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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

costs of developing and marketing a treatment drug. The granting of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. Safety and effectiveness of a drug must be established through adequate and well-controlled studies.

About Alport Syndrome

Alport syndrome is a life-threatening genetic kidney disease that impacts the body's ability to create a specific type of collagen highly expressed in the kidney and essential to normal kidney structure. In the absence of this type of collagen, the kidneys are unable to effectively filter toxins and waste products, resulting in end-stage renal disease and also hearing loss or effects on vision.  Currently, there is no approved therapy for Alport syndrome.

About RG-012 for the Treatment of Alport Syndrome

Regulus has discovered that miR-21 is highly overexpressed in mouse models of Alport syndrome. Regulus is developing RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of miR-21, which has demonstrated potent inhibition of miR-21 in vitro and in vivo, a decrease in the rate of progression of renal fibrosis, an increase in the lifespan of mice by up to fifty percent, and a favorable pharmacokinetic profile that supports the potential for a once per week dosing regimen.  In the near term, Regulus expects to initiate a natural history of disease study to gather greater information about the progression of Alport syndrome and expects to initiate a Phase I clinical study of RG-012 for the treatment of Alport syndrome in the first half of 2015.

About microRNAs

The discovery of microRNAs in humans during the last decade is one of the most exciting scientific breakthroughs in recent history.  microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression.
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SOURCE Regulus Therapeutics Inc.
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