We are transforming drug discovery by utilizing patient data at the very beginning of the development process and during every stage of our proprietary discovery approach
AcuraStem was formed in 2016 to employ the latest scientific breakthroughs to address the most challenging health problems of our time. Comprised of a team of PhDs, professors, tech entrepreneurs, and successful drug company veterans, AcuraStem harnesses the latest scientific breakthroughs and produces the best-in-class technology platform, iNeuroRx®, which consists of complex cellular models derived from ALS patients, and sophisticated assays which permit our scientists to model patient tissues in the laboratory and identify new therapeutic opportunities using these tissues.
AcuraStem’s iNeuroRx® platform has enabled the discovery of several broadly-acting therapeutic targets that may work in sporadic ALS, a high unmet need population, and in other neurodegenerative indications including Alzheimer’s disease. To date, all of the drug candidates discovered or validated on iNeuroRx® which have advanced into animal models of ALS and FTD, have done so successfully.
One of the most promising targets profiled on iNeuroRx® is the kinase PIKFYVE. The disease-modifying potential of PIKFYVE inhibition for ALS and other neurodegenerative diseases was discovered by AcuraStem’s co-founder and close collaborator, Dr. Justin Ichida. His landmark study appeared in Nature Medicine in 2018 (Shi Y et al Nature Med 2018) and illustrated how reducing PIKFYVE levels in ALS patient motor neurons helps to prevent neurodegeneration, and could translate to improved motor function and survival for ALS patients. AcuraStem is developing PIKFYVE-targeting therapeutics, the best of which will enter the clinic for ALS in 2021.