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liposomal ATM siRNA
- Development stage
- Preclinical
- Lead developer
- Methodist Hospital Research Institute
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
liposomal ATM siRNA is a liposomal formulation of small interfering RNA (siRNA) targeting ataxia-telangiectasia mutated (ATM) kinase, a key regulator of the DNA damage response (DDR) pathway. Developed by the Houston Methodist Research Institute (formerly The Methodist Hospital Research Institute), the therapeutic candidate is designed to knock down ATM expression in tumor tissues, thereby impairing DNA double-strand break repair and cell cycle checkpoints. This gene-silencing approach aims to inhibit tumor growth and sensitize cancer cells, particularly triple-negative breast cancer (TNBC), to chemotherapy (such as doxorubicin) and immune checkpoint blockade therapies without the systemic toxicity often associated with small-molecule ATM inhibitors. The formulation can be delivered directly or loaded into a porous silicon-based multistage vector (MSV) delivery system (referred to as MSV/ATM) to achieve sustained release.
- Other names
- DOPC-siATMDOPC/siATMliposomal ATM small interfering RNAliposomal-encapsulated ATM siRNAnanoliposomal ATM siRNA
02
Targets
ATM (Ataxia telangiectasia mutated protein)
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