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Regulus Names Alport Syndrome as Next Pipeline Indication


Regulus Therapeutics this week announced that has formally added its preclinical program in the rare kidney disease Alport syndrome to its drug-development pipeline — a move widely expected based on company comments and recently released animal data linking microRNA-21 to the disease.

Alport syndrome is caused by mutations in three genes that affect production of the type IV collagen family of proteins. The result is a disruption to the structure of the glomerular basement membrane, increased expression of miR-21, an increase in fibrosis, and the loss of renal function, which ultimately leads to end-stage renal disease, according to the company.

Late last year, Regulus announced that in a mouse model of Alport syndrome, inhibiting miR-21 "significantly decreased the rate of decline of renal fibrosis, restored the expression of key microRNAs involved in maintaining renal function, and increased the lifespan of the mice by 20 percent to 50 percent depending upon the genetic background of the collagen 4A3 mutation."

At the time, company officials also indicated that the disease would likely form the foundation of its next drug candidate, which has now been dubbed RD-012.

"This program underscores Regulus' focus on orphan disease indications, and we look forward to rapidly advancing and expanding our clinical portfolio of meaningful microRNA therapeutics," Regulus President and CEO Kleanthis Xanthopoulos said in a statement.

As reported by Gene Silencing News earlier this month, the Alport syndrome program is covered by Regulus' recently restructured alliance with Sanofi, which has the right to take over RG-012's development after Regulus has established clinical proof of concept.