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Regulus Therapeutics granted orphan drug designation for RG-012 by FDA

Regulus Therapeutics announced that the FDA has granted orphan drug designation to 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 the near term, Regulus expects to initiate a natural history of disease study to gather further information about the progression of Alport syndrome and to inform future clinical development plans of RG-012, a key program under Regulus' 'Clinical Map Initiative'.