AcuraStem is a near-to-clinic, 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
AcuraStem's CEO, Sam Alworth, is set to present on the company’s latest advances in the development of a PIKFYVE antisense oligonucleotide (ASO) therapeutic for diverse forms of FTD (FTD) at the Alzheimer's Drug Discovery Foundation's (ADDF) Defeating Dementia Symposium on April 13.
BioSpace Highlights the Progress of AcuraStem’s Antisense Oligonucleotide Targeting PIKFYVE and a New Program Targeting SYF2 in Preclinical Studies
AcuraStem’s progress in advancing an antisense oligonucleotide (ASO) targeting PIKFYVE and development of therapeutic candidates targeting SYF2 in preclinical studies has been featured in a newly posted BioSpace article.
Fierce Biotech featured AcuraStem's pioneering work with PIKFYVE inhibitors in a recent article highlighting promising developments in treating both genetic and sporadic forms of ALS. The article discusses two recent studies led by Dr. Justin Ichida's lab at USC and the AcuraStem team that have identified drug candidates with potential to transform the disease course for a larger patient population beyond those with known genetic causes of ALS.