Drug intelligence / Profile preview

A31

Development stage
Preclinical
Modality
Small Molecules
Administration
Oral
01

Overview

A31 is a novel small molecule tyrosine kinase inhibitor (TKI) designed to target and inhibit the constitutive phosphorylation of fibroblast growth factor receptor 3 (FGFR3). This inhibition aims to restore normal chondrocyte differentiation and promote bone growth, addressing the underlying pathology of conditions caused by activating FGFR3 mutations, such as achondroplasia and other skeletal dysplasias. The drug has shown promise in restoring the size of embryonic dwarf femurs and normalizing cell cycle regulators in Fgfr3(Y367C/+) dwarf mice, suggesting its potential for treating FGFR3-related chondrodysplasias.

02

Targets

FGFR3 (Fibroblast growth factor receptor 3)

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