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.

06/17/2026

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.

03/17/2026

ALS has resisted treatment for a long time partly because the biology is not simple. When TDP-43 breaks down in a neuron, it triggers a cascade: toxic aggregates accumulate, a gene called UNC13A loses the ability to support synaptic function, and splicing errors spread across hundreds of other genes. That cascade plays out in nearly 97% of patients, familial and sporadic alike.

AcuraStem's UNC13A program targets the splicing error that cuts off a protein the synapse depends on. The SYF2 program addresses the wider splicing disruption across the hundreds of other genes that TDP-43 loss affects. Both were developed in patient-derived neurons, targeting mechanisms that animal models were never going to show us.

Read about the science behind each program at acurastem.com/therapeutics.

03/10/2026

🚨 Latest News:

AcuraStem has been awarded a two-year Drug Discovery Consortium grant from Target ALS to advance therapeutics targeting SYF2, a recently identified regulator of TDP-43 function.

TDP-43 dysfunction is present in approximately 97% of ALS cases, including sporadic disease, and remains without an approved therapy that directly addresses it. In preclinical ALS models, suppressing SYF2 restores normal TDP-43 activity and protects motor neurons from degeneration, representing a potential therapeutic path for the broad ALS patient population.

The grant will fund mechanistic and translational studies in collaboration with:

Philip C. Wong, Ph.D., Johns Hopkins University, specializing in TDP-43 RNA splicing and fluid biomarker development Wilfried Rossoll, Ph.D., Mayo Clinic Jacksonville Inc., specializing in proteomics and neuroproteostasis in TDP-43 proteinopathy

Their expertise, combined with AcuraStem's iNeuroRx® patient-derived motor neuron platforms and SYF2-targeted antisense oligonucleotide program, will focus on defining how SYF2 modulation reshapes TDP-43 biology in ALS neurons and establishing the mechanistic foundation for clinical development.

Every experiment, every late night reviewing data, every iteration on the science that got us to this point was carried by a team that believed this work could make a difference for ALS patients. This grant exists because of them:

Philip Wong
Wilfried Rossoll
Justin Ichida
Marcel van der Brug
Wen-Hsuan (Wen) Chang
Zhihua Feng
Shu-Ting (Michelle) Hung
PhD Emily Lee

Thank you to Target ALS for your continued investment in ALS drug discovery.

Full press release at: https://pxllnk.co/TargetALSGrant

03/10/2026

Why is the scientific community so focused on UNC13A?

In nearly 97% of ALS cases and roughly half of FTD cases, the loss of nuclear TDP-43 triggers a critical error in the UNC13A gene. A cryptic exon gets inserted into the messenger RNA, carrying a premature stop signal that halts production of the proteins essential for healthy synaptic communication. When those proteins disappear, neurons lose the ability to function properly and eventually degenerate.

Our team has identified A*O candidates, using the iNeuroRx® platform, designed to block the inclusion of that cryptic exon and restore the neuron's natural function. The work happens in human patient cells, which means it reflects what is actually happening in the 90% of patients whose disease has no known genetic cause.

Explore the science behind our UNC13A program at acurastem.com/therapeutics.

*O

03/04/2026

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.

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