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transthyretin-mediated amyloidosis

The percentage of perfectly modified alleles using the new base editor is more than 6,000-fold for disease correction compared with the current BE4max editor.

Alnylam Pharmaceuticals and collaborators this week published data from phase I trials of the siRNA-based transthyretin amyloidosis drugs ALN-TTR01 and ALN-TTR02 in the New England Journal of Medicine.

Alnylam Pharmaceuticals this week said that it has begun dosing patients in a phase I study of its siRNA-based transthyretin-mediated amyloidosis treatment ALN-TTR02.

Alnylam also released its first-quarter financial results, reporting higher losses and lower revenues.

Specifically, the designation is for the use of the drug against familial amyloidotic polyneuropathy, one of the predominant forms of the disease, Alnylam said.

The company also disclosed that it and partner Cubist Pharmaceuticals have decided to put on hold the development of a second-generation version of Alnylam's respiratory syncytial virus treatment, although no details regarding the decision were provided.

The Washington Post reports that Herbert Tabor, who worked at the US National Institutes of Health for 77 years, has died at 101.

The World Bank is seeking approval for a $12 billion plan to provide low-income nations with funds to procure SARS-CoV-2 vaccines, according to Reuters.

Science writes that public health officials and others are debating whether cycle threshold values should be included on SARS-CoV-2 results.

In Nucleic Acids Research this week: online database of SARS-CoV-2 protein structures, atlas of the human brain, and more.