BrainStorm Therapeutics, Inc.

Our Mission: BrainStorm Therapeutics aims to discover and develop breakthrough medicines for complex neurological disorders using a unique "human-powered" CNS drug discovery platform. We recognize the significant challenges faced in this field, where traditional approaches have fallen short despite substantial investments.Our Approach: At the core of our approach is a "human-first" perspective. We employ patient-derived organoid models of brain diseases, cutting-edge data analytics, and the computational tools of network medicine to expedite the discovery of novel therapeutics. Our primary focus is on halting, preventing, or reversing critical molecular events that lead to synaptic network dysfunction and neurodegeneration.Key Advantages:• Early-stage identification of efficacious therapeutic candidates in a human disease model using clinically translatable disease endpoints.• Successful previous work on rare CNS genetic disorders (Rett syndrome and CDKL5 deficiency disorder) underscores the potential of our approach.• Precision medicine targeting genetically validated disease drivers in familial Parkinson's disease (PD) and sporadic PD.• Exploration of GBA1 gene mutations as a common genetic risk factor for PD.• Utilization of midbrain organoids with clinical biomarkers for high-throughput screening.• Aiming to discover disease-modifying therapeutics for dopamine neuron degeneration in PD.Our Vision: BrainStorm Therapeutics is not just another biotech company. We are committed to reducing the cost, de-risking clinical translation, and accelerating the discovery of disease-modifying therapies for PD. Our platform's scalability opens the door to applications in various neurological disorders and therapeutic areas.