We initiated dosing in our Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). The Phase I trial of ALN-TTRsc is being conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects. The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc.
Read our press release Learn more about our ALN-TTR programPosts Tagged with: RNAi Therapeutics
July 16, 2012
Positive ALN-TTR02 Clinical Data
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 releaseJuly 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 releaseJune 4, 2012
Data from our ALN-VSP Phase I Extension Study Presented at ASCO
At the annual ASCO meeting in June 2012, we presented data from our ALN-VSP Phase I extension study. Overall, the results demonstrated disease control lasting more than six months in the majority of patients treated on the extension study, including a complete response (CR) in an endometrial cancer patient who had multiple liver metastases.
View our poster (3.72 MB PDF) View our releaseMay 10, 2012
Final Results from our Phase I Trial of ALN-TTR01
Today, scientists presented final results from our Phase I clinical trial of ALN-TTRo1 at the XIII International Symposium on Amyloidosis held in Groningen. Data from this study show that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein, in ATTR patients. Knockdown of TTR, the disease-causing protein, was found to be dose dependent, rapid, and durable after just a single dose.
View our presentation (842 KB PDF) View our release Listen to KOL, Dr. Hawkins discuss the ALN-TTR program (recorded Jan. 2012) (27.7MB MP3)March 1, 2012
Delivery Strategies for RNAi Therapeutics
Our scientists presented an update on our RNAi delivery strategies at the AsiaTIDES conference this week. The presentation focuses on our recent progress utilizing conjugates and lipid nanoparticles for systemic adminstration of RNAi therapeutics.
View our presentation (2.4MB PDF)