The data demonstrated that an RNA interference (RNAi) therapeutic can achieve deep durable, and dose dependent reductions of alpha-1 antitrypsin (AAT), with the highest dose studied in healthy volunteers achieving near full suppression of the liver production of the AAT protein. In addition, at doses of 2 and 4 mg/kg in AATD patients, ARC-AAT produced similar levels of knockdown as seen in healthy volunteers. These data support the continued advancement of ARO-AAT, Arrowhead's follow-on product candidate that utilizes the company's next generation, proprietary subcutaneously administered delivery vehicle.
Presentation materials can be accessed by visiting the Events section of the Arrowhead website. Additional details including abstracts can be found on the ILC website at https://ilc-congress.eu/ by searching for the following:
Hepatic targeted RNA interference provides deep and prolonged knockdown of alpha-1 antitrypsin levels in ZZ patients
Arrowhead announced on
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