Close Menu

NEW YORK (GenomeWeb) – Arrowhead Research today announced that it has begun dosing in a Phase I trial of its siRNA-based alpha-1 antitrypsin (AAT) deficiency therapy ARC-AAT.

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. ARC-AAT is designed to lower production of Z-AAT.

Get the full story with
GenomeWeb Premium

Only $95 for the
first 90 days*

GenomeWeb Premium gives you:
✔ Full site access
✔ Interest-based email alerts
✔ Access to archives

Never miss another important industry story.

Try GenomeWeb Premium now.

You may already have institutional access!

Check if I qualify.

Already a GenomeWeb or 360Dx Premium member?
Login Now.

*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.

Nature News reports that recent proposed changes to the US National Science Foundation have raised concerns about a shift away from the agency's focus on basic research.

Noel Rose, the "father of autoimmunity," has died at 92, the Washington Post reports.

According to CNN, Legionella was discovered in buildings leased by the Centers for Disease Control and Prevention as they reopened following coronavirus pandemic-related closures.

In PLOS this week: genetic analysis of malaria parasite populations in Southeast Asia, genomic surveillance of yellow fever virus in São Paulo, and more.