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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Researchers hope to tease out the signature effects that different carcinogens leave on the genome to determine their contributions to disease, Mosaic reports.

The Wall Street Journal looks into the cost of new gene therapies.

An Imperial College London-led team reports that it was able to use a gene drive to control a population of lab mosquitos.

In PNAS this week: genomic effects of silver fox domestication, limited effect of mitochondrial mutations on aging in fruit flies, and more.