Alnylam receives OK for early stage drug testing
Drug developer Alnylam Pharmaceuticals Inc. said that three European countries have cleared it for early-stage clinical testing of a potential treatment for a hereditary disease caused by a genetic mutation.
The Cambridge company said Portuguese, Swedish, and British officials gave clearance to start testing of an RNAi therapy for the treatment of transthyretin-mediated amyloidosis.RNAi therapies are designed to stop the genetic causes of illnesses by turning off proteins that are at the root of a disease. The trial will be conducted in all three countries. The company will study the safety and tolerability of a single dose of its treatment in 28 patients with the disease.