Alnylam takes its name from the centre star of Orion’s belt, which shines with a luminosity 250,000 times greater than the sun. That’s an indication of the company’s belief in RNA interference (RNAi) therapeutics. The US biopharmaceutical company is developing gene silencing technology for TTR amyloidosis (ATTR), and has now announced some positive early-stage results with a novel therapy for the treatment of the rare and debilitating disease.
The Phase I trial tested Alnylam’s ALN-TTRsc, one in a galaxy of many RNAi drug candidates in the company’s pipeline, that suppresses production of the protein transthyterin (TTR). Build-up of the TTR protein can damage body organs and tissues, including nervous and cardiac tissue. Gene silencing technology offers the potential to switch off the mutated TTR gene implicated in ATTR. Indeed, administration of Alynlam’s subcutaneous therapy in the study reduced TTR protein levels by up to 94%.
“These new ALN-TTRsc results are a major milestone in our ATTR program, as well as our entire pipeline of RNAi therapeutics. Specifically, we have demonstrated robust, up to 94% knockdown of circulating TTR with a very encouraging safety profile. We believe this level of consistent TTR knockdown is exceptional and unmatched, and we now aim to advance ALN-TTRsc in future clinical studies with the goal of achieving approximately 90% TTR knockdown to maximize clinical efficacy,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam.
Read the press release >
The World Orphan Drug Congress Europe brings together pioneers from the orphan drug community, including key opinion leaders from the payer, HTA, public health and patient advocacy bodies. Download the brochure for the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.