While RNAi drug developers continue to pursue major indications such as cancer and hepatitis C, a number of these companies are looking to niche diseases as good opportunities to apply the gene-silencing technology.

Notably, this trend aligns with big pharma’s increasing interest in orphan and ultra-rare diseases, keeping RNAi therapeutics an attractive option for pharmaceutical firms despite the high-profile suspension of in-house RNA drug programs by Roche and Pfizer in late 2010 and early 2011, respectively.

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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.