While Alport syndrome currently remains without a cure, ongoing research is testing several strategies that hold promise, a review study shows. The review, “New therapeutic options for Alport syndrome,” was published in the journal Nephrology Dialysis Transplantation. Alport syndrome is an inherited disease caused by mutations in the…

A microRNA — a small RNA molecule that helps regulate how other genes are expressed — is present at high levels in the kidneys of people with Alport syndrome. Experiments in mice further suggest that this microRNA could be a therapeutic target for treatment of the disease. The study, “…

Regulus Therapeutics and Sanofi-Genzyme are among companies working on Alport syndrome therapies that can block RNA molecules’ actions. RG-012 is a man-made version of naturally occurring oligonucleotides, or RNA molecules that help regulate gene activity. The synthetic version, called antisense oligonucleotides, binds to RNA, preventing it from functioning normally. But what are…

Regulus Therapeutics, a pharmaceutical company dedicated to the development of microRNA treatments, announced that the design of the Phase 2 clinical program to test its product RG-012 as a potential therapy for Alport syndrome has been subject to changes aimed at speeding up patient enrollment and improving result analysis so that…

Regulus Therapeutics has completed dosing of its Alport syndrome drug candidate RG-012 in a Phase 1 trial testing several ascending doses of the treatment in healthy volunteers. The treatment was found safe and well-tolerated, and no serious adverse events were seen during the trial. The company also announced it…

RG-012, a drug to treat renal function decline in patients with Alport syndrome, is moving into Phase 1 clinical trial, says its developer, Regulus Therapeutics. The company, based in La Jolla, Calif., expects this new trial to overcome the setback announced last November on its RG-012 Phase 2 study,…