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 Alnylam’s presentation (1.6 MB PDF) View the Mt. Sinai presentation (1.2 MB PDF) Read our press releaseMay 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 programJanuary 30, 2013
Complete Study Results from our ALN-VSP Phase I Trial and Extension Study Published in Cancer Discovery
We have published complete study results from our Phase I clinical trial with ALN-VSP, an RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. Results from the study – the most comprehensive study of a systemically administered RNAi therapeutic to date – demonstrated proof of RNAi activity in man and evidence of anti-tumor activity, in addition to highlighting the safety and tolerability of multiple doses of ALN-VSP.
Read the abstract in Cancer DiscoveryDecember 10, 2012
New Pre-Clinical Data Presented with ALN-AT3 at ASH
Today we are presenting new data from our ALN-AT3 program for the treatment of hemophilia and other bleeding disorders at the 54th American Society of Hematology (ASH) Annual Meeting in Atlanta. ALN-AT3 comprises part of our “Alnylam 5×15™” strategy, which is aimed at bringing innovative medicines to patients, with a focus on RNAi therapeutics toward genetically defined targets for diseases with very high unmet medical need.
View our poster (2.3 MB PDF) Read our press releaseNovember 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.
September 4, 2012
Complete Results of our ALN-RSV01 Phase IIb Study
At the European Respiratory Society’s (ERS) Annual Congress held in Vienna September 1-5, 2012, we presented complete results from our Phase IIb trial with ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection in lung transplant patients… Continue reading…
August 6, 2012
New Data from Conjugate Delivery Platform
New data from our conjugate delivery platform was presented at the XX International Roundtable on Nucleosides, Nucleotides and Nucleic Acids held in August 2012. This delivery platform enables subcutaneous dose administration of RNAi therapeutics with a very wide therapeutic index. We are integrating this approach in our ‘Alnylam 5×15’ efforts with ALN-TTRsc for the treatment of ATTR, ALN-AT3 for the treatment of hemophilia, and potentially future programs.
View our presentation (1.6 MB PDF)
Read our press release (PDF)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 release