Alnylam is offering up the first signs that RNAi therapy may work in humans. In a small Phase II trial, Alnylam's ALN-RSV01 for respiratory syncytial virus showed statistically significant anti-viral efficacy. Subjects receiving ALN-RSV01 experienced a 38 percent reduction in infection rate, proving that the therapy was effective in humans. Gene silencing is one of the most promising new technologies in drug development and drug developers large and small are pouring money into researching its applications for various diseases
"With the positive results from GEMINI, we believe that Alnylam has demonstrated the first ever human proof of concept with an RNAi therapeutic," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "As such, these new data represent a key milestone and a major 'de-risking' event for the advancement of RNAi therapeutics, as well as important progress for our RSV program." Another Phase II trial of the drug is planned this year.
- see this Alnylam release
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