Drug intelligence / Profile preview

DNA-PK inhibitor

Development stage
Unknown
Lead developer
AstraZeneca
Modality
Small Molecules
Administration
Oral
01

Overview

**DNA-PK** refers to a small molecule inhibitor program targeting the **DNA-dependent protein kinase (DNA-PK)**, a key enzyme in the **non-homologous end joining (NHEJ)** pathway of DNA double-strand break repair. Developed by **Cancer Research Horizons** (the commercial arm of Cancer Research UK) in collaboration with **Newcastle University**, this asset is designed to act as a chemo- and radiosensitizer. By inhibiting DNA-PK, the drug prevents cancer cells from repairing lethal DNA lesions induced by ionizing radiation or genotoxic chemotherapies (such as topoisomerase inhibitors), thereby enhancing their anti-tumor efficacy. The program is currently in **Phase 1** clinical development for multiple cancer indications and is listed as an unpartnered asset in the Cancer Research Horizons pipeline, indicating its availability for licensing or co-development. The research at Newcastle University has also contributed to the discovery of other clinical-stage DNA damage response (DDR) inhibitors, including the dual ATM/DNA-PK inhibitor XRD-0394.

Other names
DNA-PKDNA-dependent protein kinase inhibitor
02

Targets

ATR (ATR serine/threonine kinase)ATM (Ataxia telangiectasia mutated protein)PI3K (Phosphoinositide-3-kinase regulatory subunit 6)DNA-PK (DNA-dependent protein kinase)mTOR (Mammalian target of rapamycin kinase)

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