Alnylam’s gene therapy shows strong efficacy data

(Reuters) – 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. …