Led by experts in disease modeling, machine learning and drug development, as a company AcuraStem Inc is usefully understood as a patient-based drug discovery platform company developing novel drug programs for neurodegenerative diseases. Led and managed by experts in disease modeling, machine learning and drug development, the firms iNeuroRx®'s technology platform combines patient-derived disease models with human genetic data. Employing the latest scientific breakthroughs to address the most challenging - but tractable - of today's health problems to include amyotrophic lateral sclerosis (ALS). The firm's best-in-class technology platform consists of complex cellular models from ALS patient cells and sophisticated assays which permit AcuraStems scientists to model patient tissues in the lab. Using this ground-breaking technology, small molecule or gene targeting libraries canbe screened to discover the most efficacious therapeutic opportunities. AcuraStems iNeuroRx® platform is a breakthrough disease modeling platform, leveraging neurons derived from patients and advanced machine learning to discover therapeutic opportunities that rescue healthy motor neurons in those affected by neurodegenerative diseases, like ALS and FTD (frontotemporal dementia). AcuraStem has found unique therapeutic targets that work broadly for many types of ALS patients. Therefore, AcuraStem is developing PIKFYVE target therapeutics for ALS and FTD. Its most advanced drug candidate, AS-202, is in IND-enabling toxicity studies.
