Сlinical trial for START Therapy, a first-ever pharmaceutical topical therapy for patients with aniridia.

1st January 2014 starts сlinical trial for START Therapy, a first-ever pharmaceutical topical therapy for patients with aniridia caused by a PAX6 nonsense mutation.  START Therapy was developed Dr. Cheryl Gregory-Evans, PhD and Dr. Kevin Gregory Gregory-Evans MD PhD at the University of British Columbia.  Drs. Gregory-Evans’ ground-breaking study on aniridic mice, to be published in the December 2013 issue of the Journal of Clinical Investigation, shows that the application of the START Therapy drop not only halted disease progression but also reversed corneal, lens, and retinal malformation defects and restored electrical and behavioral responses of the retina.

The human clinical trial will commence in January of 2014 and enroll 24 patients (children and adults) from three separate centers: (1) Eye Care Centre, Vancouver, BC, Canada, (2) Dept. Ophthalmology, Univ. of Virginia School of Medicine, Charlottesville, VA, USA., (3) Moorfields Eye Hospital, London, UK.  Patients must have a known PAX6 nonsense mutation.  Enrolled patients will receive START Therapy drops in one eye, four times a day for a period of one year.  Patients will travel to their study site quarterly for assessment.