Target intelligence / Profile preview

Leptin messenger RNA (LEP mRNA)

Target
LEP mRNA
Molecular classification
Messenger RNA
01

Overview

Leptin messenger RNA (LEP mRNA) is the ribonucleic acid transcript of the LEP gene, which encodes the hormone leptin, a primary regulator of energy balance and appetite. Produced mainly by adipose tissue, leptin signals the hypothalamus to inhibit hunger and stimulate energy expenditure, thereby maintaining stable body weight. In rare conditions such as congenital leptin deficiency, the absence of functional leptin leads to severe hyperphagia and early-onset obesity, which can be addressed by mRNA replacement therapies like Moderna's mRNA-1881 that deliver synthetic mRNA to restore protein production. Additionally, the broader leptin pathway includes the mRNA of the leptin receptor (LEPR), which is a target for RNA interference (RNAi) strategies like CCT-417 aimed at modulating receptor activity in metabolic diseases or certain cancers. While mRNA-based approaches offer a novel way to manage leptin-related disorders, therapeutic challenges include ensuring efficient delivery to target tissues and avoiding the development of leptin resistance or immune reactions to the delivery platform.

Other names
Leptin mRNAObesity factor mRNAOb mRNALEP transcriptLeptin pathway mRNAs
02

Mechanism of action

mRNA replacement therapy providing a template for the synthesis of functional leptin protein, or RNA interference (RNAi) to modulate receptor expression.

03

Biological functions

Energy homeostasisAppetite regulationMetabolismNeuroendocrine signalingImmune response modulation
04

Disease associations

Congenital leptin deficiencyLipodystrophyObesityHypothalamic amenorrheaMetabolic syndrome
05

Safety considerations

Immunogenicity of the lipid nanoparticle (LNP) delivery systemPotential for leptin resistance with chronic overexpressionInjection site reactionsOff-target effects of the delivery vehicleInflammatory response to synthetic mRNA
06

Interacting drugs

mRNA-1881

1 more in the full profile.

07

Biomarkers

Serum leptin levelsBody weightFood intakeEnergy expenditureInsulin sensitivity

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