Developing a novel Parkinson's disease drug using small molecule mimetics of Glial cell line-Derived Neurotrophic Factor (GDNF)

GeneCode is developing a novel anti-PD drug that contains a central nervous system penetrant compound imitating GDNF peptide pathway activation that alleviates both motor and non-motor symptoms in PD patients, protecting and restoring lesioned dopamine neurons as well as potentially increasing dopamine levels in brain tissue.

GeneCodes unique solution uses small-molecule compounds that penetrate the blood-brain barrier, target the GFL receptor complex (containing GDNF protein) and mimic GFL (GNDF family of ligands) biological effects in neurons, promoting survival in the affected dopaminergic neuronal pathways. Once fully developed, the PD drug would be administered weekly or less frequently as an oral treatment.

Considering the importance of GDNF family ligands and/or RET-signalling pathways for other groups of cells, applications of compounds developed by GeneCode are not limited to PD, but can be applied to cure other medical conditions such as neuropathic pain, addiction, ALS etc.