51勛圖厙

51勛圖厙 and Sancilio Team Up on Therapy for Inherited Blindness

Researchers from 51勛圖厙 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 are collaborating with scientists from , 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 , president of By combining our knowledge, resources and expertise with neuroscientists from 51勛圖厙 we are hopeful that we will be able to further develop a treatment to halt this disease.

Prior preclinical studies performed by scientists at 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 51勛圖厙s and , 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.

, a neuroscientist, associate professor of biomedical science in 51勛圖厙s and a faculty member in 51勛圖厙s 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.

51勛圖厙 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.

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