GeneCode, an Estonian private pharmaceutical development company, focused on the development of disease-modifying therapeutics to combat neurodegeneration, has developed a GDNF Mimetics platform that leverages the disease-modifying properties of Glial cell line – Derived Neurotrophic Factor GDNF to small molecules with drug-like properties. GeneCode has remarkably improved the pharmacokinetic properties of the identified hits, making them more active and effective, drug-like and more efficient to combat neurodegeneration in Parkinson´s disease (PD), which has been main development focus so far.

GDNF is a neurotrophic growth factor that that is considered to have a significant therapeutic potential also in Amyotrophic Lateral Sclerosis (ALS), Retinitis Pigmentosa (RP) and Inflammatory Bowel Disease (IBD), because of its neuroprotective / neuroregenerative properties on motor neorons, retinal neurons and its role in the maintenance of intestinal epithelial barrier. Preclinical studies have shown that GDNF has potential to slow or prevent the degeneration of motor neurons, alleviate motor function, and increase the lifespan in preclinical models of ALS. GDNF has also been shown to promote the survival of photoreceptor cells and play an important role in the maintenance of intestinal epithelium barrier.