- Huntington’s disease (HD) affects 5 in every 100,000 individuals, and combined with all other polyQ-related disorders, this number reaches 1 in 10,000 individuals.
- PolyQ diseases are caused by CAG repeat expansions inside the coding regions of specific genes, leading to an expanded and aberrant polyQ within the translated protein. The diseases are dominant, late onset, neurodegenerative, and no cure or even alleviating treatment is currently available for any of them.
- Since there are currently no available treatments, polyQ patients currently live only 15-20 years after disease onset (around the age of 40-50). Therefore, there is a high need for drug discovery and development that will improve their conditions and give patients a longer and healthier life.
A therapeutic target for Huntington’s disease (HD) and other polyQ-related diseases, which decreases the levels of effector molecules.
- First treatment for HD and other polyQ-related diseases.
- Possibility to start treatment before symptoms apparition.
- Target can also be used in other aggregate-relates diseases (e.g. Parkinson’s disease).
Since there is no therapy or available drug to treat Huntington’s disease (HD) patients nor any other polyQ-related disorder patient, our product will be the first to offer a therapeutic avenue. Thus, it will be sold regularly, with clear economic potential.
We are seeking collaboration with pharma companies with a focus on Huntington’s disease patients and other polyQ-related disorders. Moreover, our drug could also be beneficial for other aggregate-relates diseases (e.g. Parkinson’s disease).