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.
- Pharmaceuticals & Drug Trials
- American Society of Hematology