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Regulus Begins Natural History of Disease Study for Alport Syndrome


NEW YORK (GenomeWeb) – Regulus Therapeutics announced last week that it has initiated a natural history of disease study, called ATHENA, in patients with Alport syndrome.

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 microRNA-21, an increase in fibrosis, and the loss of renal function, which ultimately leads to end-stage renal disease.

Regulus is developing RG-012, a miR-21 inhibitor, for the condition and aims to have it in Phase I testing next year.

Ahead of that planned trial, the company has launched ATHENA to characterize the natural decline of renal function markers such as glomerular filtration rate, creatinine, proteinuria, and beta-2 microglobulin in Alport syndrome patients over time.

The study is set to run for two years in up to 120 Alport syndrome patients. Results are expected to inform future clinical development of RG-012, the company said.