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belcesiran
- Development stage
- Phase 2
- Lead developer
- Novo Nordisk
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Small Molecules, Peptide-Drug Conjugates → Peptide Conjugates → Peptides, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Belcesiran is a synthetic, double-stranded small interfering RNA (siRNA) therapeutic developed for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (AATLD). It is administered subcutaneously and utilizes RNA interference (RNAi) to target the gene responsible for producing abnormal alpha-1 antitrypsin (AAT) protein in the liver, thereby reducing its production. Belcesiran incorporates proprietary GalXC technology from Dicerna Pharmaceuticals. The drug was investigated in Phase 1 and Phase 2 clinical trials under programs such as SHINE and ESTRELLA, aiming to address the unmet need for therapies targeting AATLD by modulating AAT expression at the genetic level[1][2][4][5][6].
- Other names
- belcesiranDcr-a1atDcr-a-1atDcr-a 1at
02
Targets
SERPINA1 (Alpha-1-antitrypsin)
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