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Au-NH2
- Development stage
- Preclinical
- Lead developer
- Ulm University
- Modality
- Nanoparticles → Drug Delivery Systems
01
Overview
Au-NH2 is an amino-functionalized nanoparticle featuring an inert metallic gold core, developed as a potential therapeutic for acute myeloid leukemia (AML). It demonstrates selective cytotoxicity against AML cells while sparing healthy peripheral blood mononuclear cells (PBMCs) and macrophages. The drug's mechanism of action involves the induction of mitochondrial dysfunction through the inhibition of complexes I, III, and IV of the electron transport chain, leading to increased mitochondrial superoxide levels and loss of mitochondrial membrane potential. This triggers the intrinsic apoptotic pathway via BID cleavage, cytochrome c release, and caspase 3 activation. Additionally, Au-NH2 treatment results in lysosomal rupture and a rapid decrease in mTOR pathway activity, offering a potential nanotherapeutic approach for AML regardless of the patient's cytogenetic profile.
- Other names
- amino-functionalized gold nanoparticlesamino-functionalized nanoparticles with an inert metallic gold core
02
Targets
BID (BH3 interacting-domain death agonist)CASP3 (Caspase-3)COX7C (Cytochrome c oxidase subunit 7C, mitochondrial)mTOR (Mammalian target of rapamycin kinase)ND1 (Mitochondrial electron transport chain complex I)bc1 complex (Cytochrome bc1 complex (plasmodium))
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