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Isis-AATRx
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
**Isis-AATRx** is an investigational antisense oligonucleotide developed by Isis Pharmaceuticals to treat **alpha-1 antitrypsin deficiency (AATD)-associated liver disease**. It works by binding to and reducing the expression of mutant alpha-1 antitrypsin (AAT) mRNA in hepatocytes, thereby lowering the production of misfolded AAT protein that accumulates in the liver and causes toxicity. Selected as a development candidate, it targets the liver-specific production of AAT, a protease inhibitor, aiming to prevent liver damage in AATD patients with the PiZ mutation, where polymerized Z-AAT leads to cirrhosis.[1]
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