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Transthyretin-mediated amyloidosis treatment shows promise

(Reuters) – Alnylam Pharmaceuticals Inc said its experimental drug to treat an organ-damaging hereditary disorder showed promise in an early-stage trial by suppressing a protein that causes the disease.

The drug aims to treat transthyretin-mediated amyloidosis (ATTR), a disease caused by mutations in the transthyretin (TTR) gene.

“Suppression of TTR, the disease-causing protein in ATTR, was found to be rapid, dose-dependent, durable, and specific after just a single dose,” Alnylam said in a statement.

Alnylam also said there were no serious adverse events or discontinuations in the study.

The main goal of the study was to evaluate the safety and tolerability of a single dose of the drug.

The company has already begun a mid-stage study and expects to enroll about 20 patients.

Alnylam develops drugs based on RNA interference, or RNAi — a mechanism of silencing or turning on genes to prevent formation of proteins that can cause diseases.