pGDNF DNA nanoparticles

pGDNF DNA nanoparticles are a non-viral gene therapy modality developed by Copernicus Therapeutics for the treatment of Parkinson's disease. The drug consists of a plasmid DNA encoding human glial cell line-derived neurotrophic factor (GDNF), which is compacted with polyethylene glycol (PEG)-substituted lysine 30-mer peptides into rod-like nanoparticles ranging from 8 to 11 nm in diameter. This synthetic delivery system is designed to protect the DNA from degradation and facilitate its transit across the nuclear membrane pore of postmitotic cells, such as neurons. Once delivered to the brain—either via direct intracerebral injection or non-invasive intranasal delivery—the nanoparticles transfect cells in the striatum and substantia nigra, leading to the overexpression of GDNF. This neurotrophic factor provides essential support and neuroprotection to dopaminergic neurons, aiming to slow disease progression and improve motor function.

Other names

Compacted pGDNF DNA nanoparticlesCompacted DNA nanoparticlespGDNF nanoparticles