AcuraStem is a patient-based biotechnology company pioneering how treatments are developed for neurodegenerative diseases — including sporadic ALS and FTD — using our proprietary, best-in-class, disease-modeling platform, iNeuroRx®, the gold standard for discovering novel, effective and broadly-acting treatments.
The main reason drugs fail in clinical trials is due to an over reliance on animal models, which leads to a poor understanding of the real human mechanisms driving disease. AcuraStem are leaders and inventors in the generation of individual patient models that accurately recapitulate neurodegenerative disease processes. This, what we call our patient-based approach, leads to more effective treatments, and treatments that can work broadly for patients, not limited to rare, genetically defined subtypes.
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Neurodegenerative Disease Areas
A recent study published in the journal Cell Stem Cell and co-authored by AcuraStem CEO Sam Alworth, M.S., MBA, and AcuraStem co-founder Justin Ichida, Ph.D., et al., shows that suppressing the gene encoding spliceosome-associated factor SYF2 may alleviate symptoms of Amyotrophic Lateral Sclerosis (ALS) and improve neuron survival in both sporadic and genetic forms of the disease.
AcuraStem announced today that CEO Sam Alworth, will present on the company’s latest advances in the development of a PIKFYVE antisense oligonucleotide (ASO) therapeutic for diverse forms of ALS and FTD at the 13th annual California ALS Research Summit
Dr. Wen-Hsuan Chang, AcuraStem’s Head of Target Validation to present at the 33rd International Symposium on ALS/MND
Dr. Wen-Hsuan Chang, AcuraStem’s Head of Target Validation to present at the 33nd International Symposium on ALS/MND being held virtually on December 6-9, 2022. Dr. Chang will detail AcuraStem’s development program leveraging its best-in-class iNeuroRx® platform, composed of patient-derived disease models that accurately recapitulate UNC13A pathology, to develop novel and potent UNC13A antisense oligonucleotide (ASO) drug candidates. UNC13A is one of the most significant genetic risk factors for ALS and FTD.