Skip to main content
Premium Trial:

Request an Annual Quote

Arrowhead Begins Phase I Trial of AAT Deficiency Drug

Premium

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.

The trial is testing single doses of the drug, first in healthy volunteers and then in AAT deficient patients. Its objectives are to evaluate the compound pharmacokinetics and determine its effects on circulating levels of AAT.

Initial data from the study are expected later this year.

The Scan

Billions for Antivirals

The US is putting $3.2 billion toward a program to develop antivirals to treat COVID-19 in its early stages, the Wall Street Journal reports.

NFT of the Web

Tim Berners-Lee, who developed the World Wide Web, is auctioning its original source code as a non-fungible token, Reuters reports.

23andMe on the Nasdaq

23andMe's shares rose more than 20 percent following its merger with a special purpose acquisition company, as GenomeWeb has reported.

Science Papers Present GWAS of Brain Structure, System for Controlled Gene Transfer

In Science this week: genome-wide association study ties variants to white matter stricture in the brain, and more.