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ALN-AAT02
- Development stage
- Discontinued
- Lead developer
- Novo Nordisk
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Subcutaneous
01
Overview
ALN-AAT02 is an investigational, subcutaneously administered small interfering RNA (siRNA) therapeutic developed for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (alpha-1 liver disease). It works by targeting and reducing the production of alpha-1 antitrypsin (AAT) in the liver through RNA interference, aiming to prevent the accumulation of misfolded AAT protein that leads to liver injury, fibrosis, cirrhosis, and hepatocellular carcinoma in affected patients. The drug utilizes Alnylam’s Enhanced Stabilization Chemistry Plus (ESC+)-GalNAc-conjugate technology for improved delivery and selectivity. ALN-AAT02 was initially developed by Alnylam Pharmaceuticals and later partnered with Dicerna Pharmaceuticals; Dicerna was subsequently acquired by Novo Nordisk. Clinical development reached Phase 1/2 trials but has since been discontinued[1][2][3][4].
- Other names
- belcesiran
02
Targets
SERPINA1 (Alpha-1-antitrypsin)
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