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May 17, 2013
New Pre-Clinical Data Presented from our ALN-AS1 Program for the Treatment of Acute Intermittent PorphyriaWe 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 release -
March 18, 2013
Phase I Clinical Study for ALN-TTRscWe 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 program -
January 30, 2013
Complete Study Results from our ALN-VSP Phase I Trial and Extension Study Published in Cancer DiscoveryWe 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 Discovery -
January 2, 2013
RNAi Roundtable: Conjugate DeliveryIn December 2012, we hosted a roundtable discussion focused on our activities and progress with our conjugate platform to deliver RNAi therapeutics subcutaneously. The roundtable included a review of our two leading conjugate programs: ALN-TTRsc for the treatment of transthyretin mediated amyloidosis, and ALN-AT3 for the treatment of hemophilia and other bleeding disorders.
Listen to the replay View the presentation (254 KB PDF)
Recent Posts
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May 22, 2013
Alnylam Corporate Overview, May 2013 Corporate Overview, May 2013 (2.o MB PPT) -
April 25, 2013
Monocyte-Directed RNAi Targeting CCR2 Improves Infarct Healing in Atherosclerosis-Prone MiceMajmudar et al., Circulation. 2013 Apr24 (Epub doi: 10.1161/CIRCULATIONAHA.112.000116) Continue reading…
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December 8, 2012
An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe-/- mice and ameliorates anemia and iron overload in murine β-thalassemia intermediaSchmidt et al., Blood. 2013 Feb14 (Epub doi: 10.1182/blood-2012-09-453977 )
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November 21, 2012
An immobilized and reusable Cu(i) catalyst for metal ion-free conjugation of ligands to fully deprotected oligonucleotides through click reaction.
