Parkinson’s disease

Genecode Neurorestorative Poster 2017

 

• The prevalence of PD is 0,3% in whole population, 1% in people of 60 year old and up to 4% in people of 80 years of age and above
• PD significantly impairs the quality of life of affected individuals and in addition to motor symptoms patients often experience depression, anxiety, gastrointestinal problems, sexual disfunction, sleep and emotional problems as well as dementia during the later stages of disease
• No cure available, existing treatments are symptomatic (and mostly based on dopamine replacement), have side-effects and complicated by drug-tolerance development
• Progressive, meaning that the condition of the patients is getting worse over time
  The number of cases will increase in the future due to the general aging of the population

Market

• Global market sales of PD therapies reached $3.56 billion in 2012 and are expected to increase up to $5.26 billion by 2022
• Annual European costs of PD management are $13.9 billion
• Transition of the patient to residential care increases the cost of PD management by 500 times

Goal

Development of disease-modifying treatment for PD management. We aim to develop orally administred treatment restoring damaged dopaminergic neurons in the brain thus eliminating the cause of PD

Scientific background

• Neurotrophic factors are proteins that support and restore neurons
• Glial cell line-derived neurotrophic factor (GDNF) family ligands (GFLs) support and restore multiple neuronal populations including sensory neurons
• GFLs include 4 proteins: GDNF, NRTN, ARTN, PSPN
• GFLs signal via receptor complex consisting of receptor tyrosine kinase RET and a co-receptor selective for each GFL
• GDNF and NRTN alleviate PD symptoms in experimental animals and restore damaged DA neurons
• Several clinical trials showed clinical benefits of intracranially delivered GDNF and NRTN to certain populations of patients
• Administration of GDNF and NRTN to patients requires complicated stereotactic surgery

GFLs in threapy

GFLs transition to clinic is complicated:

• GFLs are poor drugs, they do not penetrate tissue barriers, their biodistribution and bioavailability is low
• Production, storage and handling of GFLs are expensive and not simple

GFL mimetics (GFLM)

Small molecules with similar biological activity to GFL, but imporved pharmacological properties such as:

• suitable PK/PD
• good bioavailability and biodistribution
• easy to produce and store

Might progress to clinic easier

GeneCode platform technologies patent

US Patent No.: US 8,901, 129 B2, Date of Patent Dec.2, 2014, European Patent No EP2509953, March 30, 2016
“METHODS OF FACILITATING NEURAL CELL SURVIVAL USING GDNF FAMILY LIGAND (GFL) MIMETICS OR RET SIGNALING PATHWAY ACTIVATORS”

The diseases, targeted by this GENECODE Patent protected inventions are listed in the background information of the Patent, section (0014):

[0014] The disorders targeted by the present invention include Alzheimer’s disease, Huntington’s disease, amyotrophic lateral sclerosis, Rett syndrome, epilepsy, Parkinson’s disease, spinal cord injury, stroke, hypoxia, ischemia, brain injury, diabetic neuropathy, peripheral neuropathy, nerve transplantation complication, motor neuron disease, multiple sclerosis, HIV, dementia, peripheral nerve injury, hearing loss, depression, obesity, metabolic syndrome, pain, cancer, and other contitions invlolving degeneration or dysfunction of cells expressing GFR α/RET.