Alnylam says it has acheived RNAi first
Alnylam Pharmaceuticals Inc. said that it has established a significant first with results from a research trial that support its theory of creating drugs and treatments based on RNA interference, or RNAi.
Specifically, the Cambridge biopharmaceutical company announced today that its Gemini trial has achieved "human proof of concept with an RNAi therapeutic, a first for the industry."
Alnylam is seeking to develop novel technologies based on RNA interference, or RNAi, a naturally occurring mechanism within cells for selectively silencing and regulating specific genes; because many diseases are caused by the inappropriate activity of specific cells, the ability to silence genes selectively through RNAi is thought to have great potential.
Today the company said that results from its Gemini trial with ALN-RSV01, an RNAi therapeutic for the treatment of respiratory syncytial virus, or RSV, showed statistically significant anti-viral efficacy.
“With the positive results from Gemini, we believe that Alnylam has demonstrated the first ever human proof of concept with an RNAi therapeutic,” Alnylam chief executive John Maraganore said in a statement. “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. While there’s more to do in our efforts, these results also highlight Alnylam’s continued leadership in the field of RNAi therapeutics, and our commitment to advancing this promising new class of medicines to patients.”
Alnylam said it has scheduled a conference call this morning to discuss the results of the Gemini tests further.
(By Chris Reidy, Globe staff)






