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December 8, 2013
20:29 EDTALNYAlnylam presents pre-clinical results on ALN-CC5 at ASH
Alnylam Pharmaceuticals announced that it has presented new pre-clinical data with ALN-CC5, a subcutaneously administered RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. These data were presented at the 55th Annual Meeting of the American Society of Hematology, ASH, held December 7 – 10, in New Orleans, and demonstrate that subcutaneous administration of ALN-CC5 in non-human primates, NHPs, led to an up to 98% knockdown of serum C5 and an up to 94% inhibition of hemolytic activity. Alnylam believes that ALN-CC5 – part of the company’s “Alnylam 5x15” product strategy – represents a novel approach for the treatment of complement-mediated diseases, with a potentially competitive profile compared with intravenously administered anti-C5 monoclonal antibody therapy. In addition, Alnylam presented two separate posters with new pre-clinical data on ALN-TMP, an RNAi therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and iron-overload disorders.
News For ALNY From The Last 14 Days
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November 19, 2014
07:52 EDTALNYInforma Business Information to hold a conference
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November 17, 2014
17:03 EDTALNYAlnylam presents new pre-clinical data on RNAi therapeutic programs
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07:09 EDTALNYAmerican Heart Association to hold a conference
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November 14, 2014
06:31 EDTALNYAlnylam to host conference call
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06:04 EDTALNYAlnylam announces positive initial phase 2 data with Revusiran
Alnylam announced positive initial Phase 2 data with revusiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of TTR cardiac amyloidosis. In the pilot Phase 2 study, revusiran was found to be generally well tolerated in TTR cardiac amyloidosis patients. Revusiran demonstrated clinical activity with an up to 98.2% knockdown of serum TTR – the disease causing protein. This included similar knockdown effects toward wild type and mutant TTR protein within V122I patients, who represent the most common genotype associated with inherited forms of TTR cardiac amyloidosis. In the five week course of treatment, there were no significant changes observed in a number of exploratory clinical measurements. Revusiran utilizes Alnylam’s proprietary GalNAc-conjugate delivery platform that enables subcutaneous delivery of RNAi therapeutics with a wide therapeutic index. The primary objective of the study was to evaluate the safety and tolerability of revusiran. The secondary objectives were to assess the clinical activity of revusiran toward serum levels of TTR and characterize the drug’s pharmacokinetic profile. In addition, a number of exploratory clinical measurements were performed at baseline and days 42 and 90 after start of dosing.
November 11, 2014
08:03 EDTALNYAlnylam adds ALN-HDV, ALN-PDL to hepatic infectious disease pipeline
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