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Alnylam Receives $7M Milestone from Genzyme


Alnylam Pharmaceuticals last month announced that it has received a $7 million milestone payment from Genzyme based upon its positive data from a phase II trial of the TTR-mediated amyloidosis treatment patisiran, formerly known as ALN-TTR02.

The disease is caused by mutations in the TTR gene, which triggers accumulation of abnormal amyloid proteins in the body. ALN-TTR02 comprises siRNAs designed to inhibit both the wild-type and mutant forms of the protein, and was licensed to Genzyme in Asia last year.

In November, Alnylam presented phase II data showing that treatment with the drug led to "robust and statistically significant" inhibition of its target protein. Around that time, the company also began enrolling patients in a phase III trial.