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 Clinical Trials
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 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 29, 2012
Top-line Results of our Phase IIb Study of ALN-RSV01
We reported top-line results from our Phase IIb trial with ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection in lung transplant patients. The primary endpoint of the study was the incidence of new or progressive bronchiolitis obliterans syndrome (BOS) at 180 days after RSV infection.
Read our press release Listen to our discussion of the top-line results View our presentation (449 KB PDF) View additional ALN-RSV program materialsMay 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)April 20, 2012
Positive Clinical Results for ALN-PCS, Reported at ATVB 2012
Yesterday, scientists presented positive results from our Phase I clinical trial of ALN-PCS at the American Heart Association’s Arteriosclerosis, Thrombosis and Vascular Biology 2012 Scientific Sessions held in Chicago. Results showed that administration of a single dose of ALN-PCS, in the absence of concomitant lipid-lowering agents such as statins, resulted in statistically significant and durable reductions of PCSK9 plasma levels of up to 84% and lowering of LDL-C of up to 50%.
View our presentation (572 KB PDF) Read our press release (572 KB PDF)December 16, 2011
Podcast: ALN-TTR Phase I Preliminary Results
We recently presented preliminary clinical results for our Phase I study of ALN-TTR01 at the International Symposium on Familial Amyloidotic Polyneuropathy. In this new podcast, Dinah Sah, Ph.D., VP of Research and lead for the ALN-TTR program, discusses the ALN-TTR01 Phase I results she presented at the Symposium, late November. (Length 00:19:32)
Listen to the podcast (22.9 MB mp3) ALN-TTR01 PhI Prelim Results (821 KB PPT)November 21, 2011
ALN-TTR01 Preliminary Phase I Clinical Results
In November 2011, we presented positive preliminary data from our ALN-TTR01 Phase I study at the VIIIth International Symposium on Familial Amyloidotic Polyneuropathy. These data show that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels in ATTR patients and demonstrate human proof of concept for RNAi therapeutics.
Read our press release ALN-TTR01 PhI Prelim Results (821 KB PPT) Listen to replay of conference call (7.1 MB mp3December 26, 2007
