What Are New Treatments and Trials for Friedreich Ataxia?

While there is currently no cure for Friedreich ataxia (FA), an active and growing landscape of clinical research is focused on developing one. The first disease-specific treatment, omaveloxolone, was approved in the United States (for patients aged 16 and older) to help slow physical decline ^[1]. Omaveloxolone is not a cure and requires careful clinician monitoring ^[1][2].

However, its approval demonstrates that treatments can successfully meet regulatory requirements. Now, researchers are testing emerging investigational therapies aimed at the core biological deficit of FA—a lack of the protein frataxin, which is caused by an expanded GAA sequence in the FXN gene ^[3]. (Note: The landscape described below is current as of late 2026, and trial statuses can change rapidly).

Investigational Therapies Aimed at Frataxin Deficiency

Because FA is a genetic condition, several experimental approaches aim to restore frataxin levels in the body’s cells, particularly in the heart and nervous system. None of the experimental approaches described below have yet been proven to restore lost neurological function or cure the disease.

Gene Therapy

Gene therapy attempts to deliver a working copy of the FXN gene into cells. This is usually done using AAV vectors—engineered, non-replicating viral delivery vehicles. While these vectors cannot cause a viral infection, they are not risk-free; they can provoke severe immune reactions and liver toxicity ^[4][5].

• Early Clinical Trials for the Heart: Two early-stage (Phase 1/2) open-label trials (NCT05302271 and NCT05445323) have tested an intravenous AAV gene therapy called AAVrh.10hFXN specifically for adults with FA-related cardiomyopathy (heart disease) ^[6].
• Initial Biomarker Results: In a pooled update of 17 participants, researchers found increased frataxin in heart biopsies and a decrease in left-ventricular mass (a measure of heart wall thickening) in some participants ^[6]. However, these are early structural and biomarker findings from a small group without a randomized placebo control. They do not prove that the therapy improves heart symptoms, coordination, or neurological function.
• Safety Considerations: These early trials carry serious risks. Out of 17 participants, four experienced serious adverse events that eventually resolved, including one case of suspected myocarditis (heart inflammation) possibly linked to the vector, and three linked to the immunosuppressant drugs required for the therapy ^[6]. Additionally, animal studies have shown that while restoring frataxin is beneficial, forcing cells to produce too much frataxin can cause mitochondrial and heart toxicity, meaning dosing must be tightly controlled ^[7][8].

Protein Replacement Therapy

Protein replacement therapy attempts to deliver functional frataxin protein directly into the mitochondria (the energy-producing structures inside cells).

• Nomlabofusp (formerly CTI-1601): This is a subcutaneous (under-the-skin) injection currently being studied ^[9].
• Early Phase Findings: Phase 1 trials (which primarily evaluate safety and dosing) involving over 50 adults showed that nomlabofusp temporarily increased frataxin levels in skin and cheek cells ^[9]. Treatment was generally well-tolerated over a short study period (up to 13 days) ^[9].
• Unproven Clinical Benefit: Frataxin increases in skin or cheek cells do not prove that enough of the protein is reaching the brain, spinal cord, or heart ^[10]. Longer-term studies are needed to see if this therapy safely improves neurological symptoms.

CRISPR and Gene Editing

CRISPR is a genetic editing tool. In FA research, scientists are exploring ways to use CRISPR to remove or interrupt the expanded GAA sequence in the DNA ^[11][12].

• Strictly Preclinical: Currently, CRISPR for FA is entirely preclinical. It is being tested in laboratory cells and animal models, and has not been shown to be safe or effective in humans ^[3][4].
• Laboratory Findings: In highly controlled lab settings, editing patient-derived stem cells successfully restored frataxin expression and improved motor function in mice ^[11][4]. The assays used in these specific experiments did not detect major “off-target” effects (unintended genetic changes) ^[11].
• Significant Hurdles: The biggest challenge facing CRISPR is delivery. It is extremely difficult to get the editing tools into enough cells in the human central nervous system and heart to make a meaningful difference ^[13]. For instance, one animal study testing AAV-delivered CRISPR found that only a fraction of heart and liver cells were successfully edited, which was not enough to boost overall frataxin levels ^[14].

How to Find and Participate in Clinical Trials

Many clinical trials are supported by the Collaborative Clinical Research Network (CCRN) in FA, an international network of research centers associated with advocacy groups like the Friedreich’s Ataxia Research Alliance (FARA) and Ataxia UK ^[15][16].

If you are considering participating in research, keep the following in mind:

• Trial Registries: Organizations like FARA maintain registries that can alert you to recruiting studies ^[15]. You can also search ClinicalTrials.gov using specific trial identifiers (NCT numbers) to find official contact information ^[17].
• Strict Eligibility Criteria: Eligibility varies drastically between protocols. A trial may require genetic confirmation, a specific age of symptom onset, specific cardiac findings, or a specific score on the mFARS (modified Friedreich Ataxia Rating Scale, a standardized measure of FA function administered by your clinician) ^[17]. Some trials may exclude patients based on ambulation status or prior treatments ^[17][18].
• Natural History Studies: Studies like TRACK-FA are observational natural history studies ^[17]. They involve MRIs and clinical tests to track how the disease progresses, which helps design future trials, but they do not provide an experimental treatment ^[17][19].
• Understand the Demands and Risks: Trial participation is voluntary. Experimental treatments may not help you, and you may be randomly assigned to receive a placebo. Consider the physical and financial burdens, such as travel, frequent blood draws, out-of-pocket costs, and the risk of required immunosuppression ^[15][17]. Always discuss research enrollment with your treating neurologist or FA specialist to weigh it against your ongoing multidisciplinary care.