ATTR

Both drugs are administered subcutaneously using Alnylam's GalNAc conjugates.

NEW YORK (GenomeWeb) – Arcturus Therapeutics has announced that it has added a second drug candidate to its transthyretin-mediated amyloidosis (ATTR) program.

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.

Alnylam Pharmaceuticals this week announced positive data from a phase II trial of its TTR-mediated amyloidosis drug patisiran, formerly ALN-TTR02, showing that the drug triggered "robust and statistically significant" inhibition of its target protein.

Alnylam Pharmaceuticals this week provided an update on its flagship TTR-mediated amyloidosis drug ALN-TTR02, stating that it has begun enrollment in an open-label extension study of a recently completed phase II trial and unveiling the compound's international nonproprietary nam

Alnylam Pharmaceuticals this week announced that it has completed enrollment in a phase II trial of its lipid nanoparticle-formulated TTR-mediated amyloidosis treatment ALN-TTR02.

Alnylam Pharmaceuticals this week announced positive interim data from a phase I study of its subcutaneously delivered TTR amyloidosis treatment ALN-TTRsc, which show the drug can cut levels of its disease-causing protein by roughly 90 percent.

While RNAi drug developers continue to pursue major indications such as cancer and hepatitis C, a number of these companies are looking to niche diseases as good opportunities to apply the gene-silencing technology.

Alnylam Pharmaceuticals this week announced that it plans to report initial data from an ongoing phase II trial of its lead drug candidate, the TTR-mediated amyloidosis treatment ALN-TTR02, this summer at an upcoming scientific meeting, and that it will launch an expansion study

Alnylam Pharmaceuticals this week announced that it has started a phase I trial of its subcutaneous TTR-mediated amyloidosis treatment ALN-TTRsc.

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