Are antagomirs the best compounds that could be practical for treating multiple human diseases? Could companies like Alnylam (ALNY) initiate first clinical trials using antagomirs relatively soon? I believe the answers to both questions are yes.

The paper published a couple of days ago by the scientists from Alnylam, the Rockefeller, and NY University (see the abstract below) provides perhaps the most convincing evidence from animal studies that RNAi-related approaches could be practical for treating multiple human diseases. While the experiments were done in mice, there is little doubt that the approach could be tailored to humans. Two points in the paper are critical. First, there is very specific and potent inhibition of the targeted miRNAs. Second, the application of the