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Arrowhead Takes Last Steps toward Moving HBV Drug into Human Testing

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Arrowhead Research said this week that it has begun the final processes required to file an investigational new drug application for its RNAi-based hepatitis B treatment ARC-520, including manufacturing, toxicology studies, and preparation of a pre-IND data package for submission to US regulators.

The drug was originally under development by Roche, which quit the RNAi therapeutics space in 2010 and sold off related assets to Arrowhead the next year (GSN 10/27/2011). Arrowhead unveiled the HBV program earlier this year, and pegged a first-half-2013 timeframe for the IND filing (GSN 5/10/2012).

"The promotion of ARC-520 as a clinical candidate, so soon after acquiring the Roche RNAi assets, represents an important milestone for Arrowhead," the company's President and CEO Christopher Anzalone said in a statement this week.

The Scan

Interfering With Invasive Mussels

The Chicago Tribune reports that researchers are studying whether RNA interference- or CRISPR-based approaches can combat invasive freshwater mussels.

Participation Analysis

A new study finds that women tend to participate less at scientific meetings but that some changes can lead to increased involvement, the Guardian reports.

Right Whales' Decline

A research study plans to use genetic analysis to gain insight into population decline among North American right whales, according to CBC.

Science Papers Tie Rare Mutations to Short Stature, Immunodeficiency; Present Single-Cell Transcriptomics Map

In Science this week: pair of mutations in one gene uncovered in brothers with short stature and immunodeficiency, and more.