GEM is a company dedicated to the research and development of new compounds based on small molecules for the treatment of rare diseases.
Currently, the company is focusing on the study of new compounds for the treatment of Huntington's disease.
New compounds for the treatment of Huntington's disease
Research groups from the IPNA chemistry department collaborate with GEM in the identification of new ‘lead’ compounds (candidates for development) with potential activity in the treatment of other rare diseases. The first efforts have focused on Huntington's disease, an inherited pathology that causes the breakdown of particular nerve cells in the brain. People are born with the defective gene but symptoms do not appear until they are in their 30s or 40s. There is currently no cure, the medicines available can help control some symptoms but cannot delay or stop the disease.