Improvements to Bestrophin Inhibitors as a Therapy for Best Disease and Other Macular Dystrophies

Technology #ua06-049

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Olaf Strauss
Ronald Lynch
Professor, Physiology
Lihua Marmorstein
Assistant Professor, Ophthalmology & Vision Science
Brian Mckay
Associate Professor, Ophthalmology & Vision Science
Alan Marmorstein
Associate Professor, Ophthalmology & Vision Science
Managed By
Rakhi Gibbons
Asst. Director, Life Sciences (520) 626-6695

Title: Target for the Treatment of Best Disease

Invention: Scientists at The University of Arizona have developed strategies that can be employed in high throughput screens for small molecule drugs targeted for the prevention of vision loss due to Best disease and other macular degenerative diseases.

Background: Caused by a mutation in the VMD2 gene, Best vitelliform macular dystrophy is a dominantly inherited, early onset, macular degenerative disease that exhibits some similarities to Stargardt’s disease, another inherited macular dystrophy, and also to age-related macular degeneration, the leading cause of incurable blindness in the United States. Macular degenerative diseases eventually result in a debilitating loss of vision requiring a preventative therapy before serious damage occurs. Creation of bestrophin specific inhibitors may lead to development of preventative therapies for Best disease, which may also be of benefit in the prevention or treatment of Stargardt’s disease and age-related macular degeneration.


  • Development of the first preventative therapy for Best disease and other macular degenerative diseases
  • Screening for inhibitors that specifically target bestrophin activity used in prevention and treatment of macular degenerative conditions


  • Effective delivery of bestrophin inhibitors can prevent or slow the loss of vision

Licensing Manager:

Rakhi Gibbons

(520) 626-6695