Several pioneering studies reported major advances in neurosciences and neurotherapeutics. Ionis Pharmaceuticals succeeded in pivotal trials for an antisense oligonucleotide treating Alexander disease, a rare neurological disorder, planning an FDA submission in 2026. Neurocrine Biosciences shared promising Phase 2 data for osavampator in major depressive disorder. Furthermore, innovative CAR-T and CAR NKT cell therapies are under evaluation for glioblastoma and autoimmune diseases, highlighting novel immunotherapeutic approaches in challenging neurodegenerative and psychiatric conditions.