We presented pre-clinical proof-of-concept data from our ALN-AS1 program for the treatment of acute intermittent porphyria (AIP) at the International Congress of Porphyrins and Porphyrias being held May 16 – 18, 2013. The research, presented by Alnylam scientists and collaborators at the Icahn School of Medicine at Mount Sinai in New York City, showed that administration of a GalNAc-conjugated siRNA targeting aminolevulinate synthase-1 (ALAS-1) blocked production of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic precursors that cause disease symptoms and pathophysiology, in a mouse model of AIP.
View our presentation (1.6 MB PDF) Read our press releasePosts Tagged with: 5×15
May 17, 2013
New Pre-Clinical Data Presented from our ALN-AS1 Program for the Treatment of Acute Intermittent Porphyria
March 18, 2013
Phase I Clinical Study for ALN-TTRsc
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 programNovember 14, 2012
New Pre-clinical Data with ALN-AAT for the Treatment of Alpha-1 Antitrypsin Deficiency
At the Liver Meeting held this week in Boston, our scientists presented pre-clinical data from our ALN-AAT program for the treatment of liver disease associated with alpha-1 antitrypsin (AAT) deficiency. AAT deficiency is a rare, genetic condition characterized by misfolding of mutant AAT (“Z-AAT”) that causes lung and liver disease.
View our presentation (2.2 MB PDF) View our poster (2.8 MB PDF)November 1, 2012
8th Annual Meeting of the Oligonucleotide Therapeutics Society
At the 8th Annual Meeting of the Oligonucleotide Therapeutics Society, held in Boston October 28-31, 2012, we presented exciting data covering several of the company’s therapeutic programs and delivery platforms.
THERAPEUTIC PROGRAMS
Therapeutic research highlighted recent clinical and pre-clinical work from our programs in transthyretin-mediated amyloidosis (TTR), hemophilia, hypercholesterolemia, and liver cancer. Click here for presentations.
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 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)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)January 4, 2012
ALN-PCS Preliminary Phase I Clinical Results
In January 2012, we presented positive preliminary data from our ALN-PCS Phase I study at Brigham and Women’s Hospital in Boston. Data show that ALN-PCS demonstrated robust, statistically significant, and dose-dependent lowering of both PCSK9 and LDL cholesterol levels.
Read our Press Release ALN-PCS Phase I Data Presentation (3.6MB PPT) Listen to Conference CallDecember 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)December 13, 2011
Alnylam Scientists Present New Data on RNAi Therapeutics at 53rd ASH Annual Meeting
In December 2011, our scientists presented multiple posters and presentations at the 53rd American Society of Hematology (ASH) Annual Meeting and Exposition held in San Diego, CA. We shared data from two programs within our “Alnylam 5×15” product pipeline, including pre-clinical data from ALN-APC, an RNAi therapeutic targeting protein C for the treatment of hemophilia,…
View the refractory anemia presentation (680 KB PPT) View the hemophilia poster (1.88 MB PDF) View the beta-thalassemia poster (112 KB PDF) View the erythropoiesis poster (435 KB PDF) Read our press release