Last week, uniQure achieved a breakthrough in developing disease-modifying therapies for Huntington’s Disease by showing that AMT-130 slowe...

Many industry insiders expected Sarepta CEO Doug Ingram to be out by now.  Instead, a new dynamic is gathering steam: Vinay Prasad, the rece...

This week, Alnylam announced in their roundabout way that it has discontinued the development of ALN-HBV for the treatment of chronic HBV...

When Biomarin late last year bought Prosensa for its experimental exon skipper drisapersen for the treatment of Duchenne Muscular Dystrop...

In a head-to-head comparative study in NatureMedicine , Goyenvalle and colleagues claim that tricycloDNAs (tcDNAs), a relatively unexplo...

With officials scrambling to put in place measures to contain the further spread of Ebola, it is highly likely that RNAi Therapeutic TKM-E...

I am writing to you today to introduce myself as a potential candidate for the CSO position at Sarepta.  Based on media reports last week ...

Following the successes with its Ebola biodefense program, Tekmira has started to present increasingly promising data for treating a rel...

I am aware that my interpretations of Sarepta’s exon-skipping data for Duchenne Muscular Dystrophy are not universally embraced.  But one ...

As most of you know, yesterday was the day that morpholino antisense company Sarepta would provide guidance on whether they would be seek...

Orphan drugs tailored to address defined genetic lesions account for a significant portion of the current value of RNA Therapeutics.  One...