Drug intelligence / Profile preview

USP22-IN-1

Development stage
Preclinical
Modality
Small Molecules
Administration
Intraperitoneal
01

Overview

USP22-IN-1 is a small molecule inhibitor of ubiquitin-specific protease 22 (USP22), a deubiquitinating enzyme that is a key component of the SAGA (Spt-Ada-Gcn5 acetyltransferase) complex. USP22 plays a critical role in regulating protein stability and gene transcription by removing ubiquitin chains from various substrates. In the context of hematological malignancies such as myelodysplastic syndromes (MDS), USP22 has been shown to stabilize nucleosome assembly protein 1-like 1 (NAP1L1), which in turn activates the PI3K/AKT signaling pathway to promote the clonal expansion of CD34+ hematopoietic stem/progenitor cells. USP22-IN-1 is primarily utilized as a pharmacological probe in preclinical research to investigate the USP22-NAP1L1 axis as a potential therapeutic vulnerability in myeloid disorders.

02

Targets

USP22 (Ubiquitin-specific peptidase 22)

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