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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.

The effects were also found to be rapid, dose-dependent, and durable, putting Alnylam on track to begin a phase III pivotal trial of the drug should data from a recently initiated phase II study prove positive, CMO Akshay Vaishnaw said during a conference call.

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At a meeting this week, researchers and others discussed the regulatory oversight needed for germline genome editing.

The US Food and Drug Administration has asked questions about Myriad Genetics' GeneSight test, according to Bloomberg.

Researchers report that neutrophil extracellular traps appear to binds gallstones together, according to New Scientist.

In Science this week: approach to infer genotype-by-environment interaction from genetic variants associated with phenotypic variability, and more.

Aug
28
Sponsored by
Horizon Discovery

This webinar will provide an overview of alternatives to the popular Cas9 nuclease used in CRISPR gene editing.

Oct
23
Sponsored by
Swift Biosciences

This webinar will illustrate how single-cell methylation sequencing can be applied to gain significant insight into epigenetic heterogeneity in disease states, advancing cancer research discoveries.