Drug intelligence / Profile preview

remodelin

Development stage
Preclinical
Lead developer
University of Cambridge
Modality
Small Molecules
Administration
Oral
01

Overview

Remodelin is a small molecule research compound that was originally identified as an inhibitor of N-acetyltransferase 10 (NAT10), a multi-functional enzyme involved in RNA acetylation (specifically N4-acetylcytidine or ac4C), ribosome biogenesis, and microtubule organization. It was discovered through a phenotypic screen for compounds that could correct the nuclear morphology defects characteristic of Hutchinson-Gilford Progeria Syndrome (HGPS). Beyond its potential in premature aging, Remodelin has been investigated in preclinical models for various cancers, including hepatocellular carcinoma and prostate cancer, where it appears to suppress cell growth and overcome resistance to certain therapies. However, its status as a specific NAT10 inhibitor has been challenged by recent studies suggesting it may be a cryptic assay interference compound, leading to ongoing research into its true molecular targets. It is currently available only as a laboratory tool and has not entered clinical development.

Other names
4-(2-(cyclopentylamino)-2-oxoacetyl)benzonitrile949912-58-71622921-15-6
02

Targets

NAT10 (N-acetyltransferase 10)

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