Ann Arbor-based biopharmaceutical company, ONL Therapeutics, announced that the U.S. FDA has granted orphan drug designation to ONL1204 for the treatment of retinal detachment.  ONL1204 is a novel, first-in-class small molecule peptide designed to protect key retinal cells, including photoreceptors, from cell death that occurs in a range of retinal diseases and conditions.  Death of these retinal cells is the root cause of vision loss and the leading cause of blindness.  ONL expects to advance ONL1204 into clinical trials for retinal detachment patients in 2016.

Retinal detachment occurs when the retina is separated from the underlying layer of cells called the retinal pigment epithelium (RPE). The RPE provides nutritional support to the highly-active photoreceptors in the retina. When there is a detachment, the photoreceptors no longer receive these nutrients and undergo cell death processes that dramatically impact a patient’s vision. Retinal detachments occur in approximately 50,000 people each year in the United States and affect people of all ages, although risk increases as people reach fifty years of age.

 

Source:

http://www.biospace.com/News/onl-therapeutics-receives-orphan-drug-designation/408615