BrainStorm Therapeutics, Inc.

BrainStorm Therapeutics is committed to transforming CNS drug discovery - including for the treatment of rare genetic disorders affecting the brain! We are developing an NSF-funded, NVIDIA-award-winning human brain organoid platform that integrates experimental and computational approaches to discover disease-modifying therapies for brain disorders. We engineer brain organoids with mutations known to drive disease and probe them with high-throughput screens to develop AI-driven digital organoid foundation models to identify and validate therapeutic targets and drug candidates that reverse disease progression. Our founding team successfully identified a therapeutic candidate for the rare genetic neurodevelopmental disorder, Rett syndrome, advancing to a Phase 2 clinical trial by screening patient-derived cortical brain organoids. Our first therapeutic programs are focused on rare genetic neurodevelopmental disorders and diseases caused by degeneration or dysfunction of midbrain dopamine neurons, including Parkinson’s disease, schizophrenia, and addiction. Our highly scalable platform can address other complex brain disorders and therapeutic areas.