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ITU512

Development stage: Phase 2
Lead developer: Novartis
Modality: Molecular Glues → Targeted Protein Degraders (TPDs) → Small Molecules
Administration: Oral

Overview

ITU512 is an investigational, oral, low molecular weight small molecule drug being developed for the treatment of sickle cell disease (SCD). It acts as a molecular glue degrader targeting the WIZ protein, which leads to increased production of fetal hemoglobin (HbF). By inducing HbF, ITU512 aims to ameliorate the clinical manifestations of SCD. The drug is currently in Phase I/II clinical trials evaluating its safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy in both healthy participants and patients with SCD. The developer and sponsor is Novartis Pharmaceuticals[1][3][4][5].

Targets

CRBN (Cereblon)WIZ (WIZ zinc finger protein)

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