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TTR amyloidosis

NEW YORK (GenomeWeb) – Officials from Alnylam Pharmaceuticals last week provided updates on the two drug candidates from the company's flagship transthyretin-mediated amyloidosis program, stating that the intravenously delivered agent patisiran is proceeding toward a possible market approval in t

NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week unveiled new Phase II data on its transthyretin-mediated amyloidosis (ATTR) therapy patisiran, which showed that the drug is capable of knocking down its disease-causing protein target by about 80 percent in patients — in line with previous

Alnylam Pharmaceuticals this week announced that its transthyretin-mediated amyloidosis drug ALN-TTRsc has received orphan drug status from European regulators.

Arcturus Therapeutics this week announced new data showing that its proprietary lipid nanoparticles are capable of functionally delivering messenger RNA into the livers of mice, which a company official said opens the door for the use of the technology with CRISPR-based drugs.

Officials from Alnylam Pharmaceuticals last week provided updates on the company’s pipeline programs, highlighting efforts involving its GalNAc conjugation technology, which enables subcutaneous siRNA delivery, and a fledgling effort in complement-mediated diseases.

Alnylam Pharmaceuticals this week reported top-line results from an ongoing phase I trial of its subcutaneously administered TTR-mediated amyloidosis treatment ALN-TTRsc, which showed the drug could knock down serum levels of its target protein by more than 80 percent..

Alnylam Pharmaceuticals this week announced positive clinical results from an ongoing phase II trial of ALN-TTR02, an investigational treatment for TTR-mediated amyloidosis, reporting that the drug was able to knock down levels of the protein that causes the disease by as much as

Alnylam Pharmaceuticals last week announced that partner Cubist Pharmaceuticals has dropped out of its deal to develop Alnylam's investigational respiratory syncytial virus drug ALN-RSV01 following a failed phase IIb trial last year.

Though not defined by any one major breakthrough as in years past, the RNAi therapeutics field made steady progress in 2012, particularly in terms of product and technology development, according to industry insiders.

Isis Pharmaceuticals this week announced that the US Food and Drug Administration has fast tracked its antisense-based transthyretin amyloidosis drug ISIS-TTRRx for use in patients with familial amyloid polyneuropathy, the rarer form of the disease.

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CBS This Morning highlights recent Medicare fraud involving offers of genetic testing.

Researchers find that many cancer drugs in development don't work quite how their developers thought they did, as Discover's D-brief blog reports.

Mariya Gabriel, a Bulgarian politician, is to be the next European Union research commissioner, according to Science.

In Science this week: a survey indicates that US adults are more likely to support the agricultural use of gene drives if they target non-native species and if they are limited, and more.