CMT Research Foundation

What CMT Research Foundation does

CMT Research Foundation (CMTRF) is a patient-led 501(c)(3) non-profit foundation established in 2018 and headquartered in Atlanta, Georgia. Its singular mission is to accelerate the development of treatments and cures for Charcot-Marie-Tooth (CMT) disease, an inherited peripheral neuropathy affecting approximately 3 million people worldwide for which no approved treatments exist. The foundation operates as a financial intermediary rather than a product company, funding academic researchers, biotech startups, and translational research programs that span gene therapy, small molecule drugs, antisense oligonucleotides, and drug delivery technologies across all major CMT subtypes (CMT1, CMT2, CMT4, CMTX). It partners with institutions including Johns Hopkins, Nationwide Children's Hospital, The Jackson Laboratory, the Cyprus Institute of Neurology and Genetics, and the University of Miami.

The foundation's business model combines traditional non-profit revenue streams (donations, corporate sponsorships, fundraising events such as the Alan Jackson tour generating $2.25 million) with a distinctive revenue-share model that embeds return-on-investment clauses in industry partnership contracts, recycling proceeds back into new research. Grant sizes range from $25,000 to $200,000 per year, with milestone-gated release of funds. Operating geographies span 12+ countries across North America, EMEA, and Asia Pacific. The foundation operates with a lean team of 1-10 employees and relies on a Board of Directors, Scientific Advisory Board, and Research Advisory Council for governance.

CMTRF's distribution model centers on direct enterprise sales-style grant-making to researchers and biotech firms, supplemented by the annual Global CMT Research Convention (400+ attendees in 2025) and educational channels including the CMT 101 video series and "Gene Therapies Demystified" webinars. The foundation has funded 6 therapeutics now in preclinical or active clinical trials, and two CMTRF-funded partners have achieved material commercial validation: DTx Pharma was acquired by Novartis in 2023 for $1 billion, and Augustine Therapeutics raised $85 million in Series A financing in 2024 with first patient dosed in May 2025.

Name

CMT Research Foundation

Legal name

CMT Research Foundation, INC.

Website

https://cmtrf.org

Company type

Private

Founded year

2018

Operating status

Operating

Headcount range

1–10 employees

Short description

CMT Research Foundation is a patient-led 501(c)(3) non-profit that funds translational research across gene therapy, small molecules, and ASOs targeting Charcot-Marie-Tooth disease, serving academic researchers, biotech firms, and the CMT patient community globally.

Ownership category

akta.pro rank

Product category

Rare Disease Research Funding

NAICS

Voluntary Health Organizations (813212)

akta.pro primary industry

Health & Medical Research Grantmaking Foundations (BPAGAKAL)

akta.pro secondary industry

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

Keywords

• Rare disease research
• Charcot-Marie-Tooth research
• Gene therapy funding
• Patient advocacy nonprofit
• Translational medicine

Headquarters

HQ city

Atlanta

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

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

Revenue model

1. Donations and Charitable Contributions: The foundation raises funds through donations from individuals, corporate sponsors, and fundraising events (such as Alan Jackson's concert tour which raised $2.25 million). The foundation also receives grants from patient advocacy groups and co-funding partners.
2. Revenue Share Program: The foundation forges partnerships that include revenue share arrangements. Every contract includes a return on the Research Foundation's initial investment which is then reinvested in new research projects. This creates a sustainable funding cycle for CMT research.

Go-to-market motion1 record

Distribution channels4 records

Marketing channels8 records

Core offering

CMT Research Foundation funds translational and preclinical research for Charcot-Marie-Tooth disease through grants of typically $25,000–$200,000 per year to academic labs and biotech companies. It maintains a CMT Research Pipeline tracker and a Clinical Trial Finder, and convenes an annual Global CMT Research Convention. It supplements donations with a revenue-share partnership program that returns a portion of downstream licensing income back to the foundation.

Product overview

CMT Research Foundation operates as a patient-led non-profit organization focused on funding CMT (Charcot-Marie-Tooth disease) research rather than selling a traditional product. Its core offerings include research funding programs with grants ranging from $25,000 to $200,000+ per year, a comprehensive CMT Research Pipeline tracking therapies under development across small molecule, gene therapy, and biologics categories, a Clinical Trial Finder directory, educational content including the CMT 101 Video Series, and the annual Global CMT Research Convention. The foundation connects researchers, clinicians, biotech companies, and patients to accelerate treatments and cures for all types of CMT.

Differentiator

Problem solved

Functional benefit

Products and services

• CMT Research Funding Programs Milestone-gated research grants (typically $25,000–$200,000 per year) awarded to academic labs and biotech companies working on gene therapy, antisense oligonucleotides, small molecules, drug delivery, and biomarkers for Charcot-Marie-Tooth disease. Intended for academic principal investigators, translational researchers, and biotech/pharma R&D teams.
• CMT Research Pipeline A research pipeline database that tracks the status of CMT research programs funded by or affiliated with the foundation, available on the CMTRF website. Intended for researchers, clinicians, patients, and donors seeking transparency into active CMT programs.
• CMT Clinical Trial Finder An online directory of clinical trials relevant to CMT patients, accessible through the foundation's website. Intended for patients, families, and clinicians seeking enrollment information for CMT-related trials.
• Global CMT Research Convention Annual international convention organized by the foundation that convenes CMT researchers, clinicians, biotech and pharma partners, and patient community members to share research progress and foster collaboration. Intended for the global CMT research and patient community.
• Industry Partnership / Revenue-Share Program Formal partnership program with biotech and pharmaceutical companies in which CMTRF co-funds CMT research and receives a percentage of any downstream licensing income generated from resulting programs. Intended for biotech and pharmaceutical business development and R&D leaders.

Quantifiable outcome

• 6 therapeutics advanced to preclinical or active clinical trials through CMTRF funding
• +3 more outcomes

Named customers4 records

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

14 partnerships are on record, tiered core, major and minor.

• Novartis (via DTx Pharma)coreStrategic or Co-development Partner · 1 January 2023CMTRF introduced DTx Pharma to CMT research and funded their first CMT research with a grant of $128,000. DTx Pharma developed a platform called FALCON (Fatty Acid Ligand Conjugated Oligonucleotide) to deliver RNA therapeutics to peripheral nerves. Novartis acquired DTx Pharma in 2023 for $1 billion and is now advancing the CMT1A drug candidate EKD060 to clinical trials.
• Augustine TherapeuticscoreStrategic or Co-development PartnerCMTRF helped Augustine Therapeutics underwrite the development of novel HDAC6 inhibitor drugs for CMT1A. Augustine completed Series A financing of $85 million and advanced lead candidate AGT-100216 to Phase 1 clinical trial, with first patient dosed in May 2025.
• Armatus BiocoreStrategic or Co-development PartnerCMTRF-funded research from Dr. Kleopa (Cyprus Institute of Neurology and Genetics) and Dr. Harper (Nationwide Children's Hospital) was licensed to Armatus Bio. The gene therapy ARM-101 for CMT1A is preparing for clinical trials after successful preclinical studies.
• Elpida TherapeuticscoreStrategic or Co-development PartnerCMTRF provided $800,000 and CureCMT4J provided $700,000 to Elpida Therapeutics to launch manufacturing of gene therapy drug ELP-02 for CMT4J. Phase I/II trial planned for early-mid 2026.
• Johns Hopkins UniversitymajorStrategic or Co-development PartnerActive project: Find a blood-based biomarker that could measure whether future CMT treatments are working. Project led by Jeremy Sullivan and Charlotte Sumner.
• Nationwide Children's HospitalmajorStrategic or Co-development PartnerMultiple active projects including gene therapy development for CMT1B by Dr. Afrooz Rashnonejad, and AAV delivery optimization for Schwann cells. Dr. Scott Harper collaborated on CMT1A gene therapy later licensed to Armatus Bio.
• Cyprus Institute of Neurology and GeneticsmajorStrategic or Co-development PartnerDr. Kleopas Kleopa developed gene therapy approach for CMT1A that was funded by CMTRF and later licensed to Armatus Bio. Research focused on lowering PMP22 protein levels in peripheral nerves.
• The Jackson LaboratorymajorStrategic or Co-development PartnerDeveloping humanized mouse models for CMT1B that express human MPZ gene to study disease mechanisms and accelerate drug development.
• University of MiamimajorStrategic or Co-development PartnerDr. Stephan Zuchner developing mouse model of CMT1J and testing RNA therapeutic approach. Collaboration with 1J Foundation.
• ReviR TherapeuticsmajorStrategic or Co-development PartnerDeveloping small molecule splice modulators for CMT1A that reduce expression of overexpressed PMP22 gene. Program paused as ReviR focuses on advancing a clinical-stage molecule for CMT.
• AcuraStem (Licensed to Takeda)majorStrategic or Co-development PartnerCMTRF funded underlying technology for PIKfyve inhibitor targeting axon degeneration in CMT2A. Technology licensed to Takeda in September 2023. Provisional patent filed May 2023.
• Shift PharmaceuticalsmajorStrategic or Co-development PartnerDeveloping morpholino-based antisense oligonucleotides (PMO ASOs) targeting excess PMP22 protein for CMT1A treatment. Lead drug candidate identified and advancing toward clinical trials.
• Nanite Inc.minorStrategic or Co-development PartnerDeveloping polymer nanoparticles (PNPs) for drug delivery across the blood-nerve barrier to deliver genetic therapies to Schwann cells. Project closed; Nanite evaluating next strategic step.
• Peripheral Nerve SocietymajorOthersCMTRF research team attends annual Peripheral Nerve Society conference in the Netherlands to learn about latest advances, network with field leaders, and accelerate CMT treatment progress.

Scale indicators6 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• Charcot-Marie-Tooth Association (CMTA): The other primary U.S.-based nonprofit solely dedicated to CMT, funding research, patient support, and clinician education. Highly comparable as a fellow disease-specific CMT research foundation with overlapping mission.
• Hereditary Neuropathy Foundation (HNF): Patient-led nonprofit focused on Charcot-Marie-Tooth disease and inherited neuropathies. Directly comparable in mission (CMT cures), funding model (research grants, industry partnerships), and patient community engagement.

Broad incumbents

• Cystic Fibrosis Foundation: Pioneer of the disease-specific research funding model that led to multiple approved therapies. Comparable as a large-scale patient-led research foundation with venture philanthropy and revenue-share models that CMTRF explicitly mirrors.
• Muscular Dystrophy Association (MDA): Major nonprofit funding research across neuromuscular diseases including CMT. Comparable in mission to accelerate treatments for neuromuscular conditions, though broader than CMTRF's CMT-only focus.
• National Organization for Rare Disorders (NORD): Umbrella organization for rare disease patient groups. CMTRF holds NORD Platinum membership. Comparable as a research-funding and advocacy nonprofit in the rare disease space, though operating at much broader scale.
• ALS Association: Major patient-led nonprofit funding ALS/MND research globally with active industry partnerships. Comparable in patient-led research funding model and disease-specific drug development focus, though operating at much larger scale.

Emerging players

• Cure SMA: Patient-led nonprofit that funded spinal muscular atrophy research leading to approved gene therapies (Spinraza, Zolgensma). Highly comparable disease-specific research foundation model with similar revenue share and milestone-gated grant approach.
• Friedreich's Ataxia Research Alliance (FARA): Patient-driven rare disease research foundation funding drug development for Friedreich's ataxia. Comparable in scale, mission, and patient-led research funding approach for a rare inherited neurological disease.
• Parent Project Muscular Dystrophy (PPMD): Disease-specific nonprofit funding Duchenne muscular dystrophy research with venture philanthropy elements. Comparable in using milestone-gated grants, industry partnerships, and patient community leverage to drive drug development.
• Myelin Repair Foundation: Patient-focused foundation funding translational research on myelin-related disorders including peripheral neuropathies. Directly relevant given CMT's underlying demyelination pathology, though operating in adjacent rather than identical disease space.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat5 records

Key risks5 records

Key highlights7 records

Customer concentration

• Xtwitter.com/CMTRF
• YouTubeyoutube.com/@cmtresearchfoundation
• Facebookfacebook.com/CMTResearchFoundation
• LinkedInlinkedin.com/company/cmt-research-foundation
• Instagraminstagram.com/cmtresearchfoundation

Revenue estimate

Valuation estimate

Number of profiles

Profiles3 records

Funding overview

Funding rounds

Investors

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M&A

Investments6 records

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Frequently asked questions about CMT Research Foundation