FAU and Sancilio Team Up on Therapy for Inherited Blindness

Researchers from FAU and Sancilio and Company, Inc. are collaborating to develop a treatment for Retinitis Pigmentosa, an inherited disease that causes severe progressive vision impairment and blindness.

By gisele galoustian | 1/30/2017

Researchers from Florida Atlantic University are collaborating with scientists from Sancilio and Company, Inc., in Riviera Beach, Fla., to begin a new research project aimed at finding a treatment for patients afflicted by Retinitis Pigmentosa (RP). RP is an inherited disease that causes severe progressive vision impairment and blindness. The disease is recognized as the leading cause of inherited blindness that affects approximately 1 in 4,000 people and can cause blindness as early as within the first year of life.

“There are currently no treatments available for this devastating progressive and degenerative eye disorder,” said Frederick Sancilio, Ph.D., president of Sancilio & Company, Inc. “By combining our knowledge, resources and expertise with neuroscientists from Florida Atlantic University we are hopeful that we will be able to further develop a treatment to halt this disease.”

Prior preclinical studies performed by scientists at Sancilio and Company, Inc. have demonstrated the potential therapeutic effect of SC412, an investigational drug that the company is currently developing for the treatment of this condition. In partnership with neuroscientists in FAU’s Charles E. Schmidt College of Science and Charles E. Schmidt College of Medicine, Sancilio and Company, Inc. will test the therapeutic effects of SC412 in a validated animal model for RP. In addition, the project also will investigate different formulations to define the best and most convenient route of administration.

Wen Shen, Ph.D., a neuroscientist, associate professor of biomedical science in FAU’s College of Medicine and a faculty member in FAU’s Brain Institute who has expertise in retinal physiology, will spearhead evaluating the new drug in the animal model. Her laboratory will carry out the initial testing of SC412 on the animal model for RP, investigating the effectiveness and safety of the compound and gathering the basic information for a preclinical trial.   

“Having positive results from these experiments will be a significant step closer to testing this molecule in human studies in the near future,” said Sancilio.

FAU neuroscience faculty members are tackling many of the cutting-edge questions in neuroscience through the integration of multiple disciplines, different model systems and a broad spectrum of technologies.