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ISIS 421062
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intraperitoneal
01
Overview
ISIS 421062 (also known as PEPCK-M antisense oligonucleotide) is a second-generation 2'-O-methoxyethyl (2'-MOE) modified chimeric antisense oligonucleotide (ASO) with a phosphorothioate backbone. Developed by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals) in collaboration with researchers at Yale University School of Medicine, ISIS 421062 selectively targets and silences the mitochondrial isoform of phosphoenolpyruvate carboxykinase (PEPCK-M, encoded by the PCK2 gene). In preclinical rodent models of type 2 diabetes, treatment with ISIS 421062 significantly reduced hepatic PEPCK-M expression, leading to lowered plasma glucose, insulin, and triglyceride levels, as well as reduced white adipose tissue and depleted hepatic glycogen without causing hypoglycemia during fasting. This makes PEPCK-M an attractive therapeutic target for the treatment of type 2 diabetes and related metabolic disorders.
- Other names
- PCK2 ASOPCK-2 ASOPCK 2 ASOPCK2-ASOPCK-2-ASOPCK 2-ASOPCK2ASOPCK-2ASOPCK 2ASOPEPCK-M antisense oligonucleotide
02
Targets
PEPCK (Phosphoenolpyruvate carboxykinase 1)
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