At the Boston University School of Medicine, our scientists presented positive clinical results from our Phase I trial with ALN-TTR02 for the treatment of transthyretin-mediated amyloidosis (ATTR). These new data show that we have achieved very robust knockdown of TTR, the disease causing protein, including up to 94% reduction of serum TTR, and a nearly 80% level of suppression sustained at one month with just a single dose. ALN-TTR02 was found to be generally safe and well tolerated.
View our presentation (758 KB PDF) Read our press releaseMonthly Archives For: July 2012
July 11, 2012
New Pre-clinical Data with our Hemophilia Program
At the World Federation of Hemophilia World Congress in July 2012, scientists presented pre-clinical data with ALN-AT3, our development candidate targeting antithrombin (AT) for the treatment of hemophilia. The new data demonstrate potent and durable AT knockdown with subcutaneous administration of ALN-AT3. ALN-AT3 was shown to improve thrombin generation in hemophilia animal models.
View our poster (slide format) (774 KB PDF) View our poster (3.3 MB PDF) View our releaseJuly 10, 2012
