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ALN-BCAT + pembrolizumab
- Development stage
- Unknown
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Monoclonal Antibodies → Antibody-Based 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 + pembrolizumab is an investigational combination therapy for advanced or metastatic hepatocellular carcinoma (HCC). ALN-BCAT is a small interfering RNA (siRNA) therapeutic designed to reduce the expression of Catenin beta-1 (β-catenin, encoded by CTNNB1), an oncogenic driver frequently mutated in HCC. ALN-BCAT is formulated using Alnylam's reLNP lipid nanoparticle delivery technology for improved tolerability and efficient delivery to liver cells. Pembrolizumab is an FDA-approved monoclonal antibody that blocks the PD-1 immune checkpoint pathway, enhancing T-cell mediated antitumor responses. The combination aims to inhibit tumor growth by suppressing β-catenin-driven oncogenic signaling and simultaneously activating antitumor immune responses through PD-1 inhibition. The combination is currently being evaluated in a Phase 1 clinical trial for its safety, tolerability, and preliminary efficacy in patients with advanced or metastatic HCC with WNT pathway–activating mutations[1][3][4][5][6][7][9].
- Other names
- ALN-BCATpembrolizumabKeytruda
02
Targets
PDCD1 (Programmed cell death protein 1 receptor)CTNNB1 (Beta-catenin)
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