BrainStorm Therapeutics

BrainStorm Therapeutics is a precision neuroscience company developing disease-modifying therapies for rare and neurodegenerative brain disorders. We integrate patient-derived brain organoids with AI to identify and validate human-relevant disease mechanisms and therapeutic targets. Our lead program in CDKL5 deficiency disorder targets a novel, first-in-class pathway and is advancing through hit-to-lead optimization. We have demonstrated regulatory validation with an IND-cleared Rett syndrome repurposing program derived from organoid data. With strong reproducibility, proprietary datasets, and early partnership traction, BrainStorm combines a scalable discovery platform with a growing pipeline of de-risked assets, enabling faster, more predictive CNS drug development and compelling partnership opportunities.