Advancing rare disease drug discovery using consistent, defined human neurons

This case study describes how the To Cure A Rose Foundation used bit.bio’s ioGABAergic human neurons to accelerate preclinical drug discovery for an ultra‑rare neurodevelopmental disorder caused by mutations in HNRNPH2. Discover how by leveraging consistent, scalable, iPSC‑derived neuronal models, the team was able to rapidly screen antisense oligonucleotide (ASO) candidates and progress promising therapies toward clinical development.