Patient-Based Therapeutics

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.

Patient Focused

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rp4.jpgThe 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

Latest News

October 27, 2026

PASADENA, Calif., October 27, 2026 ⎯ AcuraStem, a biotechnology company advancing treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD) and other neurodegenerative diseases, today announced it has been awarded a Hoffman ALS Clinical Trial Award from the ALS Association, the largest ALS organization in the world. The two-year, $1 million award supports AcuraStem’s UNC13A program and the biomarker work to support the first-in-human Phase 1 trial of the program’s lead candidate, AS-241.

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September 14, 2026

AcuraStem was recently featured in Genetic Engineering & Biotechnology News (GEN) in an article titled "Rewriting Disease: Oligonucleotides Take Aim at the Untreatable." The piece examines rapid advancements in nucleic-acid-based drugs and explores how the biotechnology sector is increasingly turning to antisense oligonucleotides (ASOs) to address severe neurodegenerative conditions.

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April 28, 2026

PASADENA, Calif., April 28, 2026– AcuraStem, a biotechnology company advancing treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, announced today that the California Institute for Regenerative Medicine (CIRM) has awarded the company $7.5 million in grant funding to support the advancement of its lead clinical candidate, AS-241, toward first‑in‑human testing.

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