Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease caused by protein aggregation in the motor neurons of the brain and spinal cord that leads to respiratory failure within 2-5 years. The best available drugs extend life by ~3 months. ALS affects about 1 in 500 people, mostly for unknown reasons, but 5-10% of cases in Caucasians are caused by a mutation in the C9orf72 gene. We have shown that this mutation leads to the expression of large aggregating poly-glycine-alanine (poly-GA), which triggers downstream pathology. We have developed a poly-GA peptide vaccine that reduces aggregates and largely prevents motor deficits in a mouse model. Our vaccine reduces neuronal damage to a similar extent when administered in already symptomatic mice. Since regular lifelong vaccination is required to maintain sufficient antibody levels, GA-VAX represents an attractive business case in the orphan disease space with ~2500 prevalent C9orf72 ALS cases in the US, DE, IT, FR, ES, UK. The ~9000 mutation carriers at risk of developing the disease within 10 years could benefit even more from our approach. Our highly complementary industry/academia team has all the expertise and resources to advance this promising treatment approach towards clinical evaluation. Intravacc provides the manufacturing and clinical development expertise for peptide/carrier conjugate vaccines, while DZNE provides in-depth knowledge of disease pathology and all necessary model systems and assays. The project includes antigen optimization, manufacturing and preclinical evaluation according to EMA and FDA regulatory requirements. This will allow us to prepare a clinical trial application in C9orf72 ALS patients. In addition, we will use this data package to raise capital for the Phase 1 trial from a patient organization or investor to further de-risk or partner with a larger pharmaceutical company to bring GA-VAX to market.