About

GeneCode, an Estonian preclinical-stage pharmaceutical company, is developing small-molecule drugs that bind and activate (Glial cell line-Derived Neurotrophic Factor) receptors for the treatment of Parkinson’s disease (PD) and other neurodegenerative conditions.

What is GDNF?

GDNF is a naturally occurring protein that supports and protects dopamine-producing neurons—the very cells that are degenerating and lost in Parkinson’s disease. GDNF is known to slow or even stop disease progression. However, because proteins like GDNF cannot cross through the blood-brain barrier, they must be delivered directly into the brain. This invasive approach has limited clinical application and yielded inconsistent results in studies. GDNF binds to its co-receptor, GFRα1, and together they recruit the RET receptor tyrosine kinase. This activates potent survival pathways that support neuronal health, enhance dopamine synthesis and transmission, promote the regrowth and maintenance of nerve terminals, and strengthen synaptic plasticity.

Our approach

GeneCode has solved this challenge by designing small molecules that activate GNDF receptors and mimic the biological effects of GDNF. Unlike proteins, these compounds can cross through the blood–brain barrier and act both in the brain and throughout the body. Our lead molecules are more potent than natural GDNF and have already shown neuroprotective effects in both animal models of Parkinson’s disease and in laboratory models using human dopamine neurons.

Why it matters?

All currently available treatments for Parkinson’s only address symptoms – they do not alter the course of the disease. GeneCode’s approach aims to deliver the first systemically available, disease-modifying therapy for patients in the early to moderate stages of PD, targeting both motor and non-motor symptoms. This could provide hope for a large population of people living with Parkinson’s.

Beyond Parkinson’s

GDNF receptors are not only found in the brain but also in peripheral nerves, the kidney, the digestive system, reproductive organs, and other tissues. Because of this, GeneCode is also exploring the potential of GDNF receptors agonist in other serious diseases, including:

• ALS (Amyotrophic Lateral Sclerosis) – severe progressive neurodegenerative disease

• Inflammatory Bowel Disease (IBD)

• Retinitis Pigmentosa (RP), a degenerative eye disease.