Drug intelligence / Profile preview

ALN-4324

Development stage
Phase 2
Lead developer
Alnylam Pharmaceuticals
Modality
Small Molecules, 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
Administration
Intravenous
01

Overview

ALN-4324 is an investigational small interfering RNA (siRNA) therapeutic developed by Alnylam Pharmaceuticals. It is designed to target growth factor receptor-bound protein 14 (GRB14), a negative regulator of insulin signaling, with the goal of improving insulin sensitivity. The drug acts via RNA interference (RNAi) to silence GRB14 expression in relevant tissues. Its primary indications under investigation are type 2 diabetes mellitus and obesity. If successful, it could become the first new insulin sensitizer approved in over three decades and may offer a novel approach for patients with genetic or hormonal forms of obesity that are not adequately addressed by current therapies[1][3][7][9][10].

02

Targets

GRB14 (Growth factor receptor-bound protein 14)

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