First miRNA drug enters human studies

Denmark's Santaris has kicked off a Phase I study of SPC3649, a potential treatment for hepatitis C infection. The trial marks the first time a microRNA drug has been tested in humans. SPC3649 will be tested for safety in 48 healthy volunteers before being administered to hepatitis patients in a Phase II trial.

MicroRNA is one of two types of RNAi found in all human cells and affects how genes are turned off and on (siRNA is the other). "The mechanism of action of this drug represents a potential breakthrough in medical science. The ability to switch off the functions of particular microRNAs may enable clinicians to modulate entire networks of genes associated with disease or ill-health," said Santaris' CEO.

- check out Santaris' release for more