AcuraStem

AcuraStem AcuraStem is a near-to-clinic, patient-based therapeutics company advancing treatments for ALS, FTD and additional neurodegenerative indications.

AcuraStem harnesses advanced cellular reprogramming and artificial intelligence technologies to transform the standard of care for neurodegenerative diseases through patient-specific treatment. Go to acurastem.com to find out more.

03/02/2026

At AcuraStem, we believe that to truly understand human disease, we must start with human cells. Our iNeuroRx® platform builds individual patient models from human iPSC-derived neurons, letting us study the shared biological pathways that drive both genetic and sporadic forms of ALS and FTD in the people who actually have them.

Our mission is to ensure that neurodegeneration is no longer a diagnosis without a cure.

Learn more about our mission at acurastem.com.

02/23/2026

Want to move from discovery to clinical trials on a shorter timeline?
Start by optimizing in human neurons early.

That’s how AcuraStem uses our proprietary iNeuroRx® platform to advance ALS and FTD drug candidates with speed and confidence.

Learn more at acurastem.com

02/20/2026

AcuraStem’s therapeutic pipeline features multiple cutting-edge programs for ALS and FTD discovered using our patient-based iNeuroRx® platform, the gold standard for discovering novel, effective and broadly acting treatments. We are advancing drug candidates that clear toxic protein buildup and correct RNA processing errors to restore healthy neuron function.

Explore our therapeutic pipeline at acurastem.com

02/10/2026

Nearly all ALS patients (about 97%) share a common disease feature: mislocalized TDP-43 protein aggregates. Instead of chasing one-off genetic mutations, AcuraStem targets these shared disease drivers using patient-derived neuron models to develop therapies with broad impact across ALS and FTD.

Learn more at acurastem.com

02/02/2026

If you want a therapy that can move the needle for more than a narrow subgroup of ALS and FTD patients, you have to start with patients.

AcuraStem’s iNeuroRx® platform creates patient-derived human neurons and supporting cells.
Then we measure disease biology in human cells and advance therapies built for the broader ALS and FTD community.
Learn more at acurastem.com

01/24/2026

The biggest takeaway from the 16th ALS Research Summit?

The mouse model era is over.

Our presentation on SYF2 demonstrated that we can rescue neuronal function by targeting the spliceosome directly in human cells, and close the gap between discovery and the clinic.
The future of ALS treatment is patient-derived.

Follow our progress at acurastem.com

*OTherapeutics

01/23/2026

A result that looks “effective” is useless if it’s unsafe in human neurons. iNeuroRx® is AcuraStem’s patient-derived human neuron platform and the gold-standard for testing efficacy and safety in the cells most relevant to neurodegenerative disease.

Learn more at: acurastem.com

01/22/2026

Today at the 16th ALS Network Research Summit in Pasadena, AcuraStem Associate Vice President, Discovery and Early Development Dr. Wen-Hsuan Chang is presenting new research on SYF2-targeting A*Os.

In a joint presentation with Justin Ichida's lab at USC, Wen will detail how suppressing SYF2 can restore normal TDP-43 function. This work is powered by the iNeuroRx® platform, the gold standard for discovering novel, effective, and broadly-acting treatments. By leveraging patient-derived neurons, the platform enables these scientific findings to translate directly into potential therapies.

Stop by our poster sessions to learn more about our patient-based ALS models and A*O therapies. AcuraStem is on-site and ready to collaborate.

Find out more at acurastem.com

*OTherapies

01/15/2026

Behind every AcuraStem breakthrough is a team focused on turning insights from patient-derived models into treatments for people living with ALS and FTD. In 2026, we continue to advance work built on iNeuroRx® and antisense approaches focused on key drivers of neurodegeneration.

Stay informed on AcuraStem news and opportunities: acurastem.com/ -form

01/06/2026

iNeuroRx® builds cellular disease models derived from patients with ALS and FTD so AcuraStem can study human neurodegeneration directly in the lab. These models help our team find high-value targets and design antisense therapies, including programs focused on genes such as UNC13A.

Explore the gold standard for discovering novel, effective and broadly acting treatments at acurastem.com

01/02/2026

AcuraStem is delivering high-impact ALS therapeutics.
Studying disease in neurons from real patients helps us discover targeted therapies that are more likely to work across diverse forms of neurodegenerative disease.

Learn more at acurastem.com

12/24/2025

In 2025, support from clinicians, advocates, and research partners helped AcuraStem use iNeuroRx® to expand patient-derived ALS and FTD models and keep its antisense programs, like UNC13A and SYF2, moving toward clinical trials.

Thank you for backing patient-based science focused on faster, more effective treatments for people living with ALS and FTD. Warm holiday wishes and a hopeful new year from team at AcuraStem.

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Monrovia, CA

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