This is the first demonstration of the efficacy of short interfering RNA at therapeutically relevant doses, and the company believes the study results establish a strong scientific foundation for broad human application of RNA interference based therapeutics.
Short interfering RNA are molecules that trigger “RNA interference” which is a natural, selective process for turning off genes. Sirna’s modified short interfering RNA molecules remain in the blood for longer than unmodified molecules, and it is this longer life in serum that is responsible for the efficacy seen in the study.
“We are extremely pleased by the significant knockdown of hepatitis B with low doses of our encapsulated siRNA,” stated Sirna Senior vice president and chief scientific officer, Dr Barry Polisky, “Sirna is building on these landmark results as we focus our human clinical program on hepatitis C”.
Sirna expects to commence early stage human clinical trials in 2006.