Drug intelligence / Profile preview

siR-PTBP1

Development stage
Preclinical
Lead developer
Gifu University
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
01

Overview

siR-PTBP1 is a chemically modified small interfering RNA (siRNA) designed to target and silence the expression of Polypyrimidine tract-binding protein 1 (PTBP1). PTBP1 is a key regulator of alternative splicing that promotes the expression of the PKM2 isoform of pyruvate kinase, a hallmark of the Warburg effect (aerobic glycolysis) in cancer cells. By inhibiting PTBP1, siR-PTBP1 induces a metabolic shift from glycolysis toward oxidative phosphorylation by increasing the PKM1/PKM2 isoform ratio. This metabolic reprogramming leads to increased intracellular ATP production, induction of apoptosis (as indicated by elevated cleaved PARP levels), and significant inhibition of tumor cell proliferation. It is being developed as a metabolism-targeted nucleic acid therapeutic, specifically for the treatment of advanced gastric cancer.

Other names
PTBP1-targeted siRNAPTBP-1-targeted siRNAPTBP 1-targeted siRNAPTBP1 siRNAPTBP-1 siRNAPTBP 1 siRNA
02

Targets

PTBP1 (Polypyrimidine tract-binding protein 1)

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