NEW YORK (GenomeWeb) – Regulus Therapeutics this week announced that the US Food and Drug Administration has granted orphan drug status to the company's preclinical Alport syndrome therapy RG-012.
The condition is a rare kidney disease 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 microR-21, an increase in fibrosis, and the loss of renal function, which ultimately leads to end-stage renal disease.
RG-012 is designed to silence miR-21. In mouse studies, inhibition of the miRNA has been shown to decrease the rates of decline of renal fibrosis while increasing the animals' life span.
Regulus is currently preparing to initiate a natural history of disease study to gather information on the progression of Alport syndrome and guide clinical development plans for RG-012.