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.

In the study, approximately 20 ATTR patients will receive increasing doses of the drug once every four weeks for two cycles, the company said. “The primary objectives of the study are to evaluate the safety and tolerability of multiple doses of ALN-TTR02 and to measure clinical activity based on serial measurement of circulating serum TTR levels.”

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The UK's Nuffield Council on Bioethics says genetically modifying human embryos could be morally permissible, according to the Guardian.

A new Nature Biotechnology paper reports that CRISPR-Cas9 gene editing can lead to large deletions or complex rearrangements that could be pathogenic.

The Wall Street Journal likens a prototype developed by Synthetic Genomics to a "biological fax machine."

In PNAS this week: strategy for reactivating Rett syndrome-linked MECP2, small molecules able to suppress Staphylococcus aureus virulence, and more.

Jul
19
Sponsored by
Thermo Fisher Scientific

This webinar will discuss how ultra-highly sensitive and customizable targeted next-generation sequencing panels are applied in inherited disease research. 

Aug
07
Sponsored by
Qiagen

This webinar will present the results of an evaluation of a web-based variant interpretation software system for clinical next-generation sequencing.