Experimental gene-silencing drug from Alnylam and Sanofi shows strong results in hemophilia
An experimental hemophilia drug developed by Alnylam Pharmaceuticals with partner Sanofi all but stopped bleeding in many patients in a clinical trial.
An experimental hemophilia drug developed by Alnylam Pharmaceuticals continues to staunch bleeding in patients followed for almost one year in an ongoing, mid-stage clinical trial, the company reported Monday.
The promising results could intensify an already heated competition to develop novel treatments for the inherited bleeding disorder. The Alnylam drug, developed in partnership with Sanofi, uses a technology called RNAi to shut down dysfunctional genes. It’s a promising approach that’s attracted billions in research dollars, but hasn’t yet been used in an actual drug approved for sale.
The company says the new data support its
