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*

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.

Chinese state news agency Xinhua reports that a preliminary investigation has found He Jiankui performed his gene-editing work illegally.

John Mendelsohn, a former president of the University of Texas MD Anderson Cancer Center, has died, the New York Times reports.

Identical twins receive different estimates of ancestry from the same direct-to-consumer genetic testing firms, CBC reports.

In PNAS this week: chromosomal features of maize, adaptations in the vinous-throated parrotbill, and more.

Feb
21
Sponsored by
L7 Informatics

This webinar will provide a first-hand look at how Gradalis, a clinical-stage immunotherapy developer, is using an information management solution from L7 to streamline its research, clinical, and manufacturing operations.