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Regulus Receives Orphan Drug Designation for RG-012, a microRNA Therapeutic for the Treatment of Alport Syndrome
Date:7/21/2014

LA JOLLA, Calif., July 21, 2014 /PRNewswire/ -- Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that the U.S. Food & Drug Administration (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 ("miR-21"), as a therapeutic for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy.

"We are pleased to have received orphan drug designation for RG-012 and are encouraged by the FDA's recognition for the need of innovative new treatments like microRNA therapeutics for rare and orphan diseases such as Alport syndrome," said Paul Grint, M.D., Chief Medical Officer of Regulus.  "Alport syndrome is a life threatening disease and patients have very limited treatment options because there is currently no approved therapy.  We believe that RG-012 represents an opportunity to make a significant impact in the lives of patients with Alport syndrome and we look forward to advancing this program into the clinic."

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'. 

About Orphan Drug Designation

According to the FDA, the Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected the
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SOURCE Regulus Therapeutics Inc.
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