Alnylam Pharmaceuticals Inc said its experimental gene-based therapy reduced the level of a disease-causing protein, which leads to a rare organ-damaging hereditary disorder, in an early-stage trial.
The therapy was also found to be generally safe and well tolerated, Alnylam said in a statement.
The trial was testing Alnylam's subcutaneously delivered therapy for the treatment of an inherited, progressively debilitating and fatal disease caused by mutations in the transthyretin (TTR) gene.
The disease, called TTR-mediated amyloidosis or ATTR, causes abnormal proteins to accumulate in body organs and tissue.
Alnylam's therapy led to consistent and statistically significant reduction of TTR protein levels of up to 94 percent in the study.
The therapy is based on Alnylam's gene silencing technology that works by "switching off" certain disease-causing genes and is widely believed to hold immense potential for treating diseases ranging from cancer to HIV.
The company now plans to start a pilot mid-stage trial by the end of this year, and assuming positive results, a late-stage study by the end of 2014.
Alnylam shares closed at $57.23 on Friday on the Nasdaq.
(Reporting by Esha Dey in Bangalore; Editing by Sreejiraj Eluvangal)