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ALN-CIDEB
- Development stage
- Phase 2
- Lead developer
- Regeneron Pharmaceuticals
- 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-CIDEB is an experimental RNA interference (RNAi) therapeutic targeting the CIDEB gene, which encodes a structural protein involved in hepatic lipid droplet formation and lipid metabolism. The drug is being developed for the treatment of metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH), both of which are chronic liver conditions characterized by excessive fat accumulation in the liver. By silencing CIDEB expression, ALN-CIDEB aims to reduce hepatic triglyceride storage and improve liver health, based on evidence that loss-of-function mutations in CIDEB are associated with protection against fatty liver disease. The drug is currently under investigation in Phase 1 clinical trials to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics.
- Other names
- GalNAc-siCIDEB
02
Targets
GSDMB (Cell death-inducing DFFA-like effector B)
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