Drug intelligence / Profile preview

kt-3283

Development stage
Preclinical
Lead developer
Rakovina Therapeutics
Modality
Small Molecules
01

Overview

KT-3283 is a novel, bifunctional small molecule drug candidate developed by Rakovina Therapeutics. It is designed to simultaneously inhibit both poly(ADP-ribose) polymerase 1/2 (PARP1/2) and histone deacetylase (HDAC) enzymes. This dual mechanism targets DNA damage response pathways in cancer cells, particularly those with impaired homologous recombination repair such as Ewing sarcoma. Preclinical studies have shown that KT-3283 exhibits significantly greater cytotoxicity against Ewing sarcoma models than single-agent PARP or HDAC inhibitors, inducing strong S-phase and G2/M cell cycle arrest and elevated DNA damage at nanomolar concentrations. The compound has demonstrated the ability to prevent metastatic growth in animal models of Ewing sarcoma and may offer a new therapeutic strategy for treatment-resistant cancers by combining two mechanisms into a single molecule[2][5][7].

Other names
KT-3283KT3283KT 3283kt-3000 series (series name, not specific to this compound)
02

Targets

HDAC (HDAC family)PARP2 (Poly (adp-ribose) polymerase 2)

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