Drug intelligence / Profile preview

SJ001011447

Development stage
Preclinical
Lead developer
St. Jude Children's Research Hospital
Modality
PROTACs (E3 ligase recruitment) → Targeted Protein Degraders (TPDs) → Small Molecules, Bivalent/Multivalent Binders → Multivalent & Scaffold-Based Small Molecules → Small Molecules
Administration
Oral, Intravenous
01

Overview

SJ001011447 is a proteolysis-targeting chimera (PROTAC) designed to induce the degradation of Lymphocyte-specific protein tyrosine kinase (Lck), a key therapeutic vulnerability identified in a significant subset of T-cell acute lymphoblastic leukemia (T-ALL). Developed by researchers at St. Jude Children's Research Hospital, the molecule functions by recruiting Lck to the Cereblon (CRBN) E3 ubiquitin ligase, which facilitates the ubiquitination and subsequent proteasomal degradation of the kinase. Unlike traditional small-molecule inhibitors like dasatinib, which provide transient inhibition, SJ001011447 achieves sustained suppression of Lck signaling. In preclinical studies, it has demonstrated potent cytotoxicity and prolonged growth inhibition in T-ALL cell lines and patient-derived samples, making it a promising candidate for the treatment of relapsed or aggressive T-ALL.

02

Targets

CRBN (Cereblon)LCK (Proto-oncogene tyrosine-protein kinase Lck)

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