AcuraStem

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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.

10/27/2026

Breaking News: AcuraStem has been awarded a $1 million Hoffman ALS Clinical Trial Award from The ALS Association. Funding from this two-year grant will support the critical biomarker strategy for the upcoming first-in-human Phase 1 trial of AS-241, our lead UNC13A candidate.

Motor neurons rely on the UNC13A gene to maintain healthy synaptic signaling. Cryptic splicing disrupts this process, stripping neurons of an essential protein. AS-241 is designed to correct that splicing error and restore healthy UNC13A protein levels. Our approach addresses the root biology relevant to the 97 percent of people living with ALS driven by TDP-43 pathology.

Scientists at AcuraStem developed AS-241 using our iNeuroRx platform, which models the disease directly in human patient-derived motor neurons. Financial backing from major funders continues to accelerate our clinical timeline. California Institute for Regenerative Medicine (CIRM) awarded AcuraStem a $7.5 million grant in April to advance AS-241 toward first-in-human testing.

Target ALS also provided a Drug Discovery Consortium grant advancing our separate program targeting SYF2, another regulator of TDP-43 function. Growing conviction among leading philanthropic and public funders affirms our strategy to deliver broadly acting treatments to patients.

Read the full announcement here: https://acurastem.com/blog/acurastem-receives-hoffman-als-clinical-trial-award-support-phase-1-trial-241-clinical-candidate-its-unc13a-program

10/09/2026

AcuraStem's Associate Vice President of Discovery and Early Development, Dr. Wen-Hsuan Chang, is presenting our latest antisense oligonucleotide (ASO) data at the 22nd Annual Meeting of the Oligonucleotide Therapeutics Society.

Wen will be in Denver from October 11 to 14 and will present poster P-026 on AcuraStem’s SYF2 ASO program for ALS and frontotemporal dementia (FTD).

The poster highlights AcuraStem’s integrated capabilities in ASO discovery and development and human iPSC-based disease models for ALS and related neurodegenerative diseases.

This is a direct opportunity to review the NeuroRx platform findings and discuss AcuraStem’s groundbreaking science with her in person.

Leave a comment below if you will be at OTS. We’d love to connect.

More details at: https://www.2026oligomeeting.com/

09/21/2026

Last week, AcuraStem CEO Sam Alworth was in Washington, D.C., meeting with congressional offices to discuss biomedical research, federal research funding and our work developing treatments for ALS and frontotemporal dementia.
We appreciate the opportunity to share our progress, discuss the research funding process and continue conversations about the path from scientific discovery to potential treatments for patients and families.
Thank you to everyone who made time to meet with us.
(AcuraStem CEO Sam Alworth with government affairs advisors Dave Larson and Jim Link during a week of meetings on Capitol Hill)

09/03/2026

🚨 Latest News 🚨

Genetic Engineering & Biotechnology News (GEN) recently highlighted AcuraStem’s therapeutic strategy targeting TDP-43 pathology in a feature on oligonucleotide drug discovery.

AcuraStem's research centers on addressing RNA dysregulation, a primary consequence of TDP-43 dysfunction implicated across ALS and several forms of dementia. Our team utilizes transcription factor-mediated lineage conversion to transform patient-derived induced pluripotent stem cells directly into induced motor neurons.

Direct conversion preserves original donor aging characteristics alongside authentic disease pathology, reproducing disease mechanisms cleanly without artificial stressors or genetic manipulation.

Integrating patient-derived motor neurons into our iNeuroRx platform allows precise design, screening, and pharmacological evaluation of antisense oligonucleotides. GEN spotlighted our work on the SYF2 gene, an ASO target showing strong capability to restore TDP-43 localization and function.

AcuraStem CSO Marcel van der Brug is featured and quoted directly in the publication, sharing his perspective on how intercepting downstream RNA errors alters the trajectory of neurodegeneration.

Read the complete article here: https://www.genengnews.com/topics/drug-discovery/rewriting-disease-oligonucleotides-take-aim-at-the-untreatable/ then head to acurastem.com to learn more about the next-generation of drug discovery.

