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TTR amyloidosis

Isis Pharmaceuticals this week said that it stands to receive a $1.1 million payment tied to Alnylam Pharmaceuticals' strategic alliance with Genyzme.

This story has been updated to include information about Arrowhead Research's reverse stock split.

Alnylam Pharmaceuticals this week released positive phase I data on its investigational TTR-mediated amyloidosis treatment ALN-TTR02, reporting that the drug triggered up to 94 percent reductions in levels of its target protein after a single dose.

Alnylam Pharmaceuticals said this week that the US Food and Drug Administration has given orphan drug status to the company's investigational transthyretin-mediated amyloidosis ALN-TTR02 as a treatment for familial amyloidotic polyneuropathy, a common manifestation of the disease

Alnylam Pharmaceuticals announced this week that it has begun a phase II trial of its TTR-mediated amyloidosis drug ALN-TTR02 to test the compound's clinical activity, safety, and tolerability.

Alnylam Pharmaceuticals this week released final data from a phase I study of its TTR-mediated amyloidosis drug ALN-TTR01, which showed the compound triggered dose-dependent and durable reductions in target protein levels.

This story has been updated from a previous version to include comment from Arrowhead Research.

By Doug Macron

The study is being conducted in Portugal, Sweden, and the UK and will enroll about 28 patients with the disease.

The company said that it expects to begin clinical testing of the drug in early 2010.

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Kelvin Droegemeier, the director of the White House Office of Science and Technology Policy, is the new acting director of the US National Science Foundation.

An opinion piece at the Guardian discusses the state of SARS-CoV-2 testing in the UK.

Wired reports the University of California, Berkeley's Innovative Genomics Institute has transformed itself into a diagnostic lab to run SARS-CoV-2 tests.

In Nature this week: direct-capture Perturb-seq approach for combinatorial single-cell CRISPR screens, potential uses of genome-editing in breeding crops, and more.