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December 9, 2012
12:26 EDTALNYAlnylam presents new pre-clinical data on RNAi therapeutics
Alnylam Pharmaceuticals announced that it has presented new pre-clinical data from its RNAi therapeutic program for the treatment of hemoglobinopathies at the 54th American Society of Hematology Annual Meeting being held December 8-11, 2012 in Atlanta. Alnylam scientists presented data showing that ALN-TMP, an RNAi therapeutic targeting Tmprss6, leads to disease modifying effects, including a correction in globin gene expression, in a model of ß-thalassemia. These studies were conducted in collaboration with Boston Children’s Hospital. Alnylam’s program in hemoglobinopathies comprises part of its ‘Alnylam 5x15™’ product strategy, by which the company aims to advance five programs in clinical development, including programs in advanced stages, by the end of 2015.
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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.

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