Drug intelligence / Profile preview

QC-01-175

Development stage
Preclinical
Modality
PROTACs (E3 ligase recruitment) → Targeted Protein Degraders (TPDs) → Small Molecules, Bivalent/Multivalent Binders → Multivalent & Scaffold-Based Small Molecules → Small Molecules
01

Overview

QC-01-175 is a heterobifunctional proteolysis-targeting chimera (PROTAC) designed to selectively degrade aberrant tau protein. Developed by researchers at the Dana-Farber Cancer Institute and Massachusetts General Hospital, the molecule is constructed by linking the tau-binding positron emission tomography (PET) tracer T807 to pomalidomide, an E3 ligase recruiting ligand. This design allows QC-01-175 to simultaneously bind pathological tau and Cereblon (CRBN), promoting the ubiquitination and subsequent proteasomal degradation of tau. QC-01-175 has demonstrated efficacy in clearing mutant tau (such as A152T and P301L) in patient-derived neuronal models of frontotemporal dementia (FTD) and rescuing mitochondrial deficits and oxidative stress in models of amyotrophic lateral sclerosis (ALS).

02

Targets

CRBN (Cereblon)MAPT (Microtubule-associated protein tau)

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