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ALN-BCAT
- Development stage
- Phase 1
- Lead developer
- Alnylam 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
- Intravenous
01
Overview
ALN-BCAT is an investigational RNA interference (RNAi) therapeutic developed by Alnylam Pharmaceuticals for the treatment of hepatocellular carcinoma (HCC), including advanced and metastatic forms. It specifically targets beta-catenin, a key component of the Wnt/beta-catenin signaling pathway, which is implicated in many human cancers such as HCC. The drug utilizes Alnylam’s advanced lipid nanoparticle (LNP) delivery technology called “reLNP,” designed to improve tolerability through enhanced biodegradability. ALN-BCAT is administered intravenously and is currently being evaluated in Phase 1 clinical trials both as monotherapy and in combination with pembrolizumab for patients with unresectable or metastatic HCC who have received at least one prior line of systemic therapy[2][5][6][7].
02
Targets
CTNNB1 (Beta-catenin)
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