NEW YORK (GenomeWeb) – Arrowhead Research announced today that it has filed an application with regulators to begin a Phase I trial of its alpha-1 antitrypsin deficiency (AAT) drug ARC-AAT in Australia.

AAT deficiency is a genetic disease that leads to decreased activity of the protease inhibitor alpha-1 antitrypsin in the blood and lungs causing lung dysfunction. It can also feature the accumulation of a mutant AAT protein known as Z-AAT in liver tissue, which leads to liver injury, fibrosis, cirrhosis, and potentially liver cancer.

Get the full story with
GenomeWeb Premium

Only $95 for the
first 90 days*

A trial upgrade to GenomeWeb Premium gives you full site access, interest-based email alerts, access to archives, and more. Never miss another important industry story.

Try GenomeWeb Premium now.

Already a GenomeWeb Premium member? Login Now.
Or, See if your institution qualifies for premium access.

*Before your trial expires, we’ll put together a custom quote with your long-term premium options.

Not ready for premium?

Register for Free Content
You can still register for access to our free content.

A former Synthetic Genomics attorney alleges that the firm discriminated against her and other female employees, according to the San Diego Union-Tribune.

Due to privacy and lab certification questions, the planned giveaway of Orig3n testing kits at a Baltimore Ravens game was suspended.

Alnylam reports positive results from its phase 3 clinical trial of an RNAi-based drug, according to Stat News.

In Cell this week: adult mesenchymal cell populations in mouse lung, genetic diversity in HPV16 and cancer risk protection, and more.