Target intelligence / Profile preview

Caspase-2 messenger RNA (CASP2 mRNA) (CASP2 mRNA)

Target
CASP2 mRNA
Molecular classification
Nucleic acid, Messenger RNA
01

Overview

Caspase-2 messenger RNA (CASP2 mRNA) is the transcript responsible for the synthesis of Caspase-2, a cysteine-aspartic acid protease that functions as an initiator of apoptosis in response to cellular stress, such as DNA damage or endoplasmic reticulum stress [1][2]. As a therapeutic target, CASP2 mRNA is primarily addressed using RNA interference (RNAi) technologies, such as small interfering RNAs (siRNAs), to prevent the production of the Caspase-2 protein [3]. By silencing this mRNA, researchers aim to inhibit the apoptotic pathways that lead to cell death in conditions like acute kidney injury (AKI) and delayed graft function following kidney transplantation [4]. The most prominent drug candidate targeting this molecule is Teprasiran (QPI-1002), which was designed to provide temporary suppression of Caspase-2 to protect renal cells during periods of high stress [5]. Beyond renal applications, targeting CASP2 mRNA has been explored for neuroprotection in glaucoma and other neurodegenerative disorders where Caspase-2-mediated cell death is a contributing factor [6]. This target represents a shift from traditional small-molecule inhibition of enzymes to the upstream prevention of protein synthesis using nucleic acid-based drugs.

Other names
CASP2 transcriptICH-1 mRNANEDD2 mRNACaspase-2 mRNA
02

Mechanism of action

RNA interference (RNAi) mediated degradation of the mRNA transcript to inhibit Caspase-2 protein synthesis.

03

Biological functions

ApoptosisDNA damage responseCell death regulationProtein translation
04

Disease associations

Acute kidney injuryDelayed graft functionGlaucomaNon-alcoholic steatohepatitisNeurodegenerative disease
05

Safety considerations

Off-target gene silencingInnate immune activation by double-stranded RNATissue-specific delivery challenges
06

Interacting drugs

Teprasiran

2 more in the full profile.

07

Biomarkers

Caspase-2 protein levelsSerum creatinineUrinary kidney injury biomarkersGlomerular filtration rate

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