WARF: P160232US05

Method and Compounds for Treating Friedreich’s Ataxia


Aseem Ansari, Graham Erwin, Matthew Grieshop

The Wisconsin Alumni Research Foundation (WARF) is working with University of Wisconsin–Madison researchers developing a potential therapeutic agent to restore frataxin levels to treat and/or prevent Friedreich’s ataxia, a rare disease with no cure or effective treatment to date.

The agent has been shown in vitro to increase frataxin gene expression in a Friedreich’s ataxia patient cell line.
OVERVIEWFriedreich’s ataxia (also referred to as FA or FRDA) is a rare but fatal autosomal recessive neurodegenerative disease. With an estimated incidence of 1 in every 40,000 people, it causes progressive damage to the nervous system and muscles, resulting in loss of coordination as well as various neurological and cardiac complications.

There is presently no cure or specific therapy to prevent progression of Friedreich’s ataxia. Onset of symptoms is typically seen between the ages of 5 and 15, and the mean age of death is approximately 38 years.

The disease is caused by mutations in the frataxin (FXN) gene. Specifically, an abnormal number of GAA trinucleotide repeat sequences results in reduced frataxin expression. The severity of the disease correlates with the number of repeats. Therefore, restoring or partially restoring frataxin in affected cells may slow or prevent disease progression.
THE INVENTIONUW–Madison researchers have developed a chimeric synthetic molecule capable of inducing increases in FXN mRNA levels in Friedreich’s ataxia patient cell lines.

The complex has the formula A-L-B wherein A- is a bromodomain inhibitor, -L- is a linker, and -B is a polyamide that specifically binds to one or more repeats of a GAA oligonucleotide sequence. Mechanistically, when the complex binds to the repeat sequence, the bromodomain inhibitor (ex., Brd4) recruits the superelongation complex (SEC) to restart the paused transcription complex.
  • Potential therapeutic to prevent, treat or delay the symptoms of Friedreich’s ataxia
  • Potent and specific activation of gene expression
STAGE OF DEVELOPMENTThe patent application shows that agents of the present technology increase frataxin levels in various FRDA patient cells.
Contact Information
For current licensing status, please contact Joshua Carson at or 608-960-9844.
The WARF Advantage

WARF: A Leader in Technology Transfer Since 1925
Since its founding as a private, nonprofit affiliate of the University of Wisconsin–Madison, WARF has provided patent and licensing services to UW–Madison and worked with commercial partners to transform university research into products that benefit society. WARF intellectual property managers and licensing staff members are leaders in the field of university-based technology transfer. They are familiar with the intricacies of patenting, have worked with researchers in relevant disciplines, understand industries and markets, and have negotiated innovative licensing strategies to meet the individual needs of business clients.

The University of Wisconsin and WARF –
A Single Location to Accelerate Translational Development of New Drugs

UW–Madison has the integrative capabilities to complete many key components of the drug development cycle, from discovery through clinical trials. As one of the top research universities in the world, and one of the two best-funded universities for research in the country, UW–Madison offers state-of-the-art facilities unmatched by most public universities.

These include the Small Molecule Screening Facility at the UW Comprehensive Cancer Center; the Zeeh Pharmaceutical Experiment Station, which provides consulting and laboratory services for developing formulations and studying solubility, stability and more; the Waisman Clinical Biomanufacturing Facility; the Wisconsin Institute for Medical Research, which provides UW–Madison with a complete translational research facility; and the innovative, interdisciplinary Wisconsin Institutes for Discovery, home to the private, nonprofit Morgridge Institute for Research and its public twin, WID, part of the university's graduate school. The highly qualified experts at these facilities are ready to work with you to create a library of candidates for drug development.