phase I data

The data also point to viral load reductions regardless of disease genotype, extent of patient liver fibrosis, or failure with other treatments.

Quark Pharmaceuticals last week announced “favorable” interim results from a phase I trial of its siRNA-based ocular neuroprotectant QPI-1007, showing the drug may improve the vision of patients with a rare eye condition.

Alnylam partner Tekmira Pharmaceuticals said it views the data as a validation of its drug-delivery technology, which will be used in two new oncology programs it unveiled this week.

The disclosure comes almost a year after Calando Pharmaceuticals published phase I data demonstrating that its own cancer drug CALAA-01 could knock down its intended target mRNA and protein inside a tumor through an RNA interference mechanism when delivered intravenously into humans.

The phase I drug, ALN-VSP02, comprises two siRNAs, one targeting vascular endothelial growth factor and one targeting kinesin spindle protein, both of which the firm said are critical for the growth and survival of cancer cells.

The data, which appeared in the online edition of Nature, "demonstrate that RNAi can occur in a human from a systemically delivered siRNA, and that siRNA can be used as a gene-specific therapeutic," the investigators wrote.

A research duo estimates in PLOS One the number of papers that have used misidentified cell lines.

UK's National Institute for Health and Care Excellence approves GlaxoSmithKline's SCID gene therapy despite cost.

Science reports that Brazilian researchers are petitioning for the reversal of budget cuts.

In PLOS this week: gene flow patterns in common ash, guidelines for using morpholinos in zebrafish, and more.