08/18/2026

The AcuraStem team is heading to Boston from August 19 to 21 to help drive the conversation at the 6th Annual Oligonucleotides for CNS Summit.

The annual Oligonucleotides Summit provides an essential venue to exchange the latest scientific discoveries and explore new avenues for collaboration in central nervous system drug development.

Our VP of Discovery & Early Development, Wen-Hsuan Chang, presents on Friday, Aug 21, 12:00 PM: "A Human-First Translational Package for CNS Antisense Oligonucleotides: From Patient Neurons to First-in-Human." The presentation will show how AcuraStem uses a human-first preclinical package (patient iPSC-derived neurons, then humanized mice, then NHP PK/PD) to de-risk ASO development for our lead ALS program, AS-241.

Earlier this year, the California Institute for Regenerative Medicine awarded AcuraStem $7.5 million to advance our lead candidate AS-241 toward a first-in-human trial, a decision that follows one of the most rigorous scientific and translational reviews in the field.

Wen is in Boston for three days only. If you are attending the 6th Annual Oligonucleotides for CNS Summit, please reach out to connect, and to learn more about our science head to acurastem.com

06/23/2026

AcuraStem is looking forward to joining the European Network to Cure ALS community in Madrid from June 24 to 26 for ENCALS 2026.

The ENCALS congress provides an essential venue to exchange the latest scientific discoveries and explore new avenues for collaboration in ALS research.

Our Co-founder and CEO Sam Alworth and Chief Scientific Officer Marcel van der Brug will be attending, and are excited to connect and discuss our recent breakthroughs and strategic priorities:

➡️ Advancing ASO programs designed to restore TDP-43 function
➡️ Translating unbiased screens of patient-derived motor neurons into viable therapeutic targets
➡️ Evaluating the impact of partial SYF2 suppression in TDP-43 driven ALS
➡️ Building partnerships that support the translation of early discovery into clinical progress

In April, the California Institute for Regenerative Medicine awarded AcuraStem $7.5 million to advance our lead candidate AS-241 toward a first-in-human trial, a decision that follows one of the most rigorous scientific and translational reviews in the field.

Sam and Marcel are in Madrid for those three days only…If you are attending ENCALS 2026, please reach out to connect and to learn more about our science head to acurastem.com

https://encals-congress.com/ENCALS2026

05/08/2026

A great few days at Target ALS 2026 in Boston.

Our CSO Dr. Marcel van der Brug presented "Dose-Response of SYF2-Targeted ASOs in Diverse ALS Patient-Derived Models" the first of three talks from our Target ALS-funded consortium, "Advancing SYF2 Antisense Oligonucleotides to Restore TDP-43 Function."

Huge thanks to our consortium collaborators Dr. Philip Wong (Johns Hopkins University) and Dr. Wilfried Rossoll (Mayo Clinic), whose biomarker and interactome work complements our patient-derived dose-response studies, and to Target ALS for the funding and for convening the field.

To everyone who stopped by and engaged with the work: thank you. Those conversations sharpen the path forward.

Follow our progress at acurastem.com

05/04/2026

AcuraStem is heading to the 2026 Target ALS Annual Meeting in Boston, May 5–7.

We're thrilled to be funded by Target ALS through the New Drug Discovery Consortia program for our SYF2 ASO project, "Advancing SYF2 Antisense Oligonucleotides to Restore TDP-43 Function," and to be presenting alongside our consortium collaborators Dr. Philip Wong (Johns Hopkins University) and Dr. Wilfried Rossoll (Mayo Clinic).

On May 5, our CSO Dr. Marcel van der Brug will give the first of the three consortium talks: "Dose-Response of SYF2-Targeted ASOs in Diverse ALS Patient-Derived Models." Dr. Wong will follow on multimodal biomarker profiling, and Dr. Rossoll will close on the SYF2-dependent TDP-43 interactome and proteostasis networks.

SYF2 emerged from an unbiased screen of patient-derived motor neurons as a top modifier of survival specifically in TDP-43 driven ALS. Together, the three projects test whether partial SYF2 suppression with our ASO can restore TDP-43 function.

Grateful to Target ALS for the support and for convening the field.

More on the science at acurastem.com

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http://www.acurastem.com/

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