward clinical trial. SURF1-AAV therapy received FDA Orphan Drug Designation. Partnership included natural history collaboration with AllStripes.
- Sumptuous Data SciencesmajorPartnership on alignment and interoperability of Leigh Syndrome Registry Data with Regulatory Submission Standards (CDISC), enabling data use in regulatory submissions.
- Probably GeneticminorPartnership to increase access to genetic testing within Leigh syndrome community. Probably Genetic provides no-cost genetic testing program including sample collection kits and genetic counseling.
- Dr. Qinglan LingcoreScientific Advisor and former postdoc in Dr. Gray's lab, now Assistant Professor at UMass Chan Medical School. Received $1 million NIH award for MT-ATP6 research. Led animal experiments for SURF1 gene therapy.
- Brestoff Lab (Washington University)major$140,000 grant for study determining effects of in vitro mitochondria transplantation in patient-derived fibroblasts.
- Dr. Ibrahim Elsharkawi (Icahn School of Medicine at Mount Sinai)major$34,000 grant for study investigating the role of gut microbiome in primary mitochondrial disease.
Scale indicators5 records
Recent moves7 records
Expansion highlights4 records
Cure MITO Foundation competitors and assessment
Company assessmentDirect peers
- Foundation for Mitochondrial Disease (FAME): Patient-led foundation raising funds for mitochondrial disease research and family support, with a similar patient-advocacy-plus-grantmaking model to Cure Mito Foundation.
- United Mitochondrial Disease Foundation (UMDF): The largest U.S. nonprofit focused on mitochondrial disease, funding research, supporting patients, and convening the annual Mitochondrial Medicine Symposium. Directly comparable mission and constituency with Cure Mito Foundation.
- MitoAction: Patient advocacy nonprofit dedicated to mitochondrial disease, operating patient support programs, awareness campaigns, and community resources for patients and families affected by mitochondrial disorders including Leigh syndrome.
Regional players
- Mito Foundation (Australia): Australian patient advocacy and research funding organization for mitochondrial disease, comparable in mission and operating model but serving a separate geography.
- Metabolic Support UK: U.K. patient support charity for people affected by inherited metabolic diseases including mitochondrial disorders. Comparable support and advocacy mission in a complementary geography.
- The Lily Foundation: U.K.-based mitochondrial disease patient charity funding research, providing family support, and convening the mito community. Operates similar programs in a complementary geographic market.
Emerging players
- Parent Project Muscular Dystrophy (PPMD): Patient-led rare disease nonprofit that funds Duchenne muscular dystrophy research and operates a robust registry, advocacy, and drug-development accelerator model directly analogous to Cure Mito's Leigh syndrome strategy.
Broad incumbents
- EveryLife Foundation for Rare Diseases: Advocacy organization advancing policies supporting rare disease patients and therapies. Engages Cure Mito in legislative and regulatory advocacy shared across rare disease patient communities.
- National Organization for Rare Disorders (NORD): The largest U.S. umbrella advocacy organization for all rare diseases, providing policy advocacy, research grants, and patient programs. A broader incumbent that Cure Mito engages alongside via the rare-disease ecosystem.
Others
- Taysha Gene Therapies: Clinical-stage gene therapy company developing AAV-based therapies for CNS diseases, including the original SURF1-AAV (TSHA-104) program partnered with Cure Mito. An ecosystem participant and former co-developer rather than a direct advocacy peer.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat6 records
Key risks5 records
Key highlights6 records
Customer concentration
Cure MITO Foundation social profiles
Digital presenceCure MITO Foundation financial estimates
Financial estimateRevenue estimate
Valuation estimate
Cure MITO Foundation leadership team
Management profileNumber of profiles
Profiles3 records
Cure MITO Foundation funding detail
Funding detailFunding overview
Funding rounds
Investors
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Cure MITO Foundation M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Cure MITO Foundation
What does Cure MITO Foundation do?
Cure Mito Foundation is a patient-led 501(c)(3) nonprofit advocacy organization that accelerates research and treatments for Leigh syndrome and mitochondrial diseases. The foundation funds and supports research projects including gene therapy, drug repurposing, mitochondria transfer, and microbiome studies; operates the world's largest Leigh syndrome patient registry with 400+ participants from 40+ countries; and provides educational resources, family support, and community programs for affected families worldwide.
Is Cure MITO Foundation a public or private company?
Cure MITO Foundation is a private company. It is classified as nonprofit foundation owned and is currently operating.
When was Cure MITO Foundation founded?
Cure MITO Foundation was founded in 2018. It employs 1 to 10 people.
Where is Cure MITO Foundation based?
Cure MITO Foundation is headquartered in Mckinney, United States, in the North America region.
How does Cure MITO Foundation make money?
Two revenue lines are on record. Donations and Fundraising is the primary driver. The others are grants and Foundation Funding.
Who are Cure MITO Foundation's main competitors?
Direct peers on record are Foundation for Mitochondrial Disease (FAME), United Mitochondrial Disease Foundation (UMDF) and MitoAction. Regional players are Mito Foundation (Australia), Metabolic Support UK and The Lily Foundation. Parent Project Muscular Dystrophy (PPMD) is listed as an emerging player. Broad incumbents are EveryLife Foundation for Rare Diseases and National Organization for Rare Disorders (NORD). Taysha Gene Therapies is listed as an others.
Does Cure MITO Foundation have an API?
No public API is recorded for Cure MITO Foundation.
What industry is Cure MITO Foundation in?
Cure MITO Foundation's product category is Rare Disease Patient Advocacy. Its primary akta.pro industry code is BPAGACAA, Disease-Specific Research & Support (e.g., Cancer, Diabetes, ALS), with a secondary code of BPAGAKAL, Health & Medical Research Grantmaking Foundations. Its NAICS code is 813212 and its SIC code is 8300.