Drug intelligence / Profile preview

LDR1259

Development stage
Phase 1
Lead developer
Leaderna Therapeutics
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
Administration
Subcutaneous
01

Overview

LDR1259 is a small interfering RNA (siRNA) therapeutic candidate developed by Chengdu Xianyan Biotechnology for the treatment of hyperlipidemia and elevated lipoprotein(a) [Lp(a)] levels. The drug is designed to target and degrade the mRNA of the *LPA* gene in the liver, which encodes apolipoprotein(a), a critical component of the Lp(a) particle. By silencing the production of apolipoprotein(a), LDR1259 aims to significantly lower circulating Lp(a) concentrations, a known independent risk factor for atherosclerotic cardiovascular disease (ASCVD) and calcific aortic valve stenosis. LDR1259 likely utilizes a GalNAc-conjugation platform to ensure targeted delivery to hepatocytes. It is currently undergoing Phase 1 clinical evaluation in China to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in individuals with elevated Lp(a).

02

Targets

ASGPR (Asialoglycoprotein Receptor 1)LPA (Lysophosphatidic acid)PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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