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Regulus Therapeutics receives orphan drug designation for RG-012.

M2 EQUITYBITES-July 23, 2014-Regulus Therapeutics receives orphan drug designation for RG-012


Regulus Therapeutics, a United States-based biopharmaceutical company, has received orphan drug designation for its RG-012, a single stranded, chemically modified oligonucleotide from the US Food and Drug Administration, it was reported yesterday.

The product binds to and inhibits the function of microRNA-21 (miR-21), as a therapeutic to treat Alport syndrome, a genetic kidney disease.

The firm is planning to start a natural history of disease trial to gather further information about the progression of Alport syndrome and to inform future clinical development plans of RG-012, a key programme under its 'Clinical Map Initiative'. Regulus chief medical officer, Paul Grint, stated that the company is happy to secure orphan drug designation for RG-012 and are encouraged by the FDA's recognition for the need of new treatments like microRNA therapeutics for rare and orphan diseases, such as Alport syndrome.

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Publication:M2 EquityBites (EQB)
Date:Jul 23, 2014
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