Drug intelligence / Profile preview

Vincere Biosciences USP30 inhibitor

Development stage
Preclinical
Lead developer
Vincere Biosciences
Modality
Small Molecules
Administration
Oral
01

Overview

Vincere Biosciences is developing a small-molecule inhibitor of USP30 (ubiquitin-specific protease 30) as a potentially first-in-class therapeutic for Parkinson's disease and other age-related conditions, including cardiorenal diseases. USP30 is a deubiquitinating enzyme located on the outer mitochondrial membrane that acts as a negative regulator of mitophagy, the selective process of removing damaged mitochondria. By inhibiting USP30, the drug aims to enhance mitophagy and restore mitochondrial quality control, which is often impaired in neurodegenerative and metabolic disorders. The lead candidate is an oral, brain-penetrant compound currently in IND-enabling studies, with clinical trial initiation planned for 2026. The program has received over $8 million in grant support from organizations such as the Michael J. Fox Foundation and the National Institutes of Health.

Other names
USP30 inhibitor (Vincere Biosciences)
02

Targets

USP30 (Ubiquitin-specific peptidase 30)

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