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Regulus And Alport Syndrome Foundation Raise Awareness Of Life-Threatening, Genetic Kidney Disease

LA JOLLA, Calif., March 25, 2014 /PRNewswire/ -- Regulus Therapeutics Inc. ( NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, and the Alport Syndrome Foundation ("ASF"), a non-profit corporation, announced today they are working together to raise awareness of Alport Syndrome, a life-threatening genetic kidney disease driven by genetic mutations with no approved therapy. 

This month, Regulus will join the Alport Syndrome Foundation in observing National Kidney Month and the first ever Alport Syndrome Awareness Month.  As part of that effort, Regulus hosted an Alport Syndrome awareness day at Regulus' corporate headquarters in San Diego, CA. Alport Syndrome patients Sharon Lagas and Ryan Landwehr were invited to provide Regulus employees with patient perspectives on the impacts of living with Alport Syndrome.  Additionally, Regulus will serve as a Spirit of Action sponsor of the Alport Syndrome Foundation 5K for Healthy Kidneys on Sunday, March 30 th, 2014 in Tempe, Arizona.  For more information, please

"We are grateful for the support from Regulus and know that it will take patients working with companies like Regulus to advance the search for potential Alport Syndrome treatment options," said Sharon Lagas, President of the Alport Syndrome Foundation. 

"Partnerships are critical to advancing scientific research for orphan diseases such as Alport Syndrome," said Kleanthis G. Xanthopoulos, Ph.D., President and CEO of Regulus. "We are hopeful that our relationship with the Alport Syndrome Foundation will advance our understanding of the disease and believe that ASF will serve as an invaluable resource to us as we develop RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport Syndrome.  We are excited to initiate a natural history of disease study for RG-012 in the third quarter this year and expect to initiate a Phase I clinical study of RG-012 in the first half of 2015."

Alport Syndrome is a genetic condition caused by mutations in the COL4A3, COL4A4 or COL4A5 genes which 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. Lack of this special type of collagen can also result in hearing loss or effects on vision. Alport Syndrome is considered an orphan disease with no approved therapy.

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