Parkinson’s disease

According to a WHO report from 2022, disability and death due to Parkinson’s disease (PD) are rising faster than for any other neurological disorder – having doubled over the past 25 years. PD affects around 0.3% of the population, with prevalence increasing with age and reaching about 1% among people over 60. As a result, many countries are investing heavily in research and development to improve care, services, and treatment options for patients and communities.

Current PD medications provide only symptomatic relief and do not alter the course of the disease. Over time, they become less effective and can cause significant side effects. Managing Parkinson’s is also costly, and expenses are expected to grow as populations age. New disease-modifying therapies could improve quality of life for millions of people with PD and their caregivers, while also reducing the economic burden on healthcare systems and society.

GeneCode is addressing this urgent need by developing a novel small-molecule therapy for Parkinson’s disease that efficiently reaches both the brain and spinal cord. RET agonists mimic the effects of GDNF (glial cell line-derived neurotrophic factor), a naturally occurring growth factor that protects and restores dopamine-producing neurons, by binding to the same GFRα1-RET receptor complex. In doing so, they may alleviate both motor and non-motor symptoms, protect and regenerate damaged dopamine neurons, and potentially increase dopamine levels in brain tissue. Unlike GDNF – which cannot cross the blood-brain barrier and requires invasive administration – our RET agonists are small molecules that penetrate the brain, activate the same receptor system, and trigger identical survival and regenerative pathways. This promotes long-term neuron survival and restores function in affected dopaminergic pathways. Once fully developed, this therapy is expected to be delivered orally on a weekly – or even less frequent – basis.