Arrowhead Preclinical Data Demonstrates RNAi Therapy Prevents and Reverses Alpha-1 Liver Disease at The International Liver Congress™
In the study presented at ILC, PiZ mice were treated with ARC-AAT for 33 weeks. Measurements of plasma Z-AAT levels, Z-AAT protein accumulation in the liver, and expression of genes previously implicated in liver injury and development of fibrosis were taken. In addition, histological evaluations of globules and inflammation and an ultrastructural evaluation of the liver by electron microscopy were performed.
Key results include the following:
Sustained RNAi reduction of Z-AAT substantially reversed the AATD
- Deeply reduced monomeric Z-AAT protein in the liver
- Reduced polymeric Z-AAT in the liver
- Prevented the dramatic increase in globule size seen in age-matched control PiZ mice
- Improved abnormal endoplasmic reticulum morphology
- Prevented inflammation
- Prevented/reduced expression of fibrosis, redox-regulation, stress, apoptosis and autophagosome-associated genes
- Resulted in abundance of healthy mitochondria
Poster Presentation Details:
Reduction of hepatic Z-alpha1 antitrypsin by RNA interference (RNAi) prevents and reverses liver disease including hepatic mitochondrial injury in the PiZ mouse
- Presentation Reference: FRI-446
- Session: Poster: Rare liver diseases (including pediatric and genetic)
Session Date and Time:
April 12, 2019at 9:00 a.m. to 5:00 p.m. CET
Christine Wooddell, et al.
Additional details, including the presentation abstract, can be found on the ILC website at https://ilc-congress.eu/. A copy of presentation materials can be accessed by visiting the Events section under the Investors tab of the Arrowhead website.
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Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA