Drug intelligence / Profile preview

RO7623066 + carboplatin

Development stage
Phase 1
Lead developer
Roche
Modality
Classical Binding Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
Administration
Oral, Intravenous
01

Overview

RO7623066 + carboplatin is an investigational combination therapy comprising the small molecule USP1 inhibitor RO7623066 and the chemotherapeutic agent carboplatin. RO7623066 is an oral, selective, first-in-class inhibitor of the deubiquitinating enzyme ubiquitin-specific peptidase 1 (USP1), which regulates the translesion synthesis and Fanconi Anemia DNA repair pathways. Carboplatin is a platinum-based chemotherapeutic causing DNA crosslinking and inhibition of DNA replication and transcription. The combination is being explored in patients with advanced solid tumors, particularly those with homologous recombination repair deficiencies, in early-phase clinical trials to assess safety, tolerability, and preliminary anticancer activity. RO7623066 was originally developed by KSQ Therapeutics and subsequently licensed to Roche[1][2][3][4][5][6][8][9].

02

Targets

DNAUSP1 (Ubiquitin-specific protease 1)

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