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.

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 A*O 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 A*O 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 A*O 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 A*Os 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 A*O 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 A*Os 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 A*O can restore TDP-43 function.

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

More on the science at acurastem.com

04/28/2026

🧬 LATEST NEWS 🧬

AS-241 is moving toward the clinic.

CIRM has awarded AcuraStem $7.5 million to advance AS-241 toward first-in-human testing in ALS and FTD.

CIRM's review is widely regarded as one of the most rigorous in translational science, and their backing is a meaningful validation of the program. The funding directly supports the work that will carry AS-241 into a Phase 1 clinical trial.

AS-241 is an antisense oligonucleotide designed to address TDP-43 pathology, which is present in approximately 97% of ALS patients regardless of underlying genetic cause. It targets a major consequence of TDP-43 pathology, and seeks to restore UNC13A function with the goal of restoring healthy synaptic communication across the broad ALS population, including the large majority of patients with sporadic disease.

The preclinical data behind this award came from our iNeuroRx® platform. AS-241 restored normal UNC13A expression and improved synaptic function in ALS patient-derived neurons, supported by in vivo studies showing safety and durable central nervous system exposure.

We're grateful to CIRM for the support, and to the patients, investigators, and partners who have shaped this program from the beginning.

Read the full announcement at: https://pxllnk.co/CIRMaward

04/20/2026

AcuraStem's scientific roots in Europe run deep.

Starting this month, so do our operational roots.

AcuraStem P2 Ltd, our new subsidiary in Nicosia, Cyprus, gives us a formal home on the continent as we push AS‑241 toward first‑in‑human testing.

Europe is where some of the world's most deeply phenotyped ALS patient cohorts and most coordinated clinical research networks already live, and many of our academic collaborators and clinical investigators have been there since day one.

A base inside the EU regulatory framework streamlines how we engage with regulators, funding programs, and the partners we've worked with for years.

Read the full announcement at: pxllnk.co/AcuraStemEurope

03/23/2026

The NIH, DOD, and CIRM receive thousands of grant applications each year and fund the ones that demonstrate the strongest science. AcuraStem has received peer-reviewed grant awards from each institution across multiple funding cycles.

Those awards reflect the strength of the iNeuroRx® platform and the rigor of the science behind our UNC13A and SYF2 programs, both developed in patient-derived neurons and designed to address the biological mechanisms that drive disease in the vast majority of ALS and FTD patients, regardless of genetic background.

Learn more about our work at acurastem.com.

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