GA-VAX

GA-VAX is a preclinical therapeutic vaccine being developed to target the toxic poly-glycine-alanine (poly-GA) dipeptide repeat proteins produced by C9orf72 hexanucleotide repeat expansions, the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The vaccine uses a peptide/carrier conjugate design in which optimized poly-GA peptide antigens are chemically linked to an immunogenic carrier to elicit high-titer anti-GA antibodies, thereby promoting neutralization and clearance of extracellular poly-GA aggregates, reduction of microglial activation and neuroinflammation, and attenuation of downstream TDP-43 pathology in C9orf72 models.[3][4][6][9] Developed through a collaboration between DZNE and Intravacc with EU EIC funding, GA-VAX is being advanced through GLP toxicology, safety pharmacology, and pivotal efficacy studies to support a first-in-human Phase 1b/2a trial in C9orf72 ALS and FTD patients.[3][6][9]

Other names

GA VAXGA poly-GA vaccinepoly-GA peptide/carrier conjugate vaccine