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Regulus Therapeutics Inc receives orphan drug designation for RG-012

Monday, 21 Jul 2014 08:00am EDT 

Regulus Therapeutics Inc:U.S. FDA granted orphan drug designation to RG-012.RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21, as a therapeutic for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy.In near term, Regulus expects to initiate natural history of disease study to gather further information about progression of Alport syndrome and to inform future clinical development plans of RG-012, key program under Regulus' 'Clinical Map Initiative. 

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