(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.