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抖M女仆 and Sancilio Team Up on Therapy for Inherited Blindness

Researchers from 抖M女仆 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.

鈥淭here are currently no treatments available for this devastating progressive and degenerative eye disorder,鈥 said , president of 鈥淏y combining our knowledge, resources and expertise with neuroscientists from 抖M女仆 we are hopeful that we will be able to further develop a treatment to halt this disease.鈥

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 抖M女仆鈥檚 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 抖M女仆鈥檚 and a faculty member in 抖M女仆鈥檚 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.聽 聽

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

抖M女仆 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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