Regio Zuid-Holland | Development of TW002 (AAV5-­-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis

Openbare samenvatting: .Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder characterized by the progressive loss of motor neurons leading to paralysis and eventually death. Currently, no cure exists for ALS and treatment only slightly prolongs survival of patients. Consequently, new drugs are in high demand. TW002 (AAV5-­-GDNF), gene therapy currently being developed by Treeway, has the potential to be an effective therapeutic for ALS. Glial cell line-­-derived neurotrophic factor (GDNF) plays an important role in maintenance of motor neuron health and GDNF may prevent the progressive loss of motor neurons, which is associated with the progression of ALS. Goal of this project is to determine the safety and efficacy of a single intrathecal treatment with TW002 in transgenic ALS rats. Successful completion of the TW002 preclinical study will be the first step in developing a gene therapy-­-based treatment for ALS. Treeway believes that TW002 has the potential to ameliorate and delay ALS associated symptoms, thus prolonging lifespan and improving the quality of life of ALS patients.