WARF: P06414US

Tat-Utrophin as a Protein Therapy for Muscular Dystrophies


James Ervasti, Kevin Sonnemann

The Wisconsin Alumni Research Foundation (WARF) is seeking commercial partners interested in developing a fusion protein for the treatment of muscular dystrophies.
OVERVIEWNo effective therapy currently exists for the most common and severe form of muscular dystrophy disease, Duchenne Muscular Dystrophy (DMD). This disease, which is characterized by the weakening of voluntary muscles, is caused by the lack of a functional version of the muscle protein dystrophin.

Another muscle protein, utrophin, has a similar sequence and has been used to compensate for the loss of dystrophin in a mouse model. However, up-regulating utrophin or increasing the expression of functional dystrophin in muscle cells is difficult and will not be a feasible treatment option until clinically approved methods of gene therapy are developed.
THE INVENTIONUW-Madison researchers have developed a fusion protein between utrophin and the TAT sequence from HIV, which is used to transport molecules into cells, for the treatment of muscular dystrophies, including DMD. This protein-based therapeutic provides the first effective treatment for these currently fatal conditions.

The TAT sequence provides a cell signal that leads to the internalization of utrophin in muscle cells. Once in the muscle cells, the utrophin is able to compensate for the lack of dystrophin, reducing or eliminating the disabling effects of dystrophinopathic conditions.

The inventors successfully treated a mouse model for DMD by injecting the fusion protein intraperitoneally. The TAT-utrophin localized to the target area of muscle, resulting in significant improvement in several key disease parameters.
  • Treatment of muscular dystrophies, including DMD
  • Because it is protein-based, this treatment circumvents the need for gene therapy.
  • Relatively low cost and low risk
  • Because utrophin is not toxic, the fusion protein can be administered in relatively high doses, making it easier to transduce therapeutically effective amounts into muscle cells
  • Successfully used in mice
  • Truncated versions of the fusion protein may be used to improve protein transduction in vivo.
Related Intellectual Property
Contact Information
For current licensing status, please contact Andy DeTienne at or 608-960-9857.
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.