NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week unveiled initial top-line data from an ongoing Phase I study of its siRNA-based hemophilia drug ALN-AT3, which showed the drug is capable of significantly knocking down its target after a single, subcutaneous dose.

Alnylam also announced that it has expanded its pipeline to include a hepatitis B program, which it acquired when it bought the RNAi assets of Merck earlier this year.

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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.