CMT Cure Accelerator

The CMT Cure Accelerator is HNF's venture-philanthropy engine, and it is where your generosity becomes science. It does more than write grant checks. It deploys grants and equity capital across the whole pipeline that moves Charcot-Marie-Tooth toward treatments: targeted research, the disease models a therapy is tested in, drug repurposing, and the registry data that makes trials possible. When you give here, you are funding the cure on purpose. Your gift is not charity. It is acceleration.

An ecosystemGrants and equity capital across the whole CMT pipeline, not a single fund.

Direct impactYour gift converts into research, not overhead.

Flagship: GDAP1A priority program for one of the most severe forms of CMT.

Milestone-reviewedProjects are evaluated by HNF's TRIAD Council.

What the Cure Accelerator funds

Every dollar is aimed at the work that gets a therapy closer to patients. HNF funds preclinical research that aligns with our mission, with no fixed application deadline, so promising science can move the moment it is ready.

Gene therapy Developing and de-risking gene therapy approaches for specific CMT subtypes, starting with the hardest-hit.

Disease models Building the cell and animal models researchers need to test whether a candidate therapy actually works.

Drug repurposing Screening already-approved drugs for new use against CMT, a faster path from lab to patient.

Natural history Funding the GRIN registry data that makes any clinical trial possible in the first place.

How your gift becomes research

1 You give Your donation goes into the Cure Accelerator, a fund built to move targeted CMT research forward fast.
2 We fund the best science HNF directs dollars to the most promising preclinical projects, evaluated against clear milestones by our TRIAD Council.
3 Researchers hit milestones Funded labs report progress against measurable goals, so support keeps flowing to the work that delivers.
4 Therapies move toward the clinic Validated models and de-risked candidates move closer to a clinical trial, and closer to patients.

The flagship program

GDAP1: a gene therapy program for CMT4A and CMT2K

GDAP1 is one of the most severe forms of Charcot-Marie-Tooth disease. It can begin as early as infancy and progress to affect muscles throughout the body, with sensory loss and foot and hand deformities. Children and families living with it cannot afford to wait.

The Cure Accelerator's flagship effort is a gene therapy program targeting GDAP1, the gene behind CMT4A and CMT2K. HNF helped fund the development of a CMT4A research model that is owned by the foundation, the kind of tool a therapy has to be tested in before it can ever reach a patient. Alongside it, stem cell work is creating patient-derived cells to support both the gene therapy and drug repurposing efforts.

The Cure Accelerator exists so a program like this does not stall for lack of the unglamorous pieces a cure actually needs: a research model to test in, patient-derived cells to study, and the funding to keep both moving. What is learned here rarely stays with one subtype either. The tools and the know-how built around GDAP1 help lay the groundwork for the other forms of CMT waiting behind it.

A smiling young boy living with CMT sits on a bed wearing space-patterned leg braces, holding his orthotic braces with his sneakers beside him.

The data engine

Powered by real patient data

Targeted research needs targeted data. Through the Genesis Project, a genomic data platform for sharing CMT genome-level information, HNF's GRIN registry data is curated and stored so researchers can understand the disease and identify patients for future clinical trials. Your gift to the Cure Accelerator helps keep that engine running.

Patient data means better research and better-designed clinical trials. That is the throughline of everything the Cure Accelerator funds.

25 years of infrastructure. Now we need speed.

HNF did not wait for pharmaceutical companies to notice CMT. For 25 years we built the data, the models, and the relationships that made it impossible to ignore. No other organization has built what HNF has, and every piece of it is actively accelerating the path to treatment. What we need now is fuel.

GRIN Patient Registry: the only IRB-approved, patient-led global registry for CMT.
CMT Biobank: the first biorepository dedicated to CMT, with biospecimens available to qualified researchers anywhere in the world.
FDA Patient-Focused Drug Development meeting: the first patient organization to convene the FDA for CMT.
Transgenic rat models: for CMT4A, CMT-SORD, and CMT2A. Real translational research, not just theory.
The TRIAD network: 17 years connecting academia, government, and industry around CMT drug development.

The science is ready. What brought us here was infrastructure, data, and a community that refused to wait. What carries us into the clinic is funding, so the science we have built can move from the lab to the people who need it most.

None of us gets there alone.

Allison Moore, CEO & Founder, Hereditary Neuropathy Foundation

Frequently asked questions

What is the CMT Cure Accelerator?

It is HNF’s venture-philanthropy engine for Charcot-Marie-Tooth disease. Rather than only writing grant checks, it deploys a mix of grants and equity capital across the whole pipeline: targeted research, the cell and animal models a therapy is tested in, drug repurposing, and the GRIN registry data that makes a clinical trial possible. The goal is to close the gap to first-in-human trials, the stage where most rare-disease science runs out of money and stalls.

Why does HNF use equity capital, not just grants?

Taking an equity position alongside grant funding gives HNF a seat at the table from early science onward, aligns our urgency with the company’s, and means returns from a successful program can flow back into the fund to pay for the next one. Your gift does not disappear into a single experiment. It compounds, and it pulls industry capital in behind it.

Is the Accelerator only about GDAP1?

No. GDAP1, behind some of the most severe early-onset forms of CMT, is a priority, but the Accelerator is the whole ecosystem: multiple high-conviction subtype programs, disease models, drug repurposing, and the registry and biobank that every program depends on. GDAP1 is the flagship, not the boundary.

Does my gift guarantee a cure?

No, and we will not pretend otherwise. There is no approved disease-modifying therapy for CMT yet. What a gift to the Accelerator buys is a real shot on goal and the speed to take it. Whether a therapy can halt, partially reverse, or prevent CMT will depend on the subtype and how early it is caught. There have been real wins, and you can see them on our Years of Firsts page.

How do I give to the Cure Accelerator?

You can give through our secure donation page at /donate, the canonical route for every gift. For a larger or multi-year commitment, HNF can walk you through exactly what your capital funds and report back on where it lands; reach out through our contact page.

Fund the cure

Give to the CMT Cure Accelerator and put your gift directly behind the science that moves Charcot-Marie-Tooth toward treatments. This is acceleration, not charity, and you are the engine.

Fuel the CMT Cure Accelerator See our wins

Want the full science behind the work your gift funds? Explore Research → or see every way to give